MPN Newly Diagnosed Archives

Your MPN diagnosis is just a starting point. Even though the path ahead may seem unclear or even insurmountable, armed with knowledge you can take control.

Let us help you become empowered to understand your diagnosis, to confidently ask questions, and to identify providers that are the best fit for you.

More resources for Myeloproliferative Neoplasms (MPN) Newly Diagnosed from Patient Empowerment Network.

MPN Patient Story: Ruth Gerwin

My journey began in 1999 when I was diagnosed with Essential Thrombocythemia (ET). All I took was a baby aspirin, even then they [platelets] soared to over 1 million.

In November 2004, I had a bad cold and had this aching on my left side. It was discovered my spleen was enlarged and I had a bone marrow biopsy. I was at that time diagnosed with Myelofibrosis (MF). I went to see Dr. Richard Silver in New York and he put me on Interferon. I saw him for 5 years and then transferred to Cleveland Clinic as my insurance company was making it harder and harder for Dr. Silver to be paid. There I was under the very capable care of  Dr. Ramon Tui. It was under his care that I did a trial for Jakafi. It only helped the spleen size for a couple of months, but it has kept some of the other side effects of the disease at bay. I still take 20 mg. twice daily. Also, in 2014 I had a double mastectomy.

In the spring of 2017, I was so horribly uncomfortable because by this time I looked 9 months pregnant with my spleen. I also had swollen legs and feet. I could hardly walk. I made a decision at that time to radiate the spleen to give me some relief. I was supposed to receive 10 treatments, but was stopped at 7 because my blood counts bottomed out. Hmg 6.0, Pl 5, WBC 0.8. I started with transfusions twice weekly of one platelet and two blood. I did this for several weeks and developed a horrible headache. I stopped the transfusions and my Dr. said to go home and call Hospice. He thought I had 2 weeks to 2 months to live. I was really sick, but as my spleen began to recover, my counts went up. By the fall of 2017, I was basically back to normal with the blood counts and, of course, out of Hospice. My family think I’m a miracle. But, the spleen, by December 2017 was becoming very uncomfortable again and I started radiation again January, 2018. This time I had 4 treatments and had to stop because of my blood dropping.

It has been suggested to me by two doctors to have my spleen removed and have a bone marrow transplant. But, I have read about this procedure and I know I wouldn’t survive as I am very sensitive to most of the medications they would have to give me. My current hematologist is looking for a trial I can do, but my bone marrow is nothing but fatty tissue. I have nothing there…not even fibrosis. I keep telling them my spleen is doing it all, but they won’t believe me. With no bone marrow tissue, I can’t do a trial. So, I don’t know what they are going to do with me. Anyone else have this problem? I’d love to hear what you are doing.

I know the Lord has a good plan for me and I just have to wait and see what it is. He is the “great physician”!  I’m just not real patient. I haven’t felt really well for a long time.

Uber Health App

The Community Transportation Association estimates that approximately 3.6 million Americans miss or delay medical care because of transportation issues that cost the health care system $150 billion each year.

To help combat this issue, Uber has created a new app called Uber Health. Earlier this month Uber announced that they are working with providers to offer reliable rides for patients, care partners, and families to get to and from doctor’s appointments and the hospital.

The app will allow medical and administrative staff to either call an Uber to drive a specific patient home, or to dispatch an Uber to the patient’s house for pick up. The app also allows users to schedule the ride up to 30 days in advance, so important appointments are never missed. Planning transportation in advance enables patients to schedule rides to and from follow-up appointments even while they are still in the healthcare facility. With the ability to schedule and manage multiple rides from a single dashboard, healthcare professionals can take their level of care to the next level with Uber Health.

How It Works

Uber Health saves patients time and money, as they can focus their attention on their health instead of worrying about how they might get to their next appointment. With the help of Uber’s cost-saving methodology, patients and healthcare professionals can save money utilizing the app over hailing taxis or paying for expensive hospital parking.

Uber Health enables older patients and those with chronic pain gain independence and mobility. Because all communication with Uber Health is completed via text message, patients no longer need a smartphone and the corresponding Uber app to access Uber Health’s benefits.

The Uber Health dashboard was designed with HIPAA standards in mind, ensuring that all aspects of the service meet health care privacy and security standards.

As a part of Uber’s beta program, over 100 healthcare organizations in the U.S, including hospitals, clinics, rehab centers, senior care facilities, home care centers, and physical therapy centers are already using Uber Health.

Dashboard

 

For more information, please visit the Uber Health site: https://www.uberhealth.com

Patient Advocacy: Understanding Your Illness

The news that you, or a loved one, has a serious illness can be a terrible blow.  You may be faced with an array of emotions ranging from shock to fear to anxiety. You will likely have many questions and concerns about what the coming days and months will bring, and the impact living with this illness will have on your life and the lives of your family. Although you may be reeling from the news, it’s important that you learn as much as you can about your diagnosis, its symptoms, how it may progress and what treatment options are available. In this article, you will learn which questions you should ask your healthcare team and where to find reliable and trustworthy information to become better informed about your health condition.

1. Obtaining Information From Your Doctors And Healthcare Team

Having answers to your questions can help you understand your illness better and feel more in control about your treatment decisions.  How much information you want is up to you. Some patients feel overwhelmed by too much information at this stage.  Others say they didn’t receive enough information.  While information upon first diagnosis is vitally important, you may be in shock and in a heightened emotional state which makes it difficult to fully comprehend all the information you are given. If possible, bring a trusted friend or family member to appointments with you to take notes. If this is not possible, ask your doctor if you can record the consultation so you can focus on listening, and go back and review what was said later.

Medical care is a conversation and to have influence in that conversation you have to speak up. Never be embarrassed to tell your healthcare team if you don’t understand something they’ve said. Sometimes doctors use medical jargon without realizing they are not explaining things in terms we understand. Repeat what the doctor has told you to be sure you understand and ask for clarification if needed.

Some questions to ask your doctor about your diagnosis:

  • What are the symptoms of this illness?
  • What should you do if you notice new symptoms or if existing symptoms worsen?
  • Do you need any further diagnostic tests?
  • What are your treatment options?
  • What are the side-effects of the recommended treatment?
  • What are the benefits vs the risks?
  • What happens if you do nothing?
  • Are there other treatment options available?

Finally, ask your healthcare team if they can recommend further reading, support groups and other resources to help you learn more about your illness.

2. Finding Reliable Information Online

As you move along the patient journey and better understand your illness, you may want higher levels of information. However, you may find the information healthcare professionals provide has not keep pace with your increased needs. This is the point where many patients turn online to seek more information. While the Internet can be a useful source of health information, it’s important to know how to critically evaluate the information you find online. Always discuss what you find with your healthcare team and ask them to put the information into context for your particular situation.

Here are some questions to help you determine the trustworthiness of online sources of information.

  • Who has produced the information?
  • Does the organization have commercial interests or another reason they are promoting this information?
  • Is the name of the organization and their aims in setting up the website clearly shown?
  • Does the site provide contact details if you have any questions?
  • Is the information on the website up to date?
  • Does it cite the source of the information that is being presented?
  • Does the site link with other reputable sites that give similar information?

3. Evaluating Medical News Reports

Whether it’s published in hard copy or online, medical news reports can mislead people into thinking a certain drug or treatment is the next breakthrough in a disease.  As patient advocates we must learn to read beyond the headlines to filter out the good, the bad, and the questionable.

The following questions will help you evaluate the reliability of medical news reporting.

  • Does the article support its claims with scientific research?
  • What is the original source of the article?
  • Who paid for and conducted the study?
  • How many people did the research study include?
  • Did the study include a control group?
  • What are the study’s limitations?
  • If it’s a clinical trial that is being reported on, what stage is the trial at?

Always try to read an original study (if cited) to critically evaluate the information presented. Understanding research literature is an important skill for patient advocates. For tips on how to read a research paper click on this link.

4. Learning From Peers

From helping us to uncover a diagnosis and finding the right doctors and treatments, to learning about everyday coping tips, turning to our peers can make all the difference in how we live with our illnesses.  Much of this peer-to-peer learning takes place through social media discussions on patient blogs and in Facebook groups and Twitter chats. On Facebook you can connect with other patient advocates and join Facebook groups related to your disease or health condition. On Twitter you have a greater mix of patients, physicians, healthcare professionals and medical researchers coming together to discuss healthcare matters. It is becoming increasingly popular for attendees at key medical conferences, such as ASCO, to “live-tweet” sessions. You can follow along on Twitter using the conference hashtag which you should find published on the conference website. Another way to learn on Twitter is to join a Twitter chat related to your health condition. Twitter chats can be one-off events, but more usually are recurring weekly chats to regularly connect people. There are chats for most disease topics and a full list can be found by searching the database of the Healthcare Hashtag Project.

Final Thoughts

Understanding your illness is the first step on the path to advocating for yourself and others.

Being an advocate involves asking lots of questions, conducting your own research, and making your preferences known to your healthcare team. By doing this, you will be better informed and in a stronger position to get the treatment that is right for you. If this feels overwhelming to you right now, go at your own pace, and reach out to others who have walked this path before you. There is an army of patients who are standing by, ready to share their healthcare wisdom and practical coping tips with you. Seeking their advice will help lessen the fear and isolation you may be feeling, give you a sense of shared experience and connection, and help you feel more in charge of your healthcare decisions.

MPN Patient Cafe® October 2017 – Tips for Managing Life After Diagnosis

MPN Patient Cafe® – Taking Back Control – Tips for Managing Life After Diagnosis from Patient Empowerment Network on Vimeo.

A panel of MPN patients and care partners discuss taking back control and share their tips for managing their new normal after diagnosis.

Fact Checking 101: Health Literacy in Real Time

There’s a medical miracle every day, if you believe headlines on popular media sites. If you just read those headlines, cancer is cured daily, as are hepatitis C, and a host of neurological conditions. Dive into the stories, though, and you’ll all too often find the “in mice” red flag, meaning that scientific experiments have indicated that mice are having terrific outcomes from whatever substance is being touted. Humans? Not so much.

Information flows at the speed of life – thank you, Internet – but information does not always equal factual truth. Which is where fact checking comes in, and what I’ll be offering tips on here. As a journalist, I’ve hunted down confirmations on stories for years – here’s a quick primer on doing it for your own health/science literacy building.

  • Snopes.com: this site is the granddaddy of online myth busting. They have a dedicated channel for health news, which is definitely a good first stop to fact check a headline touting a “cure” for an illness or condition.
  • Sense About Science USA: the US arm of the UK-based Sense About Science and AllTrials, this site takes a deep dive into advocacy and literacy building for both the public, and professionals, around medical science. They’re in the process of creating an AllAccess Patient Guide on clinical trial participation, and transparency in reporting on all trials, which will be published in the fall of this year (2017).
  • Health News Review: the editors and reviewers behind this site are professional healthcare journalists dedicated to reading and scoring the reporting on health science in major media. I think of them as Politifact For Healthcare – they don’t issue “pants on fire” or “Pinocchio” warnings, but their 5-star review system is rigorous, and great reading.
  • FactCheck.org and FlackCheck.org: these sites assess news stories and sources in many categories, from politics to science to health policy. They’re produced by the Annenberg Public Policy Center at the University of Pennsylvania, and are great resources for fact checking in all news categories, not just science.
  • Retraction Watch: this is in the Super Science Nerd Journalist zone, covering the retraction of scientific papers around the world. There’s an old news adage about corrections being buried deep beneath the front page – that rule goes double in science publishing. A paper is published, and makes big headlines. If it’s retracted weeks/months/years later, there’s seldom a screaming headline announcing the retraction, leaving the untruth out there to be misunderstood and often misused.

Building your own health and science literacy is a process. Reading the latest medical science news is a starting point, but you have to add fact checking as a critical part of your learning curve. Then use the “see one, do one, teach one” method to help your friends and family build their health literacy, teaching them how to find and fact check the science news that matters – that’s how we all build healthy, science-literate communities.

MPN Patient Cafe® July 2017 – Finding Trustworthy Information

Patient Cafe® MPN – July 2017 from Patient Empowerment Network on Vimeo.

Carol Preston is join by MPN patient, Andrea, and her care partner, Denise in this session of the Patient Cafe®. They discuss where MPN patients and their care partners can go to find trustworthy information.

Living Well with MPNs – What You Should Know About Genetic Mutations

What You Should Know About Genetic Mutations

Living Well With MPNs: What You Should Know About Genetic Mutations from Patient Empowerment Network on Vimeo.

Should you get a genetic test? JAK2, MPL, CALR, ASXL1: Mutations associated with myeloproliferative neoplasms (MPNs) can bring up lots of questions. What do they mean, and how do they impact your disease? The goal of this webinar, featuring Dr. Alison Moliterno from Johns Hopkins School of Medicine and Dr. Stephen Oh from Washington University School of Medicine, is to help patients understand genetic mutations.


Transcript:

Andrew Schorr: Hello and welcome to this Patient Empowerment Network program, produced by Patient Power. I’m Andrew Schorr joining you from Carlsbad, California. Over the next hour we’re going to talk about something that’s very personal to me and probably to you, and that is the whole idea of genetics related to living with an MPN. What does it mean? Does it change over time? What is your version of an MPN? What does it mean for prognosis? What does it mean for treatment? What does it mean for clinical trials opportunities? So we’re going to be discussing all of that.

I want to thank our sponsor, Incyte Corporation for supporting this educational activity. We’re going to cover the country and we invite your questions as we go along. Send them to MPN@patientpower.info. Many of you have. And remember if you have to bow out at some point, the replay will be available and we’ll have video clips. So we’ll reach literally a few thousand people living with MPN worldwide, and we’re happy to do that.

Let’s get started. First I want to introduce you to one of our medical experts who’s joining us. He’s been on programs before. He joins us from Washington University and the Siteman Cancer Center in St. Louis; that’s Dr. Stephen Oh, who is an MPN expert there. Dr. Oh, thank you so much for joining us.

Dr. Stephen Oh: Hi, Andrew. Thanks for having me.

Andrew Schorr: Thank you, Stephen. Okay, let’s go from St. Louis to Baltimore, Maryland and the Johns Hopkins University Medical Center and another expert in MPNs who’s been with us before, and that is Dr. Alison Moliterno. Dr. Moliterno, thank you so much for being with us today.

Dr. Alison Moliterno: Thank you, Andrew. Thank you for having me.

Andrew Schorr: Sure, pleasure. We’ve got a lot to cover. And then also I want to welcome back one of the members of our community, someone who was diagnosed many years ago, a couple of decades ago with ET, and then a year ago it became myelofibrosis. She’s a preschool teacher in Peoria, Illinois. She’s been with us on our programs before; Marsha Krone. Marsha, thank you for being with us once again.

Marsha: Thank you. Hi, Andrew.

Andrew Schorr: Marsha, let’s visit for a minute. I was diagnosed in 2011 and then eventually had a genetic test which came up with a bunch of results. One of them for me, if I get it right, JAK2V617F.

I had no idea what that was, and then a couple of other genes that to me seemed kind of like alphabet soup. And I had one of the peers of these experts here, Katrina Jamison. We went over it and she said I think it’s the JAK that’s kind of driving things. We’re going to talk about what are the driver genes, and what may not be, or what do we know at this point. Marsha, you had a genetic test, too. What did it say?

Marsha: My genetic test came out as Calreticulin type 2.

Andrew Schorr: Okay, so we’re going to figure out what that means. Dr. Oh, let’s start this way. People think genetics. I know genetics maybe had something to do with being bald, and I see you have a similar hairline; or dark hair, or brown eyes, or whatever. Hereditary genes; are we talking about that or are we talking about something different?

Dr. Stephen Oh: That’s a basic question that comes up with almost every new patient that I see.

The short answer is that when we’re talking about genetics with relation to the MPNs, we’re not talking about those kind of things that you’re born with that may affect your hairline and whatnot. These are genetic mutations that are acquired over time that you’re not born with; they’re not passed down to your children or your relatives, etc. So that is a very important distinction that I try to make clear with every new patient that I see.

Andrew Schorr: Okay, and one follow up question to you. People say okay, Doc, what gave me this genetic injury, if you will, to lead to these illnesses? Do we know?

Dr. Stephen Oh: That’s another question I cover with almost every new patient; that I think for that one I guess the answer is a little less satisfying. My answer is that for the most part it’s random chance.

What I mean by that is that we know particularly from research that’s come out n the last five or so years that all of us acquire mutations randomly over time as we age. But fortunately for most individuals, those mutations land in spots where they really have little to no consequence. But for those that, for instance, acquire the JAK2V617F mutation or acquire a Calreticulin mutation, that really becomes most likely the main driver for what ultimately becomes an MPN.

Patients of course ask did I do something wrong, was I exposed to something? And while we can’t necessarily exclude that those are factors, I think for the most part it’s that just kind of randomly these mutations landed in the wrong place.

Andrew Schorr: Dr. Moliterno, we’ve mentioned a couple of these onco genes I think you call them – cancer genes, the JAK2 gene and Calreticulin type 2, I didn’t even know about that.

Can you first of all rattle off some of them just so we know the landscape of what are genes that seem to be associated with MPNs today, knowing that this will probably expand?

Dr. Alison Moliterno: I always like to tell patients a little bit about the history when these were first diagnosed because we talk about them now as if they’re common knowledge, but they are really quite recent in our understanding. We’ve known about the myeloproliferative diseases for more than 100 years, but it wasn’t until 2005 until the driver of many of the diseases was understood to be JAK2V617F. So that discovery occurred in 2005. Before the JAK2 discovery, we didn’t understand really if it was acquired mutations and what genes were involved. JAK2 is the most common of these.

If you look at 100 patients with the classical MPN, meaning PD, ET, and myelofibrosis, 75 percent overall of those individuals will have the JAK2V617F mutation. Not long after the JAK2V617F mutation there was a discovery in mutations in M-P-L or MPL. That accounts for about 5 percent of those 100 individuals with either ET or myelofibrosis and then in 2013, that was the discovery of the calreticulin mutations that comprise about 20 percent of individuals who have ET or myelofibrosis. So 2013, that’s fairly recent and those are the three drivers.

I like to say that if you could put the same mutation, the JAK2V617F or Cal or MPL mutations that we see in our patients, if you could put those, say, in a mouse; they would drive a similar disease in the mouse so that you get polycythemia vera in a mouse if you make the mouse have the V617F mutation. That’s how we’ve kind of come to understand that they drive the disease. They may not drive all aspects of it but they drive the basic process.

Andrew Schorr: I know there’s another gene that people have seen too; ASXL1. What is that one?

Dr. Alison Moliterno: In addition to these drivers we’ve also discovered a lot of genes that seem to modify the MPN or associate with certain subtypes of MPN. So for instance I said usually JAK2 MPL or CALR can all drive platelet count high and give a disease like ET. Then what happens when patients develop myelofibrosis? We find that perhaps other lesions are acquired.

Those are genes that don’t drive myeloproliferation so much but they seem to drive the way that the chromatin or the nucleus is managed; they seem to maybe set up the other aspects of MPN that associate with myelofibrosis. And ASXL1 is probably the most common additional genetic lesion or acquired mutation that occurs in individuals with myelofibrosis.

Andrew Schorr: Okay. Dr. Oh, people may be tested and we’re going to talk about who should be tested and when. How do you know what’s the driver gene? And it sounds like this continuation of identifying genes just keeps going, too.

Dr. Stephen Oh:Certainly Dr. Moliterno gave a nice circle overview of when the three primary driver mutations were discovered.

We’re sort of lucky now that in today’s day and age we kind of look at this now as standard testing. So JAK2, MPL and calreticulin so much so that many physicians including myself, we kind of go about this in an algorithmic fashion. So for instance if I have a patient with newly diagnosed myelofibrosis, I’ll start by screening for the JAK2 mutation; if that’s negative, go to Calreticulin and if that’s negative go to MPL.

And so with those three genes, the majority of patients with any of the three main MPN subtypes, whether that’s QV, ET, or myelofibrosis; they’ll be positive for one of those three. There’s a subset that’s at least on the order of 10 to 15 percent of patients with ET and myelofibrosis who will be negative for all three of those mutations, what we’re now calling the triple negative category. But the vast majority of MPN patients will have one of these three mutations which we consider the main driver of mutations.

So, in some sense again we’re sort of fortunate that it’s become almost straightforward in terms of at least the top level genetic testing for these diseases.

Andrew Schorr: So that was my question to you, Dr. Moliterno. Some people have maybe had fights with their insurance company or their doctor as related to testing. How do you view this now and how could it be positioned on how it’s really not elected, if you will, but essential to get a clear picture of an individual patient’s situation?

Dr. Alison Moliterno: This comes up in my practice and I’m sure Dr. Oh’s practice all the time, in that in the olden days before our understanding of what causes these diseases, and again the cause was these acquired mutations and in the olden days we would use histology, looking under the microscope, looking at blood counts and sort of put a name to this polycythemia vera and myelofibrosis.

But within that was such a vast variability of what the disease actually was, that that name really did not tell us too much. Now, we’ve really come to understand that what you have is defined by these molecular lesions. They’re not just of academic interest; they actually really tell us quite a bit about what you’ve got, what your prognosis is, and where it’s going. So physicians, we really can’t function and counsel patients appropriately without this knowledge. So there’s no longer elective or of interest; it’s really critical in defining what you’ve got.

Andrew Schorr: Okay, everybody. You heard it, so this is what you’re advocating for with your MPN specialists, which hopefully you have like the two with us. This is standard operating procedure; there shouldn’t be nay question with an insurance company or anybody to help you and your doctor know what you’re dealing with

But then the next question is, Dr. Oh, where are we now even in research, where things are headed so there will be treatments that line up with the different genetic situation?

Dr. Stephen Oh: I’ll start my answer to that question by going back to when the field began to develop inhibitors of JAK2 for the treatment of patients with MPNs and extrapolating from other diseases, I think there was an assumption that the patients that would respond best or if at all to JAK2 inhibitors would be those that carry the JAK2V617F mutation. What we now know quite clearly is that even those patients that do not carry the JAK2 mutation, they also tend to respond to JAK2 inhibition. So again in the case of myelofibrosis, patients who are calreticulin mutants, they also respond to JAK2 inhibitors as well.

So that’s an example where I think in a good way the use of those kinds of treatments is not limited or defined or restricted mutational profile. But otherwise in terms of the research front, identifying or defining treatments specific to different mutations, we haven’t made a lot of headway there. For instance, you can imagine now we have identified the calreticulin mutation in many patients with MPNs; can we devise a treatment specific for that? So while again on the one hand like I mentioned, it’s a good thing that those patients do respond to JAK 2 inhibitors; can we come up with something very specific to calreticulin with the patients there?

There are certainly a number of research groups that are working on that kind of question, but we currently do not have anything specific in that regard.

Andrew Schorr: Dr. Moliterno, I want to get a question from you about that because I’m sure you’re asked. Somebody says well – Marsha could come to you and say well, I’m CALR so what do you have for me? And/or you also mentioned triple negative, and I think often I’ve heard that term in breast cancer; women who were triple negative there, and say what do you have for me in that situation? So talk to us a little bit about those situations, and does it vary now with treatment approaches yet?

Dr. Alison Moliterno: I think another aspect of this is we may not today have the specifically targeted treatment but as Dr. Oh mentioned, what we’ve learned is that these three main lesions, CALR, MPL, and JAK2 all seem to over signal or work their effects through this JAK pathway.

So even though you may not have the JAK2 mutation but you have the CALR, it seems like the end result of over signaling is in the same pathway and therefore JAK inhibitors would be beneficial in individuals who have that over signaling pathway. So that’s one thing we’ve taken away; that while even though we don’t have – and that’s a good thing because that tells us that inhibiting this pathway overall will benefit the majority of individuals with these lesions, whether they’re JAK2 or CALR or MPL.

The other thing about the profiling is how much of the mutation you have. So another I think we’ve learned is that we can measure the amount of these mutations, and that’s a variable across patients and gives us quite a bit of information about – again – the type of lesion, disease you have and gives us  better information about the prognosis and why you might have a little more fibrosis than someone else.

So again, the type of lesion you have and the amount; we’re now learning to use that information. I think another thing we’ve learned is these terms triple negative, which is a term that we understand okay, that means you don’t have any of the three drivers that we know of.

But as we’re learning more about extended mutation paneling or doing different tests to look at the entire genes, we’re finding that most of those individuals really do have lesions in the JAK2 gene, or the CALR gene or MPL that maybe aren’t in the specific areas that the test was sent for. So that’s another benefit of this revolution in being able to define these lesions personally so that your disease really can be diagnosed.

Andrew Schorr: Okay Marsha, you’ve been sitting there quietly and I wonder, is this making sense to you? You’re my co-host with this; is it making sense?

Marsha: It does make sense. When I first was diagnosed, they just said you simply have ET because you have it. Then they discovered more mutations. I like to know everything about how my body is working, so I wonder how valuable it is to have additional genetic testing to see if you have other mutations that may affect prognosis?

Andrew Schorr: Right. So Stephen, and also like serial testing at some other time? She went from ET to MF over many years, so when do you test again or how does this change?

Dr. Stephen Oh: Speaking sort of broadly at first, the more extensive genetic testing which is available through numerous laboratories now, which can test for any – quite often these panels went through 20 or 40 genes; some of them hundreds or more.

The use of those in the clinic has evolved pretty rapidly over the past three, four, five years. In my own practice five years ago I was certainly not routinely recommending that kind of testing. But today, particularly with patients with myelofibrosis, I am doing it much more frequently and the question of course is as you raised; what utility does it have in terms of prognosis.

And there especially I think in myelofibrosis, is where more and more literature has come out giving us a better handle on what effect these different mutations might have on prognosis. That’s why more and more I’m beginning to recommend this kind of testing for my patients with MF.

In the case of QV and ET, it’s a little bit further behind in terms of data to indicate what these different mutations might mean, but we have also seen more literature come out, not as often but do consider that kind of testing for those patients as well. In every case it’s kind of an individualized discussion with the patient. I always start out with how much do you want to know. Marsha said I want to know everything, so that’s the kind of patient where you say alright, well, maybe let’s do this.

Others say well, I don’t really necessarily know I’m going to interpret this; it’s alphabet soup. Do I necessarily want to know what the statistics say to expect in 20 years, etc.? Again, it’s an individualized decision with every patient.

Andrew Schorr: Dr. Moliterno, there was another patient who was going to join us who couldn’t make it because she’s being scheduled for a transplant. She’s getting her life in order for that; someone with I think myelofibrosis in the Seattle area.

But she did have three mutations identified, so let’s see if this makes sense: ALX1, if I get it right, TP53 and SFB1, if those are other ones? So the fact that she has this sort of alphabet soup, does that mean that that meant you’re headed for transplant because that shows up? You know, one knows is more – more weighty, if you will.

Dr. Alison Moliterno: This is knowledge that we’re kind of pulling in as we speak, and that the meaning of more than one mutation, different types of mutations do seem to have prognostic significance.

So as we’ve learned that generally the more mutations you have, more individuals at a certain point of time generally is concerning. It means that there’s a lot going on in that stem cell and that maybe there’s some instability to allow these mutations to occur. And then it does matter which type. Some mutations seem to have more independent prognostic than others. ASXL1 tends to be one that is seemingly more associated with developing myelofibrosis. SF3B1, one of those she has, may be a less negative prognostic indicator.

But again, these are important and having three at once is a concern, and it sounds like a reasonable plan to move forward with transplant because we understand that we don’t really have a medicine that can address all of those lesions and that this is more high risk disease.

Andrew Schorr: I want to remind our audience a couple of things. One is you’re hearing how sophisticated the testing is, and then you can imagine the interpretation.

We have two noted experts. The typical hematologist is not going to see this very often. So whether you go to Washington University, I go to UC San Diego, Marsha goes to the Lurie Cancer Center, Dr. Moliterno is at Johns Hopkins. You know some of the others where you go; you want to consult with an MPN specialist. As the data comes back, what does it mean for me now or on your journey in the future.

The other point I wanted to make is of course we want to take your questions. So send them to MPN@patientpower.info and our wonderful MPN community manager, Jamie, is standing by and she’ll be forwarding these to me. Obviously, a lot of you ask very personal questions; what should I do, Dr. Moliterno, Dr. Oh; I’ve got XYZ. And that’s not fair to do that here, so that’s our disclaimer. You want to go back to your MPN specialist – it could be one of them – to discuss it.

We did get some questions in earlier. Dr. Oh, Tammy wrote in and she said can you provide more information on being PV JAK2 negative? She says I know we’re limited in number compared to other patients. It would be nice to know the basics. How much more different are we and is our treatment any different? So, JAK negative PV.

Dr. Stephen Oh: That’s a great question and it’s a challenging question. I would say that in part because we know that in the case of PV, at least 95 percent or greater of patients with bonafide PV carry the JAK2V617F mutation and it’s at least 95 percent; it may be higher than that. And even those that are negative to the JAK2V617F mutation, there’s another small group, probably less than 1 percent, who carry an alternative JAK2 mutation, an exon 12.

 So between those two, patients with PV, again the vast majority do have a mutation in JAK2. That does leave a small sliver that you could call JAK2 negative PV, and there – it’s sort of being a skeptic – the first thing I say when I’m asked to evaluate a potential case like that is am I convinced that is the correct diagnosis? Do they truly have PV versus a potentially secondary cause of erythrocytosis?

And so there, sort of again you have to rely on the old school diagnostic criteria; do they otherwise have the features of the disease? Does the bone marrow, is it consistent with the diagnosis? Are they like a classic patient in that they have a low epo level, etc. But to get to more specifically to the question, if you’re convinced that that’s the correct diagnosis, essentially I treat the patient the same way I would treat a JAK2 positive PV patient.

In other words, I would use the same kind of treatment calls in terms of a hematocrit less than 45, treat them with cytoreductive therapy and the appropriate circumstance if they’re considered high risk, if they have had a prior history of thrombosis; aspirin considered standard therapy for these patients. Again, if I do believe that is the correct diagnosis, I essentially treat them similarly to patients with JAK2 positive PV.

Andrew Schorr: Okay, and Alison, do you concur in that?

Dr. Alison Moliterno: I do. I think we have a name for the disease, polycythemia vera, where you make too many cells due to an inherent stimulus of the bone marrow. I think now in this age, we are really coming to redefine PV as the disease that has mutations in the JAK2 gene. I’m always concerned when someone has been diagnosed with JAK2 negative PV because as Dr. Oh says, maybe they have something else.

A couple circumstances that I’ve observed is that sometimes the diagnostic testing, when it was done and it was done with JAK2V617F testing that was not sensitive enough. So again we have this issue of how much of this do you have? And some PV patients can have very low levels of the JAK2 mutation that if you use an assay in a laboratory that wasn’t very sensitive and you only detected levels of 10 or 20 percent, they would be read out as negative. That’s happened in my practice a few times where patients went high and low all over the place trying to get a diagnosis; JAK2 negative. Finally when a more sophisticated or sensitive test was done, they’re positive.

The other issue is that there are other mutations aside from V617F in the JAK2 as Dr. Oh said; there’s exon 12 and then there are some others in various parts.

So when we do some of this more expanded molecular profiling, the laboratories are able to look not just at the V617F site, which is where most tests – they only tell you yes or no at that site; but they’ll look at the entire part of the coding region of the JAK2 gene. You’ll find patients who have an unusual mutation down the way that’s like – And I think it’s important because there are some people who really don’t have a myeloproliferative neoplasm; they have something else and they’ve been told for 25 years that they have PV.

And it is nice to either make that diagnosis or not. And so I think Stephen has been in that situation also, where maybe they were labeled because that was the best information we had at the time, but they really should be reevaluated.

Andrew Schorr: And I think that’s the point for our audiences. You with the best testing, with the best specialists you can get to, you want to know what you’re dealing with; what is your situation.

Here’s a question we got so Marsh, just for you. You mentioned at the beginning, Marsha, that you have CALR type 2. Can you describe that? So let’s make sense of that. There was a support group leader, Kim, who wrote in. Kim wanted to know how do CALR types 1 and 2 manifest and progress? Dr. Oh, what’s the difference between CALR1 and CALR2? What does it mean?

Dr. Stephen Oh: There are a variety of different types of mutations in the calreticulin gene, but the so-called type 1 mutations are the most common and the type 2 are the second most common; slightly different. Functionally they may have slightly different effects, although I think as a class, these calreticulin patients have more similarities than they do differences.

But there has been some literature to suggest that overall, calreticulin mutations are felt to be associated with a more favorable prognosis, at least compared to patients whoa re JAK2 mutant or perhaps triple negative. But specifically those that have the type 1 calreticulin mutation, the data is strongest that they have the most favorable prognosis. So based on that, you could say – the simplest thing is to say if you’re calreticulin mutant, you have a more favorable prognosis.

But perhaps the more nuanced is that that is particularly so for those that have the type 1 calreticulin mutation. In Marsha’s case it’s interesting, and to me a little bit of a paradox in that type 2 calreticulin mutations are, relatively speaking, more common in ET than they are in myelofibrosis.

So in that sense it’s perhaps not surprising that she has the type 2 calreticulin mutation. But overall, of course, ET has a more favorable prognosis than MF so it’s a little, in that way, a bit of a paradox. And the other way to look at this, and this has certainly happened for myself and I’m sure it happens with Dr. Moliterno is these patients with ET or myelofibrosis who were diagnosed many years prior, before calreticulin testing became available, you then tested for the mutation and confirm they were calreticulin once that test became available.

It’s one of those sort of: well, I just confirmed what I already knew which is that they had a more indirect course over these many years.

Andrew Schorr: It’s just amazing. Fortunately we can talk about this, and hopefully the word better prognosis sounds ] great.

So I, with primary myelofibrosis diagnosed with the JAK2V617F gene, Dr. Moliterno. But when you talk about prognosis, now you’re introducing medicines that we haven’t had that long; like for me. I’ve been on ruxolitinib now four and a half years and it’s been working. So how do we – when you talk about prognosis related to genes, though, that relates to what treatments you have, right?

Dr. Alison Moliterno: Right. We have a lot of work because these genes do have meaning; what version you have, and they’re going to have meanings in terms of prognosis but hopefully also we’ll start to gain information about really how you respond to therapy. So far, it seems that it doesn’t matter too much whether you have JAK2 or CALR mutation in response for  ruxolitinib.

But I think as we extend or molecular profiling, maybe we will learn that some lesions are less responsive or less favorably responsive to have some of these agents, and might be more responsive to agents that we have in the future. So I think we have to use this information both diagnostically, prognostically, and also just kind of monitoring our treatment expectations to some of these new agents.

Andrew Schorr: Right, it’s a moving target and I know you have a few things in trials. So just to tie the knot on testing, Dr. Oh; somebody wrote in and said: look, my doctor doesn’t require genetic tests. Where do I start and how do I ask for it? Because we’re hearing the two of you saying it’s pretty standard for you to get a clear picture of what the situation is. So is, how shall I say it, self advocacy related to testing today for an MPN patient important?

Dr. Stephen Oh: I think it definitely is. These diseases, while they’re not super rare, they’re not as common as hypertension or whatnot. When you go to a hematologist or oncologist who does not see very many of these types of patients, they might not be TransDigm on the testing that’s available. And so from that standpoint, I think certainly self advocacy is important. This has been emphasized already but getting to an MPN specialist is important.

If we’re talking about in the workup stage, it’s just sort of imagine different scenarios. There certainly could be a situation where the physician, whether they’re an MPN specialist or a more general hematologist or oncologist; they may not feel there’s a testing and that could certainly be the case.

[00:54:02]

But if there is a strong suspicion of a possible MPN, then I think there’s no question this kind of testing should be done. Even in the situation I’ve encountered with some patients where the insurance may require preauthorization, if you go through that it’s almost never rejected.

Andrew Schorr: Dr. Moliterno, here’s a question we got in from Jane. Jane writes: my understanding is that JAK2 is an acquired genetic mutation, and you both spoke about that; not inherited so we’re clear on that. Is it known what variables cause this mutation? I got the impression from what Steve said, no. But then she asks; can this gene expression be reversed?

Dr. Alison Moliterno: Right. First, why did this happen. I always joke with my patients; I tend to think MPN patients tend to be the most highly educated, intelligent, beautiful and have healthy lifestyles; why would they get this? MPN or not, disease is a lifestyle or other processes that we can sort of point to. I think in terms of if we just focus on JAK2V617F, that’s an acquired mutation in the bone marrow stem cell, but we’ve come to learn that this is a fairly common mistake, acquiring this mutation.

So if you look at my bone marrow stem cells, you can find evidence that this occurs frequently as a mistake; almost like a typing error that we all make on our keyboards, and that this happens at a fairly high rate. Most of the time as Dr. Oh mentioned, it is deleted or it’s in a cell that doesn’t have a long lived lifespan so it really doesn’t propagate in the bone marrow.

 Some fascinating studies looking at the cause of this; what are the factors? So, this Danish study looked at 43,000 individuals that participated in a healthy Danish lifestyle activity. They said they had stored DNA samples from blood. These were well people; they did not have myleoproliferative disease. But they found that they could detect the JAK2 mutation, V617F in a reasonable percentage of these well individuals, and that this was more prevalent the older they got. So that over 80, I think it was .2 percent of individuals actually tested positive for this. They were able to look back and show that yes, most of these people didn’t have an MPN, although some of them did have higher blood counts.

When they followed them, some of them did go on to develop MPN. But the point of that is wow, this is a common, natural occurrence and it most strongly associated with aging. Most MPN patients are in their mid 50s when they’re diagnosed; some very young and some are much older. So again, while gee, 50 isn’t that old, 50’s not that old. So again, there must be other factors that allow that to occur. And these large population studies, I think smoking has been an association; again maybe helps accelerate that mutation growing.

There’s host factors, and that maybe we have reasons that we will make that mistake more often; s genes that we have perhaps that we’ve inherited that influence how well we have integrity in our DNA. So that just gets a little to why did this occur.

Andrew Schorr: What about turning it back, though?

Dr. Alison Moliterno: Right, turning it back and I think this is another thing that is important. Again, when we think about surveillance, we do know there are some therapies that can specifically turn down or squash that clone and I think many of us have heard or even experienced patients who are on interferon, or pegasys, pegylated interferon. And we can see that if JAK2 disease can go into a hematologic remission and even molecular remission that the JAK2 clonal burden reduces and may even go to zero.

This has also been shown with CALR mutation positive individuals in small studies that perhaps we can suppress that. So we hope someday to – it’s still a little frustrating; not all patients have that benefit. It’s not clear which patients will go into these molecular remissions of turning it down.

But I think we will get there and we hope that JAK2 inhibitors would have that effect. I think the data still there are not pointing in that direction, but I think we will ultimately be able to actually target that clone and control how it expands and turn it back.

Andrew Schorr: So Stephen, take us into your labs and into the research that’s going on around the world. How fast is this changing now? Alison talked about 2005 with JAK2 discovery and you had CALR and some of these others. What’s the rate of change now?

Dr. Stephen Oh: I think there’s different perspectives you can look at this. For those of us that are doing laboratory research, we feel in the field that things have progressed quite rapidly.

To go from 2005 discovery of the JAK2 mutation to small molecule inhibitors going into patients I think within two years or less to this one drug being FDA approved a few days later; that pace of discovery and development has been quite rapid. The identification of the calreticulin mutation in addition to MPL, those are really landmark discoveries in this field.

For patients, I think the perspective might be that wow, I wish it could move faster and we kind of know as a class in general what JAK inhibitors do; can we move onto the next level, or next phase, or next class of treatments? And I think there is where my long term outlook is very optimistic, but in the short term we don’t really have that  kind of next candidate clearly identified as to what to do next after the JAK inhibitors.

Andrew Schorr: Dr. Moliterno, we always talk about clinical trials. This relates to the progress you all are trying to make. We’re your partners in that. As you do the testing and you say oh, now we see this gene, and we see this one and this one and we’re trying to figure out who’s the bad guy, or is this new one a factor that we’ve identified? So what would you say to us as we lived hopefully long term with these conditions about considering being in a clinical trial to help you make these discoveries?

Dr. Alison Moliterno: We have had great support from our patients, not only in participating but also helping design these trials or partnering with us. As Stephen said, these are rare diseases so we don’t have 50,000 individuals to test whether aspirin works or not like you can in a cardiology trial, and get that answer in a year and make a difference for individuals.

I would say that the trials now are going to be a lot more molecularly based. So instead of just disease, MF yes or now; these trials are now going to have the molecular profiling and response to therapy as part of their design. I think that will give us a lot more information overall than we’ve had in the past. So there’s many benefits to this molecular revolution.

One is understanding the cause of the disease, the other is monitoring it over time, and then response to trials which individuals respond to; which therapies. I think we’ve all seen the television ads about Keytruda and patients, as you mentioned early on with different solid tumors and particularly lung cancer, and how really the profile of that tumor needs to specific targeted therapy, and that’s what we want for MPN.

 Then now we’re even learning that it really doesn’t matter where a cancer may have started; if it’s in the kidney or in the lung, that again the molecular – the drivers of that indicate that you may respond regardless of what organ it started in; it’s really the lesion that you’ve got. And I think we will be bringing that to MPN patient trials.

Andrew Schorr: I’m involved in an initiative called Precision Medicine for Me, and it started in lung cancer but it is about this testing to see is there either existing therapies or investigation ones that line up. Or F dot now and as you know one of the major cancer medical societies called ASCO, American Society of Clinical Oncology.

The whole mission of the new president, a guy named Bruce Johnson from Dana Farber in Boston, a lung cancer specialist, is that these precision medicine approaches of each patient and their doctor knowing what are they dealing with at the molecular level to push that out throughout oncology; certainly in the U.S. if not worldwide. So I think we’re all in this together. Marsha, you’ve been listening patiently.

Now you’ve had this transition from ET many years, which Stephen was talking about we know often a very good prognosis. But then it changed, and a little scarier. I’ve already started that point, myelofibrosis. But when you hear this, how do you feel, just listening?

Marsha: I think when I was first diagnosed I was scared to death. But I think through education, I’ve learned to calm down and I just try to tell myself that this isn’t a sprint in my case, hopefully it’s going to be a marathon.

And until then, I’m just going to learn what I can do to help myself. I’m very curious about additional mutations; however my doctor said that really would not affect the treatment I would be currently undergoing. And the big thing, insurance, has come about. He’s very concerned about whether or not insurance would cover the cost and the value of it.

Andrew Schorr: Right. Stephen, is this – it sounds like maybe we’re making progress with insurance companies; that they want us to get a clearer picture with our doctor? What’s your take on that now?

Dr. Stephen Oh: The driver mutations, the JAK2, MPL< calreticulin testing, I think it should be and almost always is covered by insurance.

But when you get into the more extensive genetic testing, that’s where the insurance companies will often balk at covering it. Then it becomes somewhat problematic as to is this really worthwhile. Obviously that the costs for it and for any particular patient can vary in terms of what insurance will cover, what the copay is, and what they can reasonably afford. In general, while I do believe that there’s much utility to this kind of testing these days, if the cost is prohibitive I do not push it and I certainly do not think it’s mandatory.

Andrew Schorr: Dr. Moliterno, and I’ll mention to our audience we have a few more minutes; if you have a question and we haven’t covered it, please send in your question to MPN@patientpower.info, MPN@patientpower.info.

Dr. Moliterno, so we have the JAK inhibitor now, ruxolitinib. There are others that are being tested in various trials. As you look not just at the genes but at the treatments, are you fairly encouraged there that you will have a broader array to line up with people’s different situations, and also what the side effects of treatment, if somebody gets anemic or whatever their profile is?

Dr. Alison Moliterno: Yes, I am very optimistic. I think many of us, again if you look back at where we were five years ago in terms of the options that we have and where we assume we’re going to be in five years, hence I think there’s lots of room for application of these therapies, for combination therapies to use therapies together, even the ones that we have. So I’m very optimistic.

And I think I would say it is very anxiety-provoking to hear you need molecular profiling, and yet your doctor is saying we don’t really need that, or we’re concerned about the cost, which is certainly a concern. One thing I always say is that’s fine; the molecular profiling isn’t going to go away as an option, and the cost will come down in the next few years. We are now able to do things that we couldn’t even imagine we could do clinically in terms of looking at all aspects of genes, and this cost will come down.

So I would say to, for instance Marsha, yes we may not need it today but we can always get it in two years when things are different in terms of the availability of it and the interpretation of it. So again, I think it is reasonable to say your treatment is this, you’re responding well; happily we don’t need this at the moment.

I would agree with that, but I’m also certain that in three or four years, we won’t be having this discussion so much about the cost of this testing.

Andrew Schorr: Dr. Oh, here’s a question we got in. you’ve got to decipher this one for us. We have some really smart people out there, like PhDs. Is a germ line ASXL1 mutation a high risk or detrimental factor in the same way as a somatic mutation? So what’s germ line, what’s somatic, and is this germline ASLX1 mutation bad news, basically?

Dr. Stephen Oh: That’s a great question, and I think it also connects to a broader question. But to first answer you, talking about our acquired mutations which is the same thing as somatics; somatic means acquired.

If you’re talking about a germline mutation, in this case ASX01, it’s not the same thing as an acquired or somatic ASX01 mutation, which is what we’re generally referring to when we talk about that gene and its affect on prognosis. That also connects to the more broader point which is that the devil’s in the details.

Whether it’s ASX01 or another gene, the exact mutation may matter a lot in terms of what the significance you attribute to that. In many of the labs that do this type of testing, they will have a column for each mutation where they’ll make a call as to what they think it’s significance is, and they’ll say yes, pathogenic or it will say no pathogenic, or it will say uncertain. Part of that interpretation depends on whether it’s the germline or somatic AKA acquired mutation.

Oftentimes they can’t say for sure because the only way to determine that conclusively is to test tissue that’s not from the MPN. So, sometimes it’s uncertain as to even whether it is germline or somatic/acquired. So again, just to be clear on this particular question; if it’s thought to be a germline ASX01 mutation, it’s unlikely to be relevant or have a prognostic impact on that particular patient.

Andrew Schorr: So Dr. Moliterno, the other question you must get from people is do I have to worry about a family member? Because when they start talking about genes, you’re talking about what your hair color is, or hairline as I was joking with Stephen, or whatever characteristics herein are hereditary.

But then we’d say gee, is there a hereditary factor to an MPN?

Dr. Alison Moliterno: That is a very important question, and long before the JAK2 discovery, we realized that about 10 percent of individuals with an MPN will have a first or second degree relative with either an MPN or another cancer. So there does seem to be heritable factors at play here. Some of the interesting – and again, it’s not because you inherited – in your individual case you inherited the JAK2 mutation from the germline directly from Mom, but you inherited a risk of acquiring it.

We’re trying to understand that and again, what does it relate to; does it relate to germline variation in genes that allow this to occur, that allow you to get mutations in blood stem cells or in other tissues?

So yes, we are still working on that. It does seem to be a component of that. And that’s not unlike CLL, chronic lymphocytic leukemia also has sort of a familial –

Andrew Schorr: And I have that, too. I’ve got them both. I understand. So I think about that all the time. Marsha, you have children, don’t you?

Marsha: Yes, I have two children. But I think even more importantly, I have a cousin who has CML and another cousin who had stem cell transplant for AML. So I think that’s interesting.

Andrew Schorr: This is all in the hematology world, so Dr. Moliterno, do we have, Marcia or me, with two blood cancers? And I have three kids; what about this and is there a testing they should have, or how do we go forward?

Marsha: We don’t have – I would to recommend that they need a JAK2 test or specific MPN testing. If they have normal blood counts and they’re well, they don’t need to be evaluated for disease at the moment. The question is are there family genes that need to be elucidated, and if you have a lot of cancer in the family, so for instance families that have a lot of breast cancer, we often will send them for genetic counseling to understand what their particular risk is. And in breast cancer and some other cancers, we do understand some genes to test that can give us that information; familial genes. So far in the MPN, we don’t really have the knowledge of which genes are at play. 

Andrew Schorr: Dr. Oh, any comment on that because I’m sure you get the same questions. People say oh, my God, should I worry about family members?

Dr. Stephen Oh: Absolutely. It comes up pretty routinely and just to echo Dr. Moliterno, I generally do not recommend any special screening for family members, of those who have an MPN. I would also point out that even if there is, certainly it’s established that there is a slight increased risk for family members of patients with an MPN. Even if the risk was, let’s say, four fold higher for a first degree relative, the overall risk of developing MPN is so low that that overall risk for a family member is so unlikely that they’re going to develop an MPN.

Andrew Schorr:  Really? That gives some comfort; maybe you too, Marsha.

Marsha: Certainly.

Andrew Schorr: We’ve covered a lot of ground over the last hour, and I think on a very important topic now as mirroring the progress related to some treatments either approved or developing is, understanding our slice of an MPN, and the working with you on monitoring that and how does that relate to care. We have two noted experts with us who are helping propel this forward. So we’re your partners in helping that. Dr. Stephen Oh from Washington University and the Siteman Cancer Center, thanks for being with us once again and giving us of your time; I really appreciate it.

Dr. Stephen Oh: My pleasure.

Andrew Schorr: Okay, and Dr. Alison Moliterno, being back with us again from Johns Hopkins in Baltimore; thank you. Thanks for both of you; your very clear explanations, and Marsha Krone, we learned a lot, didn’t we Marsha?

Marsha: We sure did.

Andrew Schorr: Okay. Marsha’s my partner here; she’s done it twice and we’ll have you back sometime, Marsha.

Also, we should tell you Marsha is a very active preschool teacher so she has these little rug rats running around all the time, and thank God you have the energy to do that and I’m really glad you do, Marsha.

Marsha: Me, too.

Andrew Schorr: All the best to you. Well, this has been a wonderful program. We want to thank the Patient Empowerment Network for leading the way in this series, and Incyte Corporation for its support of this educational activity, and Patient Power our wonderful team Alan and Jamie who make it happen behind the scenes. We’ll be having more throughout the y ear. We welcome your questions; always send them to MPN@PatientPower.info.

And all of us moving forward living with an MPN, we’re going to live well and we have wonderful physician partners to help us do that as they understand the genetics and all those people working with them to develop therapies to help us live a long and strong life. Thanks for being with us in Carlsbad, California near San Diego.

I’m Andrew Schorr. Remember, knowledge can be the best medicine of all.

Living Well with MPNs – Tips and Strategies for Managing Symptoms and Side Effects of MPNs

Tips and Strategies for Managing Symptoms and Side Effects of MPNs

As part of our Living Well with MPNs webinar series a panel of MPN experts and patients discussed managing life with an MPN. The panel shared advice on managing fatigue, itching, night sweats, enlarged spleen and other symptoms. The experts explained why symptoms occur and stressed the importance of communication with your healthcare team. Tune in to learn more.

Living Well with MPNs – Strategies for Getting the Best Care

Managing Life with an MPN: Strategies for Getting the Best Care

In this “Living Well With MPNs” webinar, our expert panel featuring Dr. Laura Michaelis from the Medical College of Wisconsin, nurse Erin Blackwell from Levine Cancer Institute, as well as patient advocate Beth Probert, discussed managing life with a myeloproliferative neoplasm (MPN). The panel shared advice for finding a specialist, making informed decisions about treatment and monitoring progress of the disease through testing and check-ups. Tune-in now to hear tips for living well from the perspective of patients and healthcare providers.


Transcript:

Andrew:                                  

Hello and welcome to this program, “Managing Life with an MPN; Strategies for Getting the Best Care.” I’m Andrew Schorr in Carlsbad, California and living with an MPN, myelofibrosis. The Living Well series is a Patient Empowerment [Network] program produced by Patient Power, and we thank Incyte Corporation for their support for this series. We’ll be doing several programs during the year so be sure to be signed up with our alerts and you’ll always know as we continue.

We have some great people with us today around the country. I’m in Southern California, northern San Diego County. Now let’s go to Milwaukee, and I want to connect with our friend Dr. Laura Michaelis, who is at the Medical College of Wisconsin. Laura, thank you for being with us on Patient Power and our Patient Empowerment program today.

Dr. Michaelis:       

Absolutely, I’m pleased to be here. I wish our weather was as good as yours must be.

Andrew:     

Yeah, it is nice. And also I should mention Dr. Michaelis is dealing with a cold, so if she sounds a little stuffy, doctors get sick, too. But thank you for being with us. Now let’s skip down to Charlotte, North Carolina and the Levine Cancer Institute and that is Erin Blackwell. Erin works with Dr. Michael Grunwald who we know well there. Erin, thank you so much for being with us in Charlotte today.

Erin:   

Thank you for having me.

Andrew:                                  

Erin of course deals with Dr. Grunwald with leukemia patients and myeloproliferative condition patients all the time, and won an MPN Heroes Award this past year at the American Society of Hematology meeting for her dedication. Okay, let’s go back to California. Beth Probert is with us from Oxnard, California. Beth is a PV patient and she is just north of LA in Oxnard. Beth, thank you for joining us.

Beth:   

Thank you so much for inviting me to the panel.

Andrew:    

Beth, let’s talk about your story just for a minute. And that is you’ve been living with PV for what, about a year or so, now and you’re 55?

Beth:     

That’s right; just about a year.

Andrew:     

Okay, how did that diagnosis happen?

Beth:  

Well, it was a little crazy. It was during a very challenging time of my life.

I was dealing with my daughter’s mystery illness and sort of forgot about myself, and finally went to have a CBC with a new primary physician. Somehow, a gynecologist – I was visiting a day before I was to get the results from my physician – had those results, and he saw them and freaked out, and pretty much told me to get my affairs in order.

Andrew:        

That is scary for you. So who told you that you had this fairly rare condition, polycythemia vera; where did that come from?

Beth:                                         

Well, the gynecologist hinted at it and of course I fled his office and got my primary care physician on the phone. She called me down a little bit, told me to come in the next day and she suspected that it was that diagnoses but then arranged for me to meet with a specialist, which happened a few days later. So I had those few days of just pure panic.

Andrew:                                  

Now, you subsequently connected with Dr. Ilene Weitz at University of Southern California, the Norris Cancer Center in LA. That’s worked out for you.

Beth:                                         

It’s been fabulous. I went there; it was a fairly long drive and I was telling myself I’ll get a second opinion and a third opinion until I find the right doctor, and I was lucky to find the right doctor.

Andrew:

We’re going to come back to how you communicate with your doctor and a little bit about the treatment you receive and how it’s working. But the thing is, you’re doing well now with the right healthcare team. You’re at peace, I guess, with your diagnosis; you’re going on with your life.

Beth:                                         

That’s absolutely correct, Andrew.

Andrew:                                  

Okay, so Dr. Michaelis, that’s where we want to get everybody to is connect with the right team and doing well with modern medicine. So first of all, she mentioned about connecting with a specialist. You’re a specialist.

There are not that many doctors who see MPNs. Is seeing a specialist, at least having a consultation, knowing what you know about the field today, is that important? You’re muted, Laura. Hit the mute. There you go.

Dr. Michaelis:                      

Sorry about that. I would absolutely say yes, at some point early on seeing a specialist is important. One of the things is Beth’s story isn’t that unusual. The findings in a CBC of something that seems very off, whether or not it’s a very high platelet count, a very high hemoglobin or hematocrit can be pretty unusual for some doctors to see and know how to deal with. Sometimes they respond relatively drastically when that’s not necessary.

I think one of the best parts about a specialist is you’re seeing somebody who’s seen multiple patients with your presentation, with your symptoms, who knows when to be alarmed and when not to be alarmed. So Beth did exactly the right thing, which was make sure she then grounded herself with her primary care doctor, and I’ll say most of the patients that get referred to me are either referred to me by a primary care doctor, or sometimes by a specialist who might be a hematologist/oncologist.

But this is a rare condition even among people who see heme and oncology, so sometimes a hematologist/oncologist will want them to see me because I see this very routinely, and have seen patients at all edges of the spectrum.

Andrew:                                  

Of course then as we talk about MPN, so everybody’s straight on this and some people may be new to it; talking about ET, essential thrombocythemia, polycythemia vera, which is Beth’s situation; and myelofibrosis, which has been my diagnosis since 2011.

In this program, we’re not going to really get into treatments and genomics and all of that. We’re really going to talk about how do you live well and get the best care. And it’s not just your relationship with the doctor. So, Erin, let’s talk about that. You work hand in hand with Dr. Grunwald. You’re part of the leukemia doctors there and the doctors dealing with blood cancer. The nurse plays a key role, and that’s part of the dialogue, right? And you’re specialized.

Erin:

I think so. I think it’s a very different role than the provider, but we are healthcare providers also so we get to nurture the patients in a little bit different way. We’re not diagnosing but I think we’ve spoken about this before; patients usually have a very good relationship with their provider but they tend to open up to the nurse or the person that they get close to, which oftentimes will be in the office setting, the nurse.

So I think I’m able to glean information that the provider didn’t about symptoms that they’re having, that maybe they didn’t share with the provider; medication issues that didn’t come up.

So I think our relationship – and I’m the bridge, is sort of how I phrase it to patients. They can always go through me to get to the provider. If they’re not sure they need to contact the provider; I’m sort of the middle man.

Andrew:

Let’s start at the beginning. Beth, when you’re OB/GYN was freaking out if you will; did you see those blood test results and see how abnormal they were?

Beth:                                         

I absolutely did, and I was joking with you earlier that not only did I see some highs on there; I saw “critical” written next to several of those results. So that was extremely alarming.

Andrew:                                  

Dr. Michaelis, most of us with any of these conditions see Hs and Ls and critical that she saw; notations from the lab. What does that mean to you?

Because we start freaking out until we learn more.

Dr. Michaelis:                      

Of course it’s important to remember that when somebody comes up with what is normal, it’s done by having let’s say 1,000 people go to the hospital, have their blood drawn, and then a bell curve is written as this is what the normal hemoglobin is. What’s important is not so much if somebody is one or two points outside of the edges of that; it’s important; what it is relative to Beth. So for example, if Beth starts at a given level and then over time, her disease gets under control, she may still be slightly high or slightly low in some zones.

But relative to her, the disease is getting better. So we don’t really pay attention to the highs or lows very much; that’s against a huge, general population. What I’m really interested in is what do Beth’s labs look like compared to where they should be, or what it would mean if her disease was well controlled.

Andrew:                                  

So you deal with that, too, Erin, is people have a test and they’re alarmed. You often have to, as I say, bring people back down to earth, right?

Erin:                                           

Yes, and unfortunately when we print labs for patients, it actually will print out “high, low, critical” so patients do become frightened. We have a really neat tool on our computer system where we can graph counts over time. So I can show you from last year where you’ve started and where you’ve come to, so you can see the improvement, hopefully, and at times not improvement. That seems to calm nerves.

Andrew:                                  

That does it for me. So okay, Beth, you started seeing a specialist and then you had a certain rhythm of visits now that you’ve had over the last year. How often do you go see Dr. Weitz?

Beth:                                         

I see Dr. Weitz now about every six weeks. At the [00:20:00] onset it was every two weeks and four weeks. But because I am doing much better, it’s now six weeks.

Andrew:                                  

Right, and we should mention in your case, your one of the folks with PV who is on an interferon. The dose has been adjusted but it’s working well for you.

Beth:                                         

It’s working very well.

Andrew:                                  

Okay, so Dr. Michaelis, let’s talk about how you establish a treatment plan. So we come in, you confirm the diagnosis, we have some tests. So, how does that dialogue go about not specific treatments but how do you establish a plan?

Dr. Michaelis:                      

That’s something I’ve learned as I’ve matured as a doctor, and I feel it’s something I’m getting – I hope to get better at. I start everybody by talking about what is the cause or what do we think the causes of the disease are. I think it’s helpful for people to understand the way the disease develops, what are the underlying levers and changes in the bone marrow.

And then we talk about 1) have we truly confirmed the diagnosis? Do we have all the tests we need? Once we get through step one, which is confirming the diagnosis, we then go through something called risk stratification. Risk stratification means that we look at what does the disease look like, what are the characteristics of the disease. Are there high risk characteristics or low risk characteristics?

We then look at the patient’s own medical history; their age, their other health issues, their what we call comorbidities; the other things they may be struggling with. And the combination of those two help me determine what’s the likelihood of something bad happening because of the disease, or how the disease interacts with the patient. Once we have that risk stratification, then I like to talk about goals. What are this patient’s goals? Some people want to do everything they can to eradicate a disease.

Some people, their important values are their quality of life or being at home with their families. I try and listen to their values and what’s important to them, and then with that, we think about this is the risk the disease poses. These are the goals that you have; what is our panel of options out here? What kind of arrows do we have in our quiver, and how should we choose that treatment based on what we can accomplish, what you want to accomplish, and what we ought to accomplish.

That ‘s the kind of dialogue, and I find that that conversation, not the one where you’re first diagnosed, but that conversation of the first discussion of should we start treatment, that’s one of my longest conversations. So I prep patients; this might be a conversation where you want to bring family members. This might be an appointment we’re going to make as a double length because it’s going to be a little bit longer. Sometimes we’ll have patients bring their iPhone or something so they can have other family members conference in.

But that decision about starting treatment and which treatment to go for and why is an important one. And finally, we end it all by what should we see if it’s working. So how are we going to measure that this treatment is working for you? What are the types of things we’re going to see, and when? And when will we know when it’s not working?

So I like to lay it out with saying have we gotten the diagnosis, do we have appropriate risk stratification, have we identified your goals, and what kind of treatments are out there that could accomplish your goals.

Andrew:                                  

I was living in Charlotte for awhile so I went to Erin’s clinic and to see Erin and Dr. Grunwald. We would talk at each visit based on what Dr. Michaelis was just saying: how am I doing, how’s it going; bout a frequency visit. Sometimes I’ve had doctors say to me, when do you want to come back? Then I’d say doctor, when do you think?

Well, I think you should come back in three months. Sounds good to me, or should I come back sooner? Erin, you’ve been part of those conversations about frequency of visits. How do you as a team determine that with the patient?

Erin:                                           

I think it’s really; again as Dr. Michaelis was referring to, it’s a conversation had by the physician, the provider; what they’re comfortable with versus what the patient is comfortable with. At times, it’s that the patient would like to be seen more frequently than they really need to be for their comfort and their peace of mind. And the physicians usually are okay with that.

Then there are times when they want to go six months without follow up, and obviously with a lot of our patients, unless they’re just doing very, very well, that’s not feasible or responsible. So it’s always a conversation and I’m the one to make their follow up appointments and to give that to them when they’ll leave. So I’ll be the last line if they have an issue with it; they let me know.

Andrew:                                  

Beth, how often do you go? How often do you see Dr. Weitz?

Beth:                                         

Now I’m seeing Dr. Weitz every six weeks, and I really took to heart what Dr. Michaelis and Erin just spoke about because it’s the process I went through. What makes it very comfortable for me now to go every six weeks, I know whenever I have a question I can reach out to Dr. Weitz through the portal email. And it’s amazing; she gets right back to me. So it helps bridge that time going from two weeks, then to four weeks, and now it’s six weeks. I don’t feel alone. I don’t feel like it’s too long because I know I could reach out to her and her support team, as well.

Andrew:                                  

Okay, let’s talk about phone calls and emails. Dr. Michaelis, are patients able to call you? Do you communicate either directly via email sometimes or through the portal that maybe your institution has – many have it; I have it at UCSD here – about visits or connection, if you will, or questions with you or your team when you’re not physically there; when the patient is not physically there?

Dr. Michaelis:                      

Absolutely. One of the technologies that is possible now because of these electronic medical records allows what we call asynchronous communication. So you wake up in the morning and you’re much itchier than you’re used to, or you have a rash, you email me. You don’t have to reach me at that exact moment in time, but I will see it. My nurse sometimes screens it, and if it’s something she thinks is more urgent she’ll walk it over to my office or she’ll page me.

If it’s something that can wait, there’s a given period in the day when I take a look at the messages that have come in, that I’ve set aside and I’ll respond to them. Sometimes a patient wants to talk by phone, which is great. And that, I always ask them leave me a time that’s a good time to talk to you; best for me after 4, before 6. And that’s a time when I tend to be able to communicate with patients. I think one of the key elements is to remember, these are not for something urgent.

If you have chest pain, if you have severe shortness of breath, if your leg gets suddenly swollen; don’t expect these emails through the electronic medical record to be that timely because it’s often something that’s done as a routine part of the day but not urgently. Those should be done by a page, and everybody should have a way to page your doctor or their nurse through a triage system. Or, if it’s a real emergency, of course go to the ER or call 9-1-1.

One of the other things I do is I give my patients a stack of cards. Because if they’re being seen in an emergency room or somewhere, I want them to give my card to that doctor and ay: this is my hematologist, I have a rare blood condition; please page her. That’s because sometimes folks live far away from me, and so if they’re up in the upper peninsula of Michigan or skiing somewhere and they have an emergency; their bleeding risks, their clotting risks are unique.

I want people who aren’t familiar with this kind of blood condition to be able to call me any time and I can say this person has PV, they’re on a low dose of hydroxyurea; these are the things you need to watch out for. If the person needs to go to surgery emergently, this is what needs to happen afterwards. So I always have my patients carry a stack of cards to distribute. And I say don’t assume any physician knows about this condition; please have them contact me.

Andrew:   

That’s a very cool idea. I hadn’t thought about that and they hadn’t told me that at UC San Diego where I go. So, now we’re going into travel season, Erin. So people may go far afield. Esther and I are actually going on a business trip to Europe; hopefully everything will be just peachy fine. So talk to me about travel.

People have anxiety with illness when we travel, and if we have a wonderful relationship with you or Dr. Michaelis, or Beth has with Dr Weitz in Los Angeles, when we get away from that tether it can be a little anxiety-producing. What do you say to people about travel, particularly as we’re looking at the travel summer season?

Erin:                                           

It’s usually a conversation at the appointment. If our patients are going to go out of town for any reason, there’s a risk of interrupting treatment or they need labs while they’re away; that’s something we can help them set up. We have a patient currently that travels to Florida every other week and is on treatment. So we worked with an institute in Florida to sort of split care. So it’s a conversation that needs to be have. And I think fortunately, our patients have direct access to us almost 24/7.

We have an Access Center that takes calls 24/7, and during the day will reach a nurse; at night a physician covers. They always have my email address, my direct phone number at my desk. So, we try to make ourselves very reachable, so if something does come up –

But as Dr. Michaelis was saying, if anything urgent and life threatening comes up; call us later and go to the ER first, that sort of thing. We reiterate those messages.

Andrew:

I’m going to come back to you in a second, Beth. Well, Beth, let me ask you this while I’m thinking about it. From the patient point of view, like I don’t know what your travel plans are. You’re giving yourself interferon shots. Do you worry about travel at all because of PV? How do you plan your life? Because you have your doctor visits, you’re giving yourself shots. How do you plan your life, and do you worry about disrupting that plan at all?

Beth:

You know, on the onset I absolutely did. When everything is running your mind, and at the time I was traveling more. So my doctor actually talked to me about if I do need to take it with me, that the airlines, you know, what they require. And she urged me to check the airlines that I typically travel to get the instructions; does it need to be refrigerated, what are the security risks?

So, I sorted that out when I first went on the medication. I was doing a little traveling for business. And of course now that I’m on an every other week dose, it does make it quite easier. But I do urge PV patients to figure that out in advance, even if it’s just a slight – Maybe it might happen, maybe it might not but to be prepared in advance is something so important. So I did check that all out.

Andrew:

Laura, you were talking about eventualities that come up for patients and the communication among doctors. For instance, I have minor surgery that I’m trying to have happen. And so in my case as a myelofibrosis patient, I have lower platelets. And so I’ve got the surgeons talking to my hematologist/oncologist and they’re going to be prepared with platelets for the surgery so I can have the surgery; they feel it’s safe.

But they planned for it and they’re all talking, and that’s the kind of thing we need to ensure is happening, right?

Dr. Michaelis:

Oh my gosh, yes. I think communication between physicians is absolutely essential in this, especially when a procedure is elective. That means there’s time. Elective is a term we use when something can be done and planned in advance instead of emergently. We do know that when patients with PV, for example, go to surgery and their hemoglobin is very high, their risk of clots after the surgery or even problems within the surgery with the anesthesia can be higher.

So you want to make sure you’re going in – to the best of your ability, you’re going in with your blood counts well controlled and under optimal management. Same with platelet counts, which elevated platelet counts after surgery, can sometimes be a problem.

The other thing is that when your doctors communicate, it means that there’s a transfer of information, there’s a transfer of knowledge. And so that surgeon, when they go to see you and does his post-operation visit, it’s going to remind him: oh yeah, this patient has that rare condition; maybe I should call that doctor and just update them, for example.

So starting that communication early and that transfer o f knowledge is not only good for you, but makes sure that there’s a sort of routine – there’s a routine collaboration on a complicated patient.

Andrew:

So Erin, what do you tell your patients so that they get everybody working together? You try to do it within the hematology area, but somebody might have diabetes, somebody might have heart issues, somebody might have whatever surgery, procedures coming up. So how do you counsel people so that you know what this condition is, that we’re living with this hematologic condition is understood and proper communication happens?

Erin:

Similarly to Dr. Michaelis, Dr. Grunwald and Dr. Gerber both give out their cards to every patient that comes to the door. They hold onto it and they know if they see another provider and there are ever questions, that they can give them the card or give them their information. Something we don’t think about all the time when we’re treating these patients with either acute leukemia or myeloproliferative neoplasms in this case, they oftentimes have comorbidities or other things going on that we have to also address and make sure they’re staying – if they do have databases, we’re monitoring their A1C and their sugars are controlled.

And just because they have this diagnosis in myelofibrosis, that they’re not forgetting to take care of the other parts of their bodies. We need all parts to work, so I think it’s really important when I see patients – I look over their labs and their vital signs and make sure we’re addressing the things that sometimes we accidentally tuck away, like if a patient’s blood pressure has been continually high and we’re not addressing, or if their sugars have been high and we haven’t checked an A1C.

I think the best thing we can do is educate patients to know those sorts of things and to know what to look for so they can be advocates for themselves. But I think it’s also important that we all are mindful of the different parts of the patient because most of our patients are pretty complicated. There’s not just one thing going on.

Andrew:

Right. I have a question for you, Dr. Michaelis. So frequency visits, just to go back to that, if you want to see me more often does that mean things are not going well and I should start – my blood pressure should go up and – you know?

Dr. Michaelis:

Not always. Certainly if I get worried about a patient, my solution to that is to pay more attention. So sometimes if I’m worried that somebody’s disease is progressing, I will see them more frequently. But more often, when I see somebody more frequently it’s because we’ve had a turn in the road.

It might be a change from hydrea to interferon. We might have changed the dose of interferon a little bit. I might be worried about –. Maybe it’s the time of year, for example flu season. A lot of patients in the fall, we have a lot more hospitalizations with all leukemia patients and also some MPN patients. So sometimes it’s just that – I usually, like Beth’s experience and Erin’s experience, the frequency of visits is often a conversation. I’m not somebody who can go to long without seeing my patients. I want to keep them close so that I monitor them.

But if somebody is getting labs, they live in the upper peninsula of Michigan, they’re getting labs every six weeks and seeing me every 12; that’s fine and just keep up with that.

Andrew:

Right. I want to mention to our audience, so we have time for your questions so send them to MPN@PatientPower.info. We’ll get to the ones we can today, and we’ll be doing a whole series of these programs and so we’ll just keep making sure that we cover what’s important to you. I want to mention in my case as a myelofibrosis patient, that Dr. Jamieson down here in San Diego, she has me go to one of their clinics within the UCSD system and get monthly labs. And then if there’s something she’s concerned about, they let me know. But otherwise, I’m on a three- or four-month schedule, whatever it may be.

Okay, let’s move on to some other things. We talked about the treatment plan, Dr. Michaelis; you talked about that. One of the things that can come up in treatment planning now today is hopefully we have a situation that continues to involve in MPNs, is clinical trials.

So you’re a researcher as well as an in the clinic clinician. So where does that fit in in the discussion, in your opinion, related to what clinical trials are happening, what might be coming, what should we put on the table for our ongoing discussion?

Dr. Michaelis:

Great question. I think there’s this false idea that clinical trials are something that are used as a last resort, and it turns out that that’s really not the case, not in MPNs and not in other conditions as well. So, clinical trials are where there’s a hope to either improve or change the standard of care. When a patient is newly diagnosed, there is oftentimes opportunities to be in a trial that might be providing the standard of care with something else, or might be testing some new agent against the standard of care.

In myeloprolifative neoplasms are one of the most novel agents; there’s a medicine called ruxolitinib which is the brand name Jakafi, and that was only approved because people participated in clinical trials and found that it was effective at the goals that were set out in the treatment of high risk or intermediate to risk myelofibrosis.

So when I talk to patients about clinical trials, I frame it again in terms of the risk stratification, what are our goals, what can we reasonably hope to change, and when should we try and do that; and I put that in the panel of options. So option No. 1 might be standard of care, option No. 2 might be a wait/watching approach; let’s just observe for awhile.

Option No. 3 might be participating in a clinical trial. Clinical trials become more important, I think, when the standard of care options haven’t worked, So in a patient that may have progressed through several lines of therapy. so in a patient that may have progressed through several lines of therapy.

Then looking even farther afield for clinical trials, newer agents, agents that are brand-new out of the lab are often tested only in a handful of spots around the country. And for people with the desire and the wherewithal to look for those novel agents, again far afield; if that’s something logistically possible. And if it’s part of your value system to be involved in something very experimental where there’s no guarantee of success, then looking into clinical trials might be helpful.

Some people don’t want to be in an experimental situation, but some people find value in being part of the process to discover new drugs. And that’s something that’s an important thing to explore with your doctor.

Andrew:

I’m just going to make my pitch. I also have another blood-related condition, chronic lymphocytic leukemia. I was in a phase II trial for that.

I got the combination therapy ten years before it was approved by the FDA as a combination. So I think that helped with my longevity. I’m on ruxolitinib, the drug you mentioned. I was not in the trial but I got it early after it was approved, and I’m very grateful, very grateful. I met even who I think was patient No. 1, Mike down at MD Anderson, and I’m very grateful; I’ve given him a big hug, you know? And so I think it’s something to consider.

Your interferon is working for you, Beth, but there are other interferons in trials and other medicines in trials should you need it. What’s your take on trials? Because I know you’ve been going, looking at all our programs and learning about it. What’s your thought about it now?

Beth:

Let me preface this by saying when I was first diagnosed, I knew I had to remain open-minded. There’s no black or white; there’s a lot of grey.

So I gave myself the commitment that I would listen to everything that’s out there that’s pertinent to me. And somewhat what Dr. Michaelis had said a few moments ago is let me identify your value system. So taking all that information in, I would absolutely consider being part of a clinical trial if it was going to benefit me or have the opportunity. And I agree again with Dr. Michaelis that it probably would depend upon where am I at. It might be working well for me today, but maybe six months, a year, two years –

The other thing I realized right away is that in as much as we all do have very rare diseases we’re speaking about today, things change. The ongoing research, what might be happening today, six months from now might be very, very different.

So taking all of that into consideration, and I do have a very strong goal for patient advocacy and hoping that my journey through this medical challenge is meaningful and could provide hope and care for others in the future.

Having said all of that, I would definitely remain open and just take the situation as it comes along.

Andrew:

Right. What a great attitude. So Erin, that comes up in your discussion. You have Drs. Grunwald and Gerber who specialize in this. I would just make a comment, so your doctors there, Dr. Michaelis in Wisconsin, other doctors we’ve mentioned along the way; these are specialists in these conditions. If you think about companies that are sponsoring trials, they’re going to go to these doctors and say would you be interested in doing this trial. So if you want to particularly have access to trials in MPNs, you want to be connected with the specialists in our conditions, right, Erin?

Because you have research going on there, right?

Erin:

We do. We have several clinical trials open at the moment that are pertinent to this particular set of patients. We don’t have every trial that’s open, but they’re also familiar with what institutions do. So, if we have to make a referral outside because of patients interested in a trial that we don’t offer, and that’s something that we routinely do.

And I just want to point out; you mentioned travel and being prepared. Because of an experience that happened recently, make sure if you travel that you take your Jakafi with you if you happen to be on it, because suddenly stopping Jakafi is not a good thing, as we educate our patients.

Andrew:

And don’t put it in your checked luggage

Erin:

Right, right.

Andrew:

Keep it right with you. I keep it so close to my heart, Erin, I understand. Jakafi, I know, it’s not something you want to stop.

Erin:

Yes, especially suddenly. It should be tapered if it’s going to be stopped or else there are withdrawals.

Andrew:

No, I’m very careful about that. Okay. So Beth, you’re living with this diagnoses that you’ve had for a year or so. So, how have you gotten your head on straight about it to go on with your life?

Beth:

Well, I knew right away that if I looked at my diagnosis as somewhat of a gift, and I mean that by I was running around, I had so many other priorities, I had no clue that anything was really wrong with me. And I shudder to think what might have happened had I not gone for that CBC. You know, I really had a very serious medical condition at the time.

And so I took the attitude that I need to be gracious I was diagnosed, because looking at the alternative, it could have had terrible consequences. And I realized – you know, it took me a couple of months to get my feet back on the ground, so to speak, to just be calmer.

And I really had to start doing some things that people had always said. Oh, you have to have balance in your life. Or you should exercise more; you need to take time out for yourself, or you need to meditate. And I just realized that I need to really be more balanced, mind, body and soul if I was going to go through this medical journey.

So I started doing some different things that worked for me. I wasn’t so good sitting there meditating, but I learned to meditate and walk. I started embarking on yoga. I really embraced myself in a plant-based diet for various reasons. It was working well for my daughter for her autoimmune and I thought there was a lot of merit for helping me. And with that came learning how to cook again; with cooking came more peace and time to think.

I know that’s sort of a long-winded answer but I had to do things differently to be able to understand that I want to live life to the fullest. And if I’m running around always worried, freaking out about this new diagnosis and my medical challenge, I wasn’t going to make it. I really had to be balanced mind, body and soul.

Andrew:

Well said; I think that’s true. So Dr. Michaelis, you have people come in. Whether you make the diagnosis or confirm a diagnosis, they’ve come from somewhere else and they’re pretty alarmed. The family members are alarmed as well. Yet, all of us want to take back control. We feel out of control; this disease we have never heard of. How do you help people? How do you counsel them so they can go on with their lives?

Dr. Michaelis:

One of the things I do is tell people that I’m paid to worry; you’re not.

My job is the worrying job; your job is the living job and so let me do the worrying. You know, I’m used to worrying about these things; I know what to worry about and that’s my job. So my job isn’t cure; I try and heal but I’m not a curer. I try to heal the best I can. If something is curable, I’ll do that. But I am good at worrying. So, that’s what I tell people to leave in the office.

The second thing is that most people have gone through episodes of their lives before where they’ve felt out of control, and things get you through that. Either your family, or the people you love that are around you, sometimes exercise or meditation. Sometimes it’s venturing back into positive habits like good eating like Beth was talking about, or being outside in nature. Some people are grounded in faith. Something that makes you feel bigger than yourself; those same strategies are helpful when you face the grief of a diagnosis like this.

And it is a grief. You’re leaving behind a perception of your body that’s not true anymore because you’ve been diagnosed with some crazy disease that you’ve never heard of. So it is a process. I would say Beth, three months is a great time to be able to have gotten grounded again, and I think the strategies that you used in sort of learning from your daughter is incredibly positive.

But you’re right; this is a new chapter for people. Like, you thought you were traveling along in one country and then the road took a turn and you’re in a different country. So you just have to sort of say well, I want to drive safely but I also have to enjoy the landscape here. So, that just means you have to adjust.

Andrew:

I’m just going to make a comment as someone now living with myelofibrosis four and a half years. So, it was terrifying and my spleen is somewhat enlarged so it’s a reminder that it’s there.

I have my medicine right next to my toothbrush morning and night so I’m reminded then. But the rest of the time, I just go about my business. And I think, and Beth you can probably relate to this, and I’ve said this a number of time. In our lives we have people say can you go on a hike with us, or can you come over for dinner, or can you take ten minutes and let’s get coffee? And often the answer is no because you’re so busy.

I’ve routinely tried to make my answer yes. And Beth, I don’t know if you, too, maybe you could say stop and smell the roses but it’s take time out to enjoy.

Beth:

I could not agree with you more, Andrew. I realized that I was just going a mile a minute all the time, and putting things off and people off.

And you do, you really do reflect on who that inner circle is that you want around you, and making time for them and enjoying that time. So yes, I absolutely agree with what you just said.

Andrew:

Erin, I’m sure you’ve had patients who’ve seen Dr. Grunwald or Dr. Gerber maybe for the initial diagnosis. They’re really troubled. And then over time, how have you counseled people to help them really just go about their lives, enjoy their families, enjoy their work, travel, whatever is important to them?

Erin:

I think that’s the key is finding out what is important to them, and then you can really help them understand that although this is a diagnosis that affects how they live the rest of their lives, and in no way should we minimize that. But we also want them to be able to do what you do, where you take your medication and you go about your day the way that you would like to. I think a large part of the reason our patients don’t always do that is how they feel.

A lot of times they suffer from fatigue, either from anemia or secondarily or just from the disease itself.

It’s hard for them to feel up to doing those sorts of things so I think it’s really important to find to what kind of lifestyle they lead, how we could maybe improve upon that, and then involve any members of the interdisciplinary team that we need to. Sometimes patients need social support, sometimes they need – we have a social worker on campus that’s able to help patients especially with that first visit it they’re having trouble coping.

We have resources available that can help these patients. But I think the key to it is understanding what life looks like to them, what they enjoy about it, what’s important to them, and how we can get them back to doing those things.

Andrew:

Amen. So Dr. Michaelis, let’s talk about communication, not just the emails and the web portals and phone calls, but sort of transparency in communication, if you will. So, that’s on a number of levels. You mentioned fatigue, Erin, and fatigue, I’m fortunate I think I really haven’t been affected by it in any big way.

Beth, I’m not sure about you but there are things sometimes that we don’t confront, or don’t make positive adjustments to. How do you help people first of all communicate about what’s really going on? You mentioned a phone call about itching awhile ago. But whatever the symptom may be and where you all put your heads together to say how can we help you live well, deal with this, improve it if we can, and if we can, what adjustments can we make? Let’s talk about that open communication.

Beth:

Absolutely. One of the things is Erin is absolutely right; this cluster of diseases has a lot of symptoms. And it used to be that policies looked at the blood numbers and said ah, they’re not that bad; you’re fine.

It’s the work of Dr. Mesa who really brought this idea of symptom management and identifying and quantifying the symptoms that patients have, that has led us to be able to say the people with myelofibrosis and myeloproliferative neoplasms are more tired, they have more difficulty; we’ve heard about the itching and the bone pain. There’s also more difficulty sleeping, more chances to depressions, sometimes difficulty fulfilling work obligations. So, truly there are some symptoms.

Now, some of those get much better. They get better sometimes with medications, for example. They can get better if the anemia is well controlled. We do think that probably exercise is helpful, although there are still clinical trials going on with that. But your doctor is never going to know if you don’t tell them. And if the doctor doesn’t ask on a routine basis, and have you really used some metrics to quantify it?

So some of us use surveys. Every time a patient comes in, they might do a certain survey that says: in the last week, how often have you been in pain? For the last week, have you noticed depression? Or for the last month, have you found more difficulty sleeping?

Those kind of things, and we can compare just like you charted your CBC over time; you can also look at your symptoms over time and it’s something quantifiable. It also gives you a hard number to measure; is my treatment working? Look, you say you don’t feel differently but look what you were saying your pain was before, and look what it is over the last three visits. So that helps to really bring people back to the fact that while things aren’t perfect, maybe we’ve made some improvements.

But I absolutely think that not only keeping a diary of your symptoms at home, so if you just keep a little three-ring binder or something that says I notice that my migraines are happening at once a week versus twice a week as before; making sure you arrive at that doctor’s office with data in hand can be really helpful at grounding them back to the fact that this is a symptom-based disease, a lot. And if you don’t treat the symptoms, you’re missing a key part of helping that patient thrive.

Andrew:

Erin, you’re nodding your head. And I know when you walk into the Levine Cancer Institute, you give us a clipboard.

There’s stuff to answer, and they do that in San Diego. Maybe you have that, Beth, at USC in Los Angeles, as well. So that, really being honest, the patient, and maybe the family member saying – if they’re with you – hey, you really haven’t been sleeping well. You really have been avoiding taking a shower because you said you’re having itching. That’s important, right?

Erin:

Yes, sir. And it’s sort of funny. These are constitutional symptoms that the patients feel. We give them an assessment every time they come to see us; one on their initial consultation that’s slightly different, and then subsequent follow up they’ll fill one out that is the square of 100, based on their symptoms. The max score, if they had every symptom at its worst would be 100; and if they had no symptoms, it would be a zero.

So we are able to track the progress. But it always tickles me that a lot of times the caregiver will fill it out on the patient’s behalf. Sometimes we might get more accurate information that way because the patients don’t want to complain, per se.

So the caregiver knows they’ve complained to them, whereas they don’t want to complain to us so it is interesting to see.

Andrew:

Okay, let’s go on and take some questions. And if you have a question, send it to MPN@PatientPower.info. Laura, this one’s for you. This is from Sandra. Sandra writes in: 2012 through a number of blood tests, I was diagnosed with an unclassified MPN. I see a hematologist once per year for blood work. My chart currently says MPN disease stable, and I’ve never been on any medication. Is it possible to be unclassified or are my doctors just waiting for the disease to progress?

Dr. Michaelis:

There is definitely a condition called MPN-NOS. That tends to be something called – it often is an MDS/MPN overlap.

I will tell you the World Health Organization recently redid their numeric – the way they sub-classify these. So one thing you might want to ask the next time you see your physician is, is my disease still classified as MPN-NOS? Or does the new WHO diagnosis, the new WHO classification give me a more precise definition? I will tell you also that sometimes repeated bone marrow biopsies, or at least a second bone marrow biopsy might be necessary to see what’s happening at this time.

Diseases evolve over time, so if it’s been five years since your last bone marrow biopsy, and if somebody is considering treating you, then I would certainly get a repeat bone marrow biopsy to get an accurate picture of your diagnosis.

Andrew:

Okay, here’s another question probably for you as the provider, here. I believe the name is Maria; could be Maria but I think it’s Maria. Or, we’ll call her Mari.

Does a B2 vitamin supplement, 1 milligram tablet a day orally, do any harm for a patient with PV? And then she also asks about other supplements such as valerian root or melatonin.

Dr. Michaelis:

About the B vitamins, I don’t know that it would do any harm. We do tend to avoid too many vitamin supplements in the early phases of these diseases because these diseases are where the bone marrow is growing on its own. It’s kind of like it stopped listening to the body’s controlling signals and the stem cells, which are the interior parts of the bone marrow, are growing without control and that’s why people’s hemoglobin or platelets are high.

So you don’t want to feed that. I don’t think it’s dangerous to take the B2 vitamin, but I also don’t know what help it would have.

With regard to a supplement like valerian root, that’s not really – I don’t know anything about that. What we have here, for example, is a specialist – is somebody who deals with alternative – she’s a pharmacist. She knows a lot about alternative medicines. And what I ask people who are interested in taking a lot of supplements is to have an appointment with her and verify that nothing is going to get in the way of the therapies that I provide. Because I’m responsible for the toxicities of any therapy that I administer. And so what I really want to make sure is that no supplements are going to hurt in that way.

Melatonin is a relatively common supplement. It’s excellent for sleeplessness that happens to be about circadian rhythm so your light exposure. I think it’s especially helpful in the northern part of U.S. I’m here in Wisconsin, and I’ll tell you it’s hard to readjust your sleep when you’re going from a 14-hour night to a 4-hour night based on where you live. It can be helpful and I don’t think that there’s much danger that’s been reported to melatonin when used as directed.

Andrew:

Erin, I know you have a pharmacist – I’ve met him – there at the Levine Cancer Institute. So checking in with the pharmacist about prescription medicines and supplements; that’s not a bad idea, is it?

Erin:

We actually have four dedicated pharmacists to our clinic, but it’s not just us; it’s lymphoma, myeloma and leukemia clinic. But yes, we use them frequently. They’ll consult with patients and at routine visits, they’ll check in. they actually have the ability to make patients – they’re essentially really nice medication lists that tell them when to take their medications at the scheduled time. It’s a printout for them that helps them organize when to take their medications.

So the pharmacists are very involved, and if I ever have a question about a cold medication; will it interfere with the medication they’re on, or supplements, then I will start with the pharmacist and go from there.

Andrew:

Okay, let’s go on.

Here’s a question we got from Judy. Judy asks: I’m finding it difficult to coordinate care among my doctors. Do you have tips on accomplishing that? She say: not all of my doctors are interested in being part of these communications. So Dr. Michaelis, you passed out the cards but how do we make it happen when we, the patient, and maybe you helping us with a serious condition, know it’s really important?

Dr. Michaelis:

It’s hard to change other people’s behavior. I think you offer. I think another thing to do is when you go to your hematologist, ask if a copy of the note, the consultation note, can be also sent to you so you can keep at least copies of some of the written communication that’s happening.

You can’t fix bedside manner. You can find doctors who have good bedside manner, and don’t be afraid to shop around. Nobody gets offended; you’re not offending anybody.

If you find a doctor that doesn’t want to listen to the other people taking care of you, then that’s a concern. People should be collegial. You’re the only person in the room that matters so treat it like that.

Andrew:

Amen. Actually, we have an email from Charlyn that’s related to that. She has this question: What if your hem/onc doctor does not want to give you a referral, such as to a subspecialist like you? She’s saying we’re close enough to be seen by a specialist at Stanford, so in northern California, but need the referral to have it covered by our insurance. So the more general oncology maybe doesn’t want to let go, and you have to go based on insurance. Any thoughts about that?

Dr. Michaelis:

It’s not something that I encounter that much because I’m on the receiving end of that. I guess I would, like every human interaction, I would explore about why.

Why do you not think I should go up and see? If it’s about – I just want at least one consultation to make sure I’m keeping up with things – I’m not sure. It may be that the physician doesn’t think that your disease is serious enough to be seen by – that you need to be seen at an advanced or a tertiary care center. And then maybe there’s a limitation in the number of referrals you get and they want to wait until you actually need to be seen for something where things are going in the wrong direction.

But I would just treat your doctor like a human and ask them why not; let me know. And sometimes you even have to set up an appointment just for a conversation. Just say I’d like to set up an appointment to come in, have a 20 minute conversation where you just say this is what I want to explore with you; that kind of thing. When in doubt, just ask why.

Andrew:

Right. I think be a consumer, Beth, right? You’ve got to advocate for yourself.

Beth:

That’s absolutely true. And you know, it’s important to educate yourself and as Dr. Michaelis said earlier, we’ve lost control when we are affected by these types of diseases. Sometimes that might be within the conversation, too, with your oncology/hematologist to say I believe I would have more control if I also explored having a consultation or working with an additional specialist. And it’s reminding your physician that it is about you gaining control and empowering yourself to be educated. And part of that educating might be expanding your team of care.

Andrew:

Okay, I’m going to do one more question and then remember, we have a whole series of these programs.

This came in from Ina: Dr. Michaelis, my mom was recently diagnosed with ET this week; she had a bone marrow biopsy. Maybe you’ve had one, Beth; I’ve had a bunch of them. And please advise why the bone marrow biopsy is needed and what we should expect to take place when we meet with the hem/onc next week to review the results.

Dr. Michaelis:

That’s an excellent question. Essential thrombocythemia can often be – you can suspect it on the basis of the peripheral blood, but you cannot confirm it and clearly know whether or not the condition is essential thrombocythemia or myelofibrosis without examining the bone marrow. So the recommendations, and your doctors followed the standard of care in diagnosis, is to perform a bone marrow biopsy at diagnosis for everyone suspected of having a myeloproliferative neoplasm. It used to be you didn’t need it for PV, but now that’s a part of the recommendations as well.

So, I think they did the right thing by doing the bone marrow biopsy. When you get together with your doctor, you should expect first off for them to clarify exactly what the diagnosis is, and what data has led them to that. The second thing you should ask them is what risks does this pose to my mom; what are the things we need to worry about? Does this make it more likely for her to have bleeding or blood clotting? Is there anything we need to do about that? Is there a reason why she needs to start treatment? And that treatment should be chosen or based on that risk stratification.

So I would say that your conversation should be about what data led to the diagnosis, how sure we are of that diagnosis, and whether or not her risk status require that she be considered for treatment.

Andrew:

Wow, great answer. That really gave me a lot of information. You mentioned Dr. Ruben Mesa, who we all know along the way. He’s going to be with us on a program we’re going to do in August.

So for all of our viewers today, be sure to be signed up with Patient Power so you get our email alerts, and you’ll be kept informed. There are a lot of programs coming up. There’s even an event that’s going to happen I think June 24th in Chicago. So we’re all plugged in. Dr. Michaelis has been with us a number of times; Erin has been with us before.

So we have this sort of community in the MPNs now. I think virtually now, more than ever before; you are not alone. Beth, you saw Esther and I on Facebook Live, you were telling me. We’re all connected, now and Beth is on a program. Beth, I want to wish you well with your PV, and we’ll have you on another program sometime. Thank you for joining us from Oxnard, north of LA today.

Dr. Michaelis:

Good luck, Beth. Thank you.

Beth:

Thank you both. Thank you.

Andrew:

And Erin, thank you for joining us from Levine Cancer Institute. Please tell Dr. Grunwald hi; give him a hug from me.

Erin:

I will be happy to.

Andrew:

Thank you for being with us once again, the MPN Hero.

Erin:

I was in good company.

Andrew:

Oh, thanks. Laura Michaelis from the Medical College of Wisconsin up there where you have either a lot of daylight or not much daylight in Wisconsin, thanks for being with us. And I know you have a cold; feel better, okay?

Dr. Michaelis:

Thank you very much. Thank you.

Andrew:

Okay. Well, we’ve had a great program. Remember, keep informed about our series. The Living Well series is really designed to keep plugging away at the different issues that we face as MPN patients and family members. I want to thank the Patient Empowerment network; this is really their program, produced by Patient Power. We also want to thank the Insight Corporation for their ongoing support.

In Carlsbad California where it’s sunny today, I hope it’s nice weather where you are. I’m Andrew Schorr, and as I like to say: remember, knowledge can be the best medicine of all.

MPN Patient Cafe® March 2017 – Life with an MPN

MPN patient and moderator, Andrew Schorr, leads a panel of MPN patients in this session of Patient Cafe®. The panel of patient advocates includes David Denny a Myelofibrosis (MF) patient, Andi Malitz an Essential Thrombocythemia (ET) patient, and Alisa Rouse a Polycythemia Vera (PV) patient. The panel shares their inspiring stories, how MPN has impacted their life, and what they do to live well with their disease. Lastly, they remind us to:

  • Advocate to get the right treatment for you
  • Remember you are not alone
  • Use your disease for good

Check out the full video below to hear from four MPN patient advocates.

Patient Cafe® MPN – March 2017 from Patient Empowerment Network on Vimeo.

How to Weigh Up the Benefits and Risks of Treatment…and Why It’s Important That You Do

Do clinicians have accurate expectations of the benefits and harms of treatments and screening tests?

new study in JAMA Internal Medicine concludes not. In a systematic review of 48 studies (13 011 clinicians), the researchers found that clinicians rarely had accurate expectations of benefits or harms, more often underestimating harms and overestimated benefits. Among the findings, obstetricians and neurologists underestimated the risk of birth defects from anti-epileptic drugs and GPs overestimated the benefit of prostate cancer screening. Transplant surgeons were biased towards an inaccurately low estimate of graft failure and all types of doctors were unaware of the risk of radiation exposure from imaging.

What do these findings mean for patients? Inaccurate clinician expectations of the benefits and harms of interventions can profoundly influence decision making and the standard of care patients receive. Patient activist, blogger, and author of the upcoming book “Heart Sisters: A Survivor’s Take on Women and Heart Disease” (Johns Hopkins University Press, November 2017), Carolyn Thomas, believes this to be “a consistently systemic issue for patients, too: most believe medical interventions will help more/harm less than they actually do”. It’s a wake-up call for patients who have a critical role to play in understanding and weighing up benefits and risks for ourselves, in order to get better treatment. And it’s a further reminder of the importance of shared decision making to reach a healthcare choice together, as opposed to clinicians making decisions on behalf of patients.

However, understanding the risks associated with a treatment is not necessarily straight-forward. The challenge for busy clinicians is that there isn’t always the time to read and digest the latest research to inform their practice. Medical commentator, physician, and cancer survivor, Elaine Schattner, believes that because medical knowledge changes so rapidly it’s hard for clinicians to keep pace. “This may be especially true in oncology,” she points out, “as patients become expert in their own conditions and needs, they may prefer to look up information on their own, and share their findings with their physicians.”

A lengthy article published this month in ProPublica, examines what it calls “an epidemic of unnecessary and unhelpful treatment” requested by patients and delivered by doctors, even after current research contradicts its practice. “It is distressingly ordinary for patients to get treatments that research has shown are ineffective or even dangerous”, writes David Epstein. “Some procedures are implemented based on studies that did not prove whether they really worked in the first place. Others were initially supported by evidence but then were contradicted by better evidence, and yet these procedures have remained the standards of care for years, or decades.” Epstein points to a 2013 study which examined all 363 articles published in The New England Journal of Medicine over a decade — 2001 through 2010 — that tested a current clinical practice. Their results, published in the Mayo Clinic Proceedings, found 146 studies that proved or strongly suggested that a current standard practice either had no benefit at all or was inferior to the practice it replaced. Of course, this is not to say that myriad treatments don’t indeed improve and save lives, but it’s important to ask questions and do your own research before making a decision on which treatment is the best for you.

Start by asking your doctor to explain all the treatment options open to you, including what would happen if you do nothing. Recognise that all treatments are inevitably associated with some risk of possible harm. Ask your doctor to quantify that risk beyond a purely descriptive term, such as “low risk” (what your doctor considers a small and acceptable risk may be unacceptable to you). Next, do your own research. In order to make an informed decision, you will need to gather reliable information on which to base your choice. Fully exploring the risks and benefits of treatment involves doing your own evidence-based research (using evidence from medical studies that have looked at what happens to many thousands of people with your condition). In a previous article, I shared with you some helpful guidelines for assessing medical information. Most media reports about the benefits of treatments present risk results as relative risk reductions rather than absolute risk reductions, so you will need to understand the difference. Absolute risk of a disease is your risk of developing the disease over a time period. We all have absolute risks of developing various diseases such as heart disease, cancer, stroke, etc. Relative risk is used to compare the risk in two different groups of people. For example, research has shown that smokers have a higher risk of developing heart disease compared to non-smokers. Ask your doctor to differentiate between absolute and relative risk. Check out the NNT website which provides non-biased summaries of evidence-based medicine. “NNT” stands for a statistical concept called the “Number-Needed-to-Treat” – as in “How many patients need to be treated with a drug or procedure for one patient to get the hoped-for benefit?” The core value of the NNT is its straightforward communication of the science that can help us understand the likelihood that a patient will be helped, harmed, or unaffected by a treatment. It provides a measurement of the impact of a medicine or therapy by estimating the number of patients that need to be treated in order to have an impact on one person. Because we know that not everyone is helped by a medicine or intervention — some benefit, some are harmed, and some are unaffected, the NNT tells us how many of each.

You may also want to hear about what other people with your condition have chosen to do and what their experience has been. But remember that just because something has/hasn’t worked for someone else, it doesn’t mean it will/won’t work for you. Orthopedic surgeon, Dr Nicholas DiNubile, recommends patients ask their doctors, “If this were you, or one of your immediate family members, what would you do and/or recommend?” While this may be useful, you must ultimately decide what benefits and risks are important to you. Can you tolerate the side-effects? Are you happy with the way the treatment is administered? Would you find it stressful to live with the risk of any serious side effects, even if the risk is small? What matters is whether you think that the benefits outweigh the risk of any side effects. Everyone is different. The treatment recommended for you may not be the best treatment for your particular lifestyle. Being an advocate for your own health care involves asking lots of questions, doing your own research, and making your preferences known to your doctor. By doing this, you will be better informed and in a stronger position to get the treatment that is right for you.


Related Reading
Clinicians’ Expectations of Treatments, Screening, and Test Benefit and Harm
The three questions that every patient should ask their doctor
Strategies to help patients understand risks

ePatient Virtual Courses

The ePatient virtual classrooms are designed to empower patients in all their healthcare matters.

ePatient 101

ePatient101: How to be an Empowered Patient, is an online course for anyone interested in becoming an empowered patient, empowered caregiver, or patient advocate. Through this online course taught by Alex Barfuss, you will learn:

  • The meaning of the term “ePatient”
  • Why being an ePatient is so important in today’s healthcare system
  • How you can save time and money and get better overall value from your health care providers
  • How to advocate for yourself
  • Tools, tips and best practices to help manage your or your loved one’s chronic disease

Caregiver 101

Caregiver 101 is full of useful tools for caregivers and taught by Caregiving.com founder, Denise Brown. By taking this course, you will learn:

  • How the carer/caree relationship can be a health relationship
  • How to find balance
  • How to find more time for your self
  • How to ask for support
  • Tips, tools, and tactics to be a better carer/caree
  • Curated links and resources
  • Videos
  • Knowledge quizzes
  • Support from a community of caregivers at cargiving.com

Chronic Lymphocytic Leukemia (CLL) 101

We are excited to be partnering up with Intake.me to bring you CLL 101. We wanted anyone struggling with a recent CLL diagnosis to become empowered through knowledge and support. By taking this course, you will receive:

  • An overview of CLL
  • Facts about CLL
  • Curated links and resources
  • Videos
  • Knowledge quizzes
  • Printable checklists with questions to ask your doctor
  • Why you should immediately get a second, expert opinion
  • Tips on building your healthcare team, and how your local doctor can work with a CLL expert to provide the best treatment
  • Where to find the latest CLL research, clinical trials, and other treatment options
  • Ability to ask questions from other CLL 101 students

These courses are part of the Intake.me experience and are free to everyone. You can sign up be clicking one of the buttons below. Enjoy!

Mobile Doctor’s Appointments? Do They Work?

Dr. On Demand Dashboard

Dr. On Demand Dashboard

Hate the doctor’s office and don’t want to go to Urgent Care or the E.R.? There’s an app for that. Doctor On Demand is a mobile application that allows you to have a video appointment with a doctor from your own home. On their website they claim, “At Doctor On Demand we provide fast, easy and cost-effective access to some of the best doctors, psychologists, and other healthcare providers in the country. Our patients can have Video Visits with these providers on their smartphone or computers at any time of day.” Recently a friend of mine used this app for the first time because of a strange lump in her armpit, so I decided to sit in to see what it was all about and if it can actually replace an in-person visit.

Before

First thing you do is download the app, create an account, and fill out your health and insurance information. Then, you are given the option to choose a specific doctor or specific time. If you chose the specific time route, you are assigned a doctor. My friend chose a specific time and told me that it was a very easy, user-friendly process. To prepare for the appointment, you fill out all your symptoms and take pictures of your problem (if applicable) to have on hand. I asked my friend why they chose Doctor On Demand over a traditional doctor, Urgent Care, or E.R., and she said this way she won’t have to waste time stuck in a doctor’s office, could be seen quicker, and the appointment times worked better with her work schedule. However, she was hesitant because the doctor would not be able to feel or see the issue in person, which may affect the diagnosis.

During

Once your call begins, the doctor begins by reviewing your chart before coming on camera. Next, the doctor comes on camera and asks to explain the problem and the symptoms she was having. The appointment is set up like a FaceTime call. The doctor assigned to my friend was very friendly and attentive. This when those photos you took beforehand are useful because then she asked my friend to upload the photos for her to look at.

After

After the issue was thoroughly explained, the doctor was unable to diagnose what was going on without further testing. She did explain the several possibilities of what could be occurring and what tests may be needed. However, in the end the doctor did recommend that my friend go see a doctor in-person to get an accurate diagnosis.

Overall

In conclusion, my friend was reassured that it didn’t require immediate attention and that she shouldn’t worry. She also felt better and more relax about the few days it would be before she could be seen by a doctor. She and I would both recommend using Dr. On Demand, especially for the simpler alignments, such as colds, because the doctors are able to write prescriptions to your local pharmacy saving you the time wasted in a waiting room. My friend had this to say about her overall experience:

“This was a quick solution to put my mind at ease that something more serious was not going on before I was able to schedule an in-person doctor’s appointment”

15 Tips To Get the Most From Your Doctor’s Visit

beautyHave you ever had the experience of leaving the doctor’s office wishing you had remembered to ask a certain question? Or have you left it until the very end to tell your doctor about the real reason for your visit? These so-called “doorknob” questions – bringing up an important concern just as you are leaving the office – can mean your doctor won’t have time to adequately address your concerns. When the average time it takes for a doctor’s visit is fifteen minutes, it’s easy to feel rushed and forget what you wanted to say, or to leave an appointment unsure of the information you have heard. But with a little advance preparation you can learn how to make the most of those fifteen minutes. Follow these fifteen tips to become a more empowered and engaged partner in your own health – and the health of those you care for.

1. When you call to make your appointment, explain clearly why you need to see the doctor. Let the receptionist know how much time you will need to schedule for the visit. If you have any special needs, such as wheelchair access or interpretive needs, let the office know in advance.

2. Be sure to that where you make your appointment accepts your insurance. You can call or go online to your insurance website to see a directory of in-network providers.

3. If this is your first visit to a new physician, gather together any past medical records and family medical history to take along with you.  If you’re seeing other doctors and have information they’ve provided, bring this along too.

4. Write down a list of your symptoms before the visit. It’s a good idea to keep a diary so you can chart your symptoms over time. Include details of the type of symptoms you are experiencing, when these symptoms began, and what makes them better or worse.

Use this common medical mnemonic to guide you.

(O)-P-Q-R-S-T

  • Pain (“Where does it hurt?”)
  • Quality (“What does it feel like?”)
  • Radiation (“Does it move anywhere?”)
  • Scale (“How bad is it? How much does it affect you?”)
  • Timing (“When did it start? How long does it last? Does it come and go? Is it gradual or sudden in onset? What makes it better or worse?”)
  • Other (“Any other symptoms?”)

5. Set the agenda at the start of your visit. Did you know that a patient has an average of 23 seconds to state their concerns before a physician interrupts? According to an article published in The Journal of the American Medical Association, only 28% of doctors know their patient’s full spectrum of concerns before they begin to focus on one particular concern, and once the conversation is focused, the likelihood of returning to other concerns is only 8%. Doctors have a limited amount of time for office visits. In order to use their time wisely they usually set the agenda and control the visit as much as possible. To avoid this happening to you, prepare in advance the top two or three concerns you want to raise with your doctor. Are you looking for a diagnosis? Do you need a new treatment plan or a modification of an existing plan? Are you looking for help with feelings of fatigue or depression? Don’t forget to describe your emotional state and any personal circumstances which may influence your physical health. Write down your main concerns so you are ready to verbalize them clearly at the beginning of  your visit.

6. If you use a self-tracking device, like a Fitbit, download your data and summarize the findings beforehand.

7. Bring a list of all medications you are currently taking, including over-the-counter medications, vitamins, herbs, or supplements. If you have a smart phone or tablet, it’s useful to take pictures of your medication and supplement labels to show the doctor.

8. During your visit, tell your doctor you would like to take notes. If you would prefer to record your notes via your smartphone, ask your doctor if it is ok to do so.

9. Medical care is a conversation. So to have influence in that conversation you have to speak up. If you don’t want the treatment your doctor recommends (or you’re not sure), it’s reasonable to ask if there are other treatment options available. Never be embarrassed to tell your doctor if you don’t understand something she has said. Sometimes doctors use medical jargon without realizing they are not explaining things in terms we understand. Repeat what the doctor has told you to be sure you understand and ask for clarification if needed

10. If you find it difficult to speak up for yourself, or you are facing a potentially challenging diagnosis, bring a friend or family member along for support. This person can also take notes and help you remember what was discussed later.

11. Always be honest with your doctor. You may not like to admit how much you drink, or smoke, or if you have stopped taking your medication because of expense or side effects, but your doctor needs to know about these and other lifestyle matters to ensure you are receiving optimum care.

12. Ask you doctor to explain any test results to you, Request a copy of the results for your own files.

13. Before you leave, be sure you understand what needs to happen next. Do you need any further diagnostic tests? When will you get the results? If you have just received a diagnosis, what are your treatment options? If you have questions or concerns later how should you contact your doctor? You can also ask if your doctor recommends any specific reading materials or websites about your condition.

14. If you have been given a prescription for a new medication, do you understand how and when the medication should be taken? Are there any side-effects, for example drowsiness, you should watch for? How will you know if the medication is working? What happens if you miss a dose?

15. After your visit, review and file your notes along with any test results or other documentation and billing you received. Schedule any follow up tests or appointments right away.

Your relationship with your doctor is one of the most important you have. Advance preparation will help you use your own time and your doctor’s time more efficiently and effectively. When people take an active role in their care, research shows they are more satisfied and do better in how well treatments work. Preparing for your doctor’s visit is an important step toward becoming a partner in your own health care and a better advocate for your health and well-being.

Least Invasive First

Dr. Winn Sams

Dr. Winn Sams

Editor’s Note: This blog was written by Winn Sams, D.C. Dr. Sams practices in Columbus, NC a small town snuggled in the foothills of the western part of the state.  A native of Charlotte, NC with a B.A. in Economics from the University of North Carolina- Chapel Hill, Dr. Sams graduated from Sherman College of Chiropractic in 2002 summa cum laude and valedictorian of her class. From her own experience where personal health directives and choices were not heard nor respected, she decided to create a site where uniqueness and diversity could be anchored in healthcare. Being a healthcare provider, she knew how important it is for the “whole” person to be not only known, but included in a plan of care. Thus, Least Invasive First was born.


Recently, my youngest daughter broke her right arm and dislocated her elbow. The ER referred her out to an orthopedist nearby. We showed up at the appointment with a lot of questions and wanting to know what our options were. The doctor entered the room, did not make eye contact with me nor my daughter’s friend, who was sitting next to me. His handshake was a mere extension of his hand to us (friend and myself), kind of like a king might do to his subjects to kiss his ring. He said he would like to order a CT scan of my daughter’s elbow and do surgery. I asked were there any other options and he said “No” and that he would be back in a few minutes. He never came back, but his nurse showed up to schedule the surgery. I was furious and let her know my dissatisfaction, clearly acknowledging that it wasn’t her fault, but we would not be coming back.

Now, you have to understand I am a Doctor of Chiropractic. I see patients every day and I would never treat anyone the way we were treated. There was no informed consent , no shared decision making in developing a treatment and no respect for who my daughter was (or us for that matter) as a unique person seeking care. EVERYONE deserves all of the above! So, we left that office and made an appointment with another Orthopedist, who was absolutely fabulous. Our experience was night and day from the first one. We felt like we were a part of creating our plan of care, throughout the whole appointment and were at peace with the planned surgery, leaving there feeling like we were in good hands.

My concern is this. When we are in pain or an emergency situation, we usually are not thinking straight. We just want someone to help us get out of pain and/or tell us what is wrong. We may accept the first Doctor that we encounter, as he/she knows more than us. As far as what a Doctor is taught in school, the knowledge of how the body works and their expertise/experience, that is true. HOWEVER,  the patient still has to be included in the whole process, otherwise, you are giving your power over to someone to do as they deem fit TO you. That is a recipe for disaster.

Data and evidence based science measure outcomes that can be repeated. That is a big help when trying to choose a plan of action, but healing and how our bodies RESPOND to said procedures or medications is not an exact science. This is where our uniqueness comes in. Some people are allergic to medications or do not need to start out with the highest dose, as their bodies may actually react unfavorably to what may be the standard practice. Some people would like to try other options first, if possible. In the best interest of all, seeing how that choice works and then moving on to more invasive choices if necessary. It is imperative that your Doctor know as much about ALL of you to make the best plan of care. But, you don’t have to back down or be ashamed of your choices if they don’t match up with your provider’s. Remember, a Doctor is only a person ( yes, just a person like you and I) who has certain training and experience in particular fields. You cannot assume that your Doctor has your best care in mind, when they don’t have a clear picture of who you are on all fronts.

So, with all of this in mind, I developed a site called Least Invasive First, www.leastinvasivefirst.org, where you can keep all of your advance health directives and info in one place, with everything digitally accessible at any time. You can upload forms and/or pictures into your profile that provide information, that in especially stressful times, you have available at the click of a button. Medications can be listed with dosage, so you can edit them as they change. You can also give your username and password information to a family member, so they have access to your information if you are unresponsive or not able to make decisions for yourself. There are a lot of creative ways that this service can be used.

Fortunately, this concept works well for the Doctor and/or hospital side too. I have interviewed many of both and all have voiced a resounding affirmation that information the patient provides would be a tremendous help. I am glad to offer a way to potentially change healthcare and it starts with you!