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Why Myeloma Patients Should Speak Up: Advice from a Nurse Practitioner

 

Why Myeloma Patients Should Speak Up: Advice from a Nurse Practitioner from Patient Empowerment Network on Vimeo.

Speaking up, or self-advocating, may influence a myeloma patient’s health outcome. Charise Gleason provides a brief explanation of why patients should ask questions and seek advice from their healthcare team without hesitation.

Charise Gleason is a nurse practitioner specializing in myeloma and serves as the Advanced Practice Provider Chief at Winship Cancer Institute of Emory University. Learn more about Charise, here.

See More From The Pro-Active Myeloma Patient Toolkit

Related Resources:

How to Get the Best Multiple Myeloma Care No Matter Where I Live?

Myeloma Targeted Therapy: Why Identifying Chromosomal Abnormalities is Key

How Targeted Therapy Works to Treat Myeloma

Transcript:

Charise:

I think for patients it’s important to ask questions. I think sometimes they feel like, “I’m sorry I’m asking this,” or, “I’m sorry I’m telling you all about my side effects.” And that’s what’s supposed to happen at our visits.

So, I think patients and their family members or caregivers are their best advocate. And they should never feel bad about asking questions, reaching our, reminding their team of things, and being that advocate. We know about side effects. And we know about these treatments. And we can tell them, but we’re not experiencing them. So, there’s nothing that’s too small. And with everything we do, come some sort of side effect. So, it’s really a team approach to manage these things. And you never want patients to be suffering through. And reaching out to your team, even between visits, is really important.

Writing things down. Coming to a visit with a list of questions. These visits go quickly and if you come with those five things you really want to ask, or more… But have them written down so you don’t miss it in your visit so you feel like you’re part of that discussion and you’re getting the information that you need.

Key CLL Treatment Decision-Making Factors

Key CLL Treatment Decision-Making Factors from Patient Empowerment Network on Vimeo

What should a patient consider when deciding on a chronic lymphocytic leukemia (CLL) treatment approach? Dr. Susan O’Brien, a Hematology-Oncology specialist, provides key factors that help guide treatment choices for patients with CLL.

Dr. Susan O’Brien is the Associate Director for Clinical Science, Chao Family Comprehensive Cancer Center.

See More From The Pro-Active CLL Patient Toolkit


Related Resources

 

Should You Discuss a CLL Clinical Trial with Your Doctor?

What Are Common CLL Treatment Side Effects?

What Should You Know About the Future of CLL Treatment?

Transcript:

Katherine:

Dr. O’Brien, once it’s determined that it’s time to move forward with treatment, what do you take into consideration to help guide the treatment choice?

Dr. Susan O’Brien:     

Well, the good news and the bad news are kind of the same. The bad news is it’s a very complicated decision, but the good news is the reason it’s complicated is because we have a lot of good options. So, as I said, there are some people for whom chemotherapy would still be an option. One of the benefits of that is that it’s intravenous, i.e. there’s no copays for the patient. It’s administered over a finite period of time. Generally, six months.

And then, most patients will get several years of remission after that where they don’t have to be on any treatment. However, we now have what we call the small molecules or the targeted therapies and those come in two major categories. One is called BTK inhibitors. And there we have two drugs available in the same family, if you will. One is ibrutinib. One is acalabrutinib.

And then we have a different category of oral treatment where we only have one drug, which is a BCL-2 Inhibitor, which is Venetoclax. So, what these drugs do, they’re not chemotherapy, but they interfere with certain proteins in the CLL cell. And by doing that, cause the cell to die off.

Katherine:                  

Okay. What do you feel is the patient’s role in this decision?

Dr.Susan O’Brien:      

Well, I think the patient plays a key role, which they usually do when there’s options because then you have – you with your doctor have to make a choice. So, for example, we talked about chemotherapy is time limited and you generally will be done after six months in contrast, with the BTK inhibitors, those are given indefinitely. They’re pills but given indefinitely for several years.

With Venetoclax it’s given with an antibody, which is given intravenously but the Venetoclax can be stopped after 12 months. So, the side effect profiles are different also. So, we have to take into consideration the duration of the therapy as well as the side effect profiles in determining what might be best for that patient.

How Do You Know If Your CLL Treatment Is Working?

How Do You Know If Your CLL Treatment is Working? from Patient Empowerment Network on Vimeo.

How do you know if a chronic lymphocytic leukemia (CLL) treatment approach is effective? Dr. Susan O’Brien, a Hematology-Oncology specialist, explains how CLL treatment response is monitored.

Dr. Susan O’Brien is the Associate Director for Clinical Science, Chao Family Comprehensive Cancer Center.

See More From The Pro-Active CLL Patient Toolkit


Related Resources

 

Should You Discuss a CLL Clinical Trial with Your Doctor?

What Are Common CLL Treatment Side Effects?  How to Be A Partner in Your CLL Care

Transcript:

Katherine:

But how is that treatment monitored to evaluate its effectiveness?

Dr. Susan O’Brien:

Well, generally the things we’re – the same things we’re look – the same things we’re looking at when we treat. Right? So, we’re looking at abnormal blood counts. We’re looking at enlarged lymph nodes or spleen. We have symptoms. So, those three things are looked at when the patient is on the therapy. Are the lymph nodes shrinking? Are the blood counts improving? Are their symptoms getting better?

So, the same way pretty much that we would follow a patient who’s not on a clinical trial is the way we follow them on a clinical trial. Now, if it’s a very new drug which has never been given to humans before, let’s say, those trials probably have more frequent surveillance than we might do with a drug that we are familiar with and know what to expect with it. So, sometimes the trials might have more surveillance, more visits, more tests.

But generally, if those tests or visits are required – are not considered standard of care, the companies pay for them. So, usually what’s billed to the insurance is only what we would do treating any CLL patient with an already available drug.

So, it doesn’t wind up costing the insurance or the patient any more to be on a clinical trial. And they might get actually – there is some data the patients on clinical trials get better care because they’re being monitored very carefully as part of the trial.

Should You Discuss a CLL Clinical Trial with Your Doctor?

Should You Discuss a CLL Clinical Trial with Your Doctor? from Patient Empowerment Network on Vimeo.

Dr. Susan O’Brien, a Hematology-Oncology specialist, explains why patients with chronic lymphocytic leukemia (CLL) should consider a clinical trial and the role trials play in treatment and care.

Dr. Susan O’Brien is the Associate Director for Clinical Science, Chao Family Comprehensive Cancer Center.

See More From The Pro-Active CLL Patient Toolkit


Related Resources

 

What Should You Know About the Future of CLL Treatment?

What Are Common CLL Treatment Side Effects?  How Do You Know If Your CLL Treatment is Working?

Transcript:

Katherine:

Dr. O’Brien, where do clinical trials fit in in all of this? Should patients discuss clinical trials with their physicians?

Dr. Susan O’Brien:

Absolutely. If we think of these great drugs that we have now, and I’ve mentioned ibrutinib, acalabrutinib, Venetoclax. Before those drugs were available, the only options were chemo. So, that means that people that went on the clinical trial, so let’s say with ibrutinib, have access to a really treatment changing revolutionary drug in CLL years before it was commercially available.

So, clinical trials can be a great way to have access to drugs or combinations. So, for example, right now there are some clinical trials looking at combinations of a BTK inhibitor and a BCL-2 inhibitor. So, the patient might say, “Well, why can’t you give me that combination, doctor?” “Well, technically I could.” If the drug is approved by the FDA, a physician can prescribe it really pretty much anywhere they see fit.

However, does insurance pay for it? That’s the trick. And these are very, very expensive drugs. And so, outside of an FDA approved combination, it probably wouldn’t – I wouldn’t be able to prescribe that combination because it wouldn’t get paid for and it would cost thousands and thousands of dollars. But on a clinical trial in general, the drugs are paid for.

Katherine:                  

Mm-hmm.

Dr. Susan O’Brien:     

And so, clinical trials are testing, for example, combinations now, which are not standard and there are some preliminary data from some of these trials that look really promising, i.e. two drugs may be better than one. There are also patients who, perhaps we’re talking about younger patients now, who have kind of worked their way through the available therapies. And so, they might not have a standard therapy that’s really gonna work for them. And for whatever reason they might not be a good candidate for stem cell transplant.

And so, innovative or totally novel drugs that we don’t have that class of drugs available at all are also being tested in clinical trials and allow people access to them. So, sometimes it’s – I think some people think of it as, well, a last resort if the drugs that are out there don’t work. But don’t think of it that way, because as I mentioned, these combination trials are for people who’ve never had prior therapy, but their disease has progressed enough to need treatment and could potentially offer, at least at a preliminary level, looks like a dynamite combination of drugs.

So, it’s not just for people who failed other drugs or whose disease has failed other drugs. That could be one group that is particularly important for, but even patients who’ve never had treatment, there may be clinical trials that they would be highly interested in participating. And again, it generally has a big financial benefit too, because remember oral drugs have copays for cancer patients.

Treatment Approaches in AML: Key Testing for Personalized Care

Treatment Approaches in AML: Key Testing for Personalized Care from Patient Empowerment Network on Vimeo.

When it comes to Acute Myeloid Leukemia (AML), genetic testing (or biomarker testing) is essential in helping to determine the best treatment approach for YOU. In this program, AML expert, Dr. Naval Daver reviews key decision-making factors, current AML treatments and emerging research for patients with AML.

Dr. Naval Daver is an Associate Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. More about Dr. Daver here.

Download Program Resource Guide

See More From INSIST! AML

Related Resources:

 

How is Acute Myeloid Leukemia (AML) Treated?

 

Effective AML Combination Treatment: Pairing Old and New Therapies

 

Confused About AML Genetic Testing and Treatment? What You Need to Know

Transcript:

Katherine:                   

Welcome to INSIST! AML. A program focused on empowering patients to insist on better care. Today we’ll discuss the latest advances in AML, including the role of genetic testing and how this may affect treatment options. I’m Katherine Banwell, your host for today’s program. And joining me is Dr. Naval Daver. Welcome, Dr. Daver. Thank you so much for being here. Would you introduce yourself?

Dr. Daver:                    

Hello. Yeah. Thank you very much, Katherine. It’s a pleasure to join this discussion and meeting. I’m the Associate Professor in the Department of Leukemia at the MD Anderson Cancer Center. I focus on the treatment of acute myeloid leukemia and MDS, including the development of a number of clinical trials that are using targeted therapies and immune therapies for this disease. And with the great and dramatic progress, we’re seeing in acute myeloid leukemia; I think it is now more important than ever for patients to be aware of the options and be able to select the most appropriate therapy with their physicians.

Katherine:                   

Before we get into the discussion about AML, a reminder that this program is not a substitute for seeking medical advice. Please refer to your own healthcare team. Dr. Daver, I know the field of AML research is advancing rapidly. Would you give us an overview of the current treatment types in AML?

Dr. Daver:                    

There has been dramatic progress in the treatment of acute myeloid leukemia, especially in the last three years. We’ve had eight new drugs approved for the treatment of acute myeloid leukemia. The most progress I think that has happened so far is in the identification of particular molecular mutations and targeting those mutations with targeted therapies.

The mutations that are most important right now and have target options for FLT3 mutations, F-L-T-3, and the drugs that have been USDA-approved for this are an agent called Midostaurin, which is a first-generation FLT3 inhibitor and combination chemotherapy.

And then, more recently, another agent called Gilteritinib, as a single agent in relapse refractory FLT3 AML. The other mutational group that is also very important, and therapeutically needs to be checked, is IDHN1 and IDH2. And there are now two IDH inhibitors, IDH1 inhibitor, Ivosidenib, and IDH2 inhibitor, Enasidenib, both of which have been approved by the United States FDA for relapse patients with IDH1, IDH2 mutations. So, I think it’s really critical now to check for particular molecular mutations and to appropriately add the particular targeted therapy or select the particular targeted therapy in patients who have the mutation.

The other major area of advancement, and probably, if not the most important breakthrough that has happened, is the development of a new drug called Venetoclax. This is a BCL2 inhibitor. It’s new in AML, but in fact, it has been used for many years in CLL, which is chronic lymphocytic leukemia.

And this drug, in combination with Azacitidine in the frontline setting in older patients with AML who are not good candidates for intensive induction, has shown very high response rates, almost 70 percent CR-CRi, which is more than double of the 20 to 25 percent we were getting with Azacitidine alone.

And it’s now been approved by the US FDA and, in my opinion, and many of the experts really is the new standard of care and should be used in all older patients who are not good candidates for intensive chemotherapy given both the very high response rates, as well as now mature data showing significantly improved overall survival and a good tolerability.

So, there are many other breakthroughs. But I think these targeted agents, and Venetoclax, probably are the most impactful today.

And we’re focusing a number of new combinations building around this.  

Katherine:                   

What are common mutations in AML?

Dr. Daver:                    

Yeah. So, the most common mutation in AML is F-L-T-3, FLT3 mutation. This is both prognostically important mutation, presence of an FLT3 in a newly diagnosed AML, has been shown in many large publications by the German Cooperative Group, British Cooperative Group, our group, and others, is associated with an inferior survival.

Also, now, on top of that, it is also a therapeutically important mutation in addition to having negative prognostic value because the addition of FLT3 inhibitors seems to dilute, to a large extent, the negative prognostic value.

So, we believe that if we can identify FLT3 mutations at FLT3 inhibitors, we can definitely improve the outcome of those patients. The second most common is what we call NPM1 mutation, and that tends to occur with FLT3. About 55 percent of patients with an FLT3 mutation will have a coopering NPM1.

NPM1 is very interesting. With NPM1 mutation is present on it’s own without a FLT3, it’s actually associated with favorable outcome. It’s a favorable prognostic marker. However, if NPM1 is present with a FLT3, and especially if the FLT3 has a high quantity, high allelic load, then the NPM1 loses its favorable impact. So, now we’re kind of moving beyond just; do you have one mutation or not, which is what we thought 10 years ago, to; well, yes, you have this mutation, but what about the core-occurring mutation and even beyond. What about the burden, or what we call the variant allele frequency of that mutation?

So, for good or bad and I think it’s good in the end because it’s going to improve the patient outcomes, that we are getting more, more in-depth and there’s no longer quote, unquote, AML.

So, there’s a lot more granularity and analysis that is required even before starting treatment. And this is the thing that, in the community, we’re educating the doctors a lot, is that it’s okay to wait four to six days, especially if the patient does not have a very proliferative leukemia, to get the important bloodwork to identify the appropriate molecular and chromosome group.

So, that we can select the right treatment which will improve outcome rather than just rushing into standard treatment and missing a particular molecular chromosome group.

Katherine:                   

True. It might not be – the genetic testing might not be right for everyone.

Dr. Daver:                    

Right. Right.

Katherine:                   

What is genetic testing in AML?

Dr. Daver:                    

So, genetic testing in AML is basically what we call molecular profiling.

So, it’s looking at the presence of particular molecular mutations. For example, at MD Anderson, we do what we call 81 gene panel. So, this looks at 81 different genes for mutations in the bone marrow of newly diagnosed acute myeloid leukemia. Now, how did we come up with 81 genes? So, this was actually done by literature analysis and review of previously published preclinical and translational studies, and we basically selected all mutations that had been shown to occur in two percent or more of thousands of AML patients. And we found 81 such mutations. So, that any mutation that had a two percent or higher frequency in known published or public databases was included.

And that’s how we’re able to analyze for the mutation. So, it’s still possible that there may be some very rare mutations that are present, and those may be important for research. But they don’t change our treatment decision today. And so that’s what we call genetic profiling. Some people call it molecular mutation analysis. Some people call it next-generation sequencing.

But basically, this is looking for mutations in particular genes that are known to occur in AML. Now of those 81 genes; and some people do a 100 gene panel, some do 50, so those are variables; but among those, there are four or five that are most important: the FLT3, as we discussed, where we can use FLT3 inhibitors; IDH1 and two, because we can use IDH1 and IDH2 inhibitors; TP53 is a very important mutation because it has very high risk and adverse prognosis.

And there are now new drugs coming that may be very effective in TP53. So, we are checking for that. Those drugs are in trials, but the trials are showing very promising data and could be a great option if a patient is known to have a TP53.

Those drugs are Magrolimab, CD47 antibody, and APR-246. So, these are the four most important therapeutic mutations.

There are also some mutations that have prognostic value even though we cannot target them. These include mutations like RUNX1, DNMP3A, ASXL1.

One does not need to know the list. But the point is that these mutations may help determine whether a patient falls into intermediate-risk group or high-risk group, which then impacts the decision as to whether we need a stem cell transplant or not. So, it really is important to get this molecular profiling. It’s actually available in the United States commercially. And any clinic or hospital is able to actually order it. And insurance will cover it in 100 percent of the cases.

Katherine:                   

Wow, that’s great. What should – when should patients be tested, and how is testing done?

Dr. Daver:                   

Yeah. So, the basic testing for any suspected new acute leukemia is to get a bone marrow biopsy. That has to be done.

That should be done very quickly because all of the information that will be generated to make the treatment decision will come off the bone marrow biopsy.

Katherine:

What about retesting, Dr. Daver? Is that necessary?

Dr. Daver:                    

Yeah. So, retesting is necessary in – not for everything, I think.

But let’s say someone had treatment induction and relapsed a year later. So, we would definitely retest: 1) to confirm with the bone marrow’s relapsed AML, get the blast percentage because we need that before restarting treatment, so we know what was the starting point to know how the patients doing after treatment if he’s responding. 2) Molecular testing, for sure, should be repeated. We usually repeat the molecular testing such as FLT3, IDH1, IDH2, because there are drugs that can target these mutations in a relapse.

And more interestingly, we actually have published, and other groups have also published, that there are some patients who may not have those mutations at baseline but may actually acquire or have detectible mutations at relapse. So, if you don’t have FLT3 at baseline, your physician may assume that the FLT3 is not there, not do mutational testing. But in fact, that may not be true. So, it is important to retest about 15 percent, one five percent, in our publications can acquire a detectible FLT3. Which is critical because this could then change your treatment.

IDH1 and two are rarely lost or acquired, but we have seen a few five percent or so cases of that. So, it’s still better to check for that. And then TP53 we check for because now we have these new research clinical trials, phase one, two, that are showing some very encouraging activity in TP53. So, these are probably the main things to retest for.

There’s also some new clinical data emerging with a new drug called menin inhibitor that targets a particular chromosome abnormality, MLL rearrangement. This is again in a phase one setting, so the data may not be widely disseminated. But we’re seeing some very encouraging activity with menin inhibitors.  

And so, we are 100 percent checking for the MLL rearrangement chromosome, which can be done on FISH, or routine chromosome.

And if that is there then trying to get on one of the menin inhibitor trials, they’re opening about 25, 30 centers with different menin inhibitors, would be a very, very good option because we think these will be the next molecular or chromosome-targeted breakthrough in AML.

Katherine:                   

We’ve been discussing how molecular testing results lead to targeted therapy. How do targeted therapies work?

Dr. Daver:   

Targeted therapy means that we’re targeting a particular mutation. Now we may be targeting in different ways. So, some of the drugs, like FLT3 inhibitors, these are the most established and oldest targeted therapies in acute myeloid leukemia, been in development for about 18 to 20 years, work by blocking a particular receptor, the FLT3 receptor.

That receptor, when blocked, removes the growth and proliferation signal to the leukemia blast. And that receptor is much more preferentially and heavily expressed on the surface of the acute myeloid leukemia cell as compared to the normal, healthy myeloid or lymphoid cell. So, basically, we are shutting down the growth signals, resulting in eventual death of the leukemia blast and that’s how FLT3 inhibitors work. So, it’s a more of a direct activity resulting in cell death over a few days and quick action. On the other hand, we have what also is called targeted therapies but act very differently. These are IDH1, IDH2 inhibitors.

So, when you use an IDH1 or two inhibitor, they do go to the IDH1 and two receptor on the surface of the acute myeloid leukemia cell, but actually, they don’t result in the death of the cell. They actually cause what we call differentiation.

So, they promote that immature abnormal leukemia cell to undergo maturation and become a normal myeloid cell, which, over time, will die because normal cells have a finite lifespan, and they will die. As compared to leukemia blasts, which can live on much, much, much longer. And so, this process is called differentiation. So, FLT3 inhibitor, very different direct cell death. IDH inhibitor, very different from most maturation differentiation of immature cells to mature cells and takes longer. So, this is important clinically because with FLT3 inhibitors. We see responses quickly, one to two months. IDH inhibitors it takes longer, three to five months.

And so, targeted therapy is not one and all the same. You may be targeting a particular receptor, but the modality of action downstream may be very different.

Katherine:                   

What’s the treatment regimen for targeted therapies, and how long are patients treated with these types of therapies?

Dr. Daver:   

Yeah. I mean, that’s an area of big research. There’s no one field of answer yet for – and I don’t think there will be.

Of course, eventually. So, it really depends on; 1) What setting we’re using it in? Newly diagnosed, relapsed AML. In relapsed AML, with most targeted therapies, whether you’re use is a single agent, like FLT3, IDH1, IDH2, TP53, MLL-targeted agents, the goal is to get a patient to transplant.

Transplant, meaning allogeneic stem cell transplant using a sibling donor or a match-generated donor.

Because in relapsed AML without transplant, irrespective of the genetics and chromosomes, all relapsed AML have very poor outcome. The survival is only 20 percent or less without transplant.

If we can get a patient to transplant, we do have a good chance of long-term survival. So, the goal is transplant. And we usually use a targeted therapy for short, finite period, two to four months, to get a remission, get to transplant, hope that will cure the disease.

In front line, it’s quite different. We’re using induction chemotherapy with FLT3 inhibitors. In some research trials, we’re adding IDH1 and two inhibitors. We’re using Venetoclax, which is a kind of a targeted therapy.

Also, the BCL2 in combination with hypomethylating agents. And here, the targeted therapy is often used indefinitely. At least for one or two years. But in our approach and our guidelines, we continue the FLT3 inhibitor, IDH1 or two inhibitor or Venetoclax, as long as patient is tolerating it and does not have disease progression.

So, these are being used kind of similar to CML, chronic myeloid leukemia, where we use tyrosine kinase inhibitors or myelofibrosis, where you use jak inhibitors. They don’t cure the disease, but they continue to control the disease as long as you take them.

And in the end, we call this functional cure.

If somebody takes a FLT3 inhibitor and lives 20-plus years, semantically, he was never a cure, like an infection gets cured. But functionally, to me, he lived a normal life, and he was cured.

Dr. Daver:                    

And so, that’s how we’re using those inhibitors in the frontline setting different from the relapse setting.

Katherine:                   

How do these newer therapies differ from more traditional chemotherapy?

Dr. Daver:   

Yeah. Dramatically different. Completely different from traditional chemotherapy. So, to put it in more layman terms, traditional chemotherapy is like a nuclear bomb. Right? You – There’s a lot of things there in the marrow. You don’t know what’s good. You don’t know what’s bad. Blow it all up and hope that, when the new plants grow, the good ones grow and the bad ones were kill. And, in fact, this is true, to a large extent. Traditional chemotherapy, not to put it down, is actually been curative in a large population of AML for the last three decades. Our group and British MRC and Polish, and many groups have published up to 50 to 65 percent cure rates, especially in younger patients, below 65, with traditional chemotherapy.

So, this is not bad. People always get depressed with leukemia. But if you look at solid tumors, I mean, they have never achieved cure rates above 10 to 15 percent till the last decade or so. So, we were still getting 60, 65 percent cure rate. Two out of three.

So, traditional chemotherapy has done great work. But it was that approach. Just nuclear explosion. Take it all out, and hope good stuff comes.

Now the targeted therapy’s like a sniper. It’s actually looking for the particular leukemia cells and trying to take them out one by one with minimum collateral damage to your healthy bone marrow cells, which are important to produce red cells, platelets, white cells. So, guess what? There’s much less toxicity. You don’t see the hair loss with these agents. You don’t see the mouth sores and mucositis. GI complications are much less; infection risk is usually less.

Not to say they don’t have their own side effects. Unfortunately, even the targeted therapies have unique side effects. But, in general, those side effects are much less impactful in a negative quality-of-life way and much more manageable and tolerable. So – And, in the end of the day, they’re actually often more effective.

So, for example, with the FLT3 inhibitor, the study that was done with Gilterinib and Quizartinib, two very potent FLT3 inhibitors, was looking at a single-agent FLT3 inhibitor versus three-drug, high-intensity combination nuclear chemotherapy. And if I told this to any layperson, they would say, oh my God, that’s completely unfair comparison. You’re going to use three drugs, IV chemo, strong chemo, and compare it to one oral targeted pill. There’s no way the pill can be even equal, leave apart, win.

But guess what? The targeted therapy actually won. It not only was equal. It doubled the response rates, it reduced the toxicities and early mortality and led to improved overall survival, the gold standard. So, this shows that even though they are sniper, they can actually be much more effective with less toxicity. So, it’s a win-win. Better, tolerable, and more effective. Now the next stage within then decade, we think, it’s not one or the either, it’s really a combination. So, we’re reducing the dose of chemotherapy. So, we’re not making it as nuclear as it was. It’s still intense. But much more tolerable. And we’re compensating for that by adding the targeted therapy.

And, in fact, in the end, we expect much higher responses and survival with much better tolerability and lower early mortality. But I don’t think we’re at a stage where traditional chemotherapy is gone. Maybe 10, 12 years from now, as many more developments come, we’ll get there. But I think it still has a role, especially in the younger AML patients.

Katherine:                   

Dr. Daver, you mentioned the – some common side effects of chemotherapy. What about some of the newer therapies? Do they also have side effects?

Dr. Daver:                    

Yeah. Absolutely. I mean, every therapy we have in leukemia has a side effect. There’s no drug I can mention that is just devoid of them. Of course, some are less, and some are more. So, to be more specific, I think, for example, IDH1, IDH2 inhibitors, these are probably one of the most tolerable treatments we have in all of leukemia treatment. In general, they don’t cause much myelosuppression. Meaning, drop in blood counts. They don’t cause hair loss. They don’t cause mouth sores and GI upset in majority of people.

They’re always some patients who may. But what they can cause are two things: Number one, is they can cause what we call the differentiation syndrome.

And differentiation syndrome means the blasts that are going from the immature state to the mature state; in that process, they can cause an inflammatory reaction. And this can manifest with fever and cough, and chest pain, hypoxia. It’s something that’s actually very, very easily treatable, giving steroids for three or four days will take care of it. But many times, people were not aware of this. And so, often, we saw this was missed in the community.

So, that’s one specific example. With the FLT3 inhibitors, sometimes we see that they can cause more prolonged drop in blood counts, and count recovery can be delayed. Or we can sometimes see that they may cause some cardiac signals; increase in cardiac intervals. Again, something that, with close monitoring, bloodwork, keeping the electrolytes normal, can be managed. But I don’t want to go through the whole list. But the point is that there are specific and unique side effects that can be seen with particular targeted therapies.

And again, this is a learning curve where we have done these trials for eight to 10 years. So, we became familiar. But when the drug is approved, it’s a – it’s kind of a night-and-day situation in the community. They didn’t have the drug yesterday. They have it today. But there may not be any learning curve there. So, I think that’s where a lot of education and interaction with our colleagues is now coming into play.

But also, patients, I think, need to take this a little bit into their own hands, and also read about the label, read about the drug. So that, if they have side effects, if they actually ask their doctor and say, do you think this could be differentiation? I read about it. Yeah, most people will at least think about it. And I think this could be helpful to make sure that things are not missed. So, we do want patients to be more interactive and kind of  take things into their own hand. Because there are so many new drugs out there that their doctors may not be fully familiar yet.

Katherine:                   

Well, let’s talk about patient advocacy. What are some of the key tests that patients should ask for after they’ve been diagnosed?

Dr. Daver:                    

Yeah. Absolutely. So, I think the key things that patients should want to get the information is: 1) Knowing the bone marrow blasts.

I mean, that’s really basic. Just knowing what leukemia it is. What are the blast percentage? 2) Is, I think, chromosome analysis is very critical to get that information and to make sure we’re not missing acute promyelocytic leukemia, or core-binding factor leukemia, which have different treatments and very favorable outcomes, and would never, in general, never require a allogenic transplant. At least in majority of cases.

And 3), which is the one where we still see that it may sometimes not be available or be missed, is molecular testing.

I think it’s very critical to request molecular testing. And among molecular testing, especially FLT3, maybe IDH1 and IDH2, and TP53.

So, I think these are the most important data sets. Cytogenetics, key molecular mutations, bone marrow blasts, and confirmation of the type of leukemia before we embark on any treatment.

Katherine:                   

How can patients feel confident, do you think, in speaking up, and becoming a partner in their care?

Dr. Daver:   

Yeah. I mean, this is always a touchy area because physicians may feel that this is kind of encroaching on their territory or telling them what to do. And this is always a major challenge. I think when you go for the clinic visits, just to have a list of your questions written down and having them prepared and prioritizing them.

I always say, have your top-three questions ready.

We’ll try to do the others. But we’ll do the top three. And I think, when you have a new diagnosis of AML, the top three should be: what is the type of leukemia I have, and what are the bone marrow blasts? Number one. Do we have any chromosome and molecular information? Number two. And number three: Are there any specific treatments for my specific AML based on that chromosome molecular information? Or do we need additional information, and can we wait for that safely? I think these are the three very reasonable questions which, I think again, most leukemia experts will automatically be discussing this.

But, I think, for a patient, I think that’s important information to make sure they get before proceeding. If there’s time, the fourth question will be: Is – Are – Do we have a choice between high intensity, low intensity? And if we do, what are the pros and cons? In some cases, there may be a choice. In some cases, it may very clear that high intensity is the way to go, or low intensity is the way to go. But still, I think it’s often good to discuss that with your physician.

So, these are probably the four things one can bring up reasonably without the physician feeling that this is going to take forever, and I cannot discuss this. And then a lot of the AML treatment happens in-patient. So, there will be a lot of time for additional discussion. I tell my patients that, look, once we get the basics and the treatment decided, which is what we do in clinic, then you’ll be in the hospital most of the time. If it’s induction chemo for four weeks. Even if it’s Venetoclax, often they’re admitted for five to seven days, they will have more time then to discuss with the physician, the nurses, on a daily basis, and get more of the nitty-gritty.

Things like diet, exercise, lifestyle. Can I meet friends? I think you should not try to bring those things up right in the first visit. Because that may dilute the key information. So, I think staggering it, keeping in mind that many physicians are extremely busy, and getting that information in pieces over time, is probably productive for you and for the doctor.

Katherine:                   

With Covid-19 affecting all our lives right now, what should AML patients be considering at this time?

Dr. Daver:   

There’s a lot of guidelines on general approaches to managing things in COVID. And all of those guidelines heavily center, as we would think intuitively, on precautions.

Hand washing, minimizing contact, avoiding crowded places, trying to get treatment, potentially locally, if there are equivalent options available. We have not changed any of our frontline – we discuss this a lot every week in our faculty meeting.

This is discussed especially, as you know, because Houston currently is a major center affected heavily by COVID, and so, we have discussed whether we should move in a universal way to lower-intensity therapy for all patients. And we haven’t. And there’s pros and cons to that. When we do induction chemotherapy higher intensity, we, in fact, admit our patients for 28 days.

o, actually, even though it’s high intensity, the patient is more protected because they are in the room. Isolation rooms, sometimes. And they have minimum contact with outsiders. So, with COVID, actually, there’s very little opportunities or chances for them to get it. But the chemo is intensive. So, if they did get COVID, then it could be much more difficult or risky, or even fatal. On the other hand, low-intensity therapy is good because it’s low intensity and the risk of COVID, the frequency may or may not be changed; we don’t know. But the intensity we think could be lower because the immune system has not been suppressed.

However, low-intensity therapy very often is given outpatient. And so, then you have the benefit of lower intensity but the risk that you are going to be driving back and forth to the medical center, getting bloodwork, exposed to people in the waiting room, this and that. So, what we decided, after a lot of discussion among a big leukemia expert faculty in our group, was that we will still decide the optimum treatment based on the leukemia chromosome, molecular, age, fitness of the patient like we’ve always done.

And then we just have to try to encourage the patients to do as much precautions as possible. The other thing with the COVID, I think is very important is that, even though you may not be able to travel to your academic institution nearby because it’s harder to travel now, it’s still a good idea to try to get a consultation. We are doing a number of phone or email consultation, either directly with the patient, and even more frequently with their community doctor.

So, I get every day, four or five emails from academic even, and community physicians just saying, I have this patient, new AML, relapsed AML, whatever the case may be, here’s the mutation chromosome information, and I was going to do this. But the patient asked that I run this by one of my top academic colleagues. So, maybe MD Anderson. Some, I’m sure, are talking to Sloan. Some are, I know, are talking to Dana Farber. Cornell, whatever it may be. So, you can always request that. And maybe 100 percent of physicians may or may not do that.

And we’re seeing this collaboration actually. One of the positive things of COVID is we’re seeing these collaborations becoming better and better over time.

Katherine:                   

Oh, excellent. If a patient does need to go to clinic for a visit, what safety measures are in place?

Dr. Daver:   

Yeah. So, there’s a few things we’re doing in clinic is; one is we have staggered our clinics. So, instead of having everybody come at 9:00 or 10:00 a.m., and having 30 people in the waiting room, we really have more time slots.

And we ask people to come three of them at a time in the waiting room. We’re minimizing it three to five patients at most

Of course, there’s a lot of sanitization, dispensation units everywhere, encouraged to use those. The other important thing which, unfortunately, is a double-edged sword, is that we have had to minimize the number of friends, relatives, spouses, that can come with patients.

In fact, the policy at MD Anderson, like most cancer centers, is that nobody is allowed with the patient unless the patient is physically really impaired, as in wheelchair-bound or cannot go to the restroom. Of course, there are exceptions. But generally, I know, and I actually benefit a lot from it too, when patients have their family because the emotional support also helps our medical team to get information across. The patient may be sometimes stressed and forget things. So, what we’re doing more and more is doing phone calls.

So, what I would recommend is, as soon as doctor comes in, say, hey, doctor, can I call my daughter or my wife? I want her to listen to everything. Perfect. I don’t mind. There’s a speaker on. Good.

So, that helps with communication. But those are the big changes we have done from the clinic perspective. Still seems to be working relatively smoothly. We’re still seeing almost about the same number of patients in clinic that we were before COVID. And we have, fortunately, and knock on wood, not seen big numbers of leukemia patients with COVID. And we think the primary reason is because leukemia patients are just very cautious from the beginning. Even before COVID, they knew the risks, and we want them to continue that as much as possible.  

Katherine:

Dr. Daver, thank you so much for joining us today.

Dr. Daver:   

Thank you very much. Always a pleasure.

Katherine:

And thank you to all of our partners.

To learn more about AML and to access tools to help you become a more proactive patient, visit powerfulpatients.org. I’m Katherine Banwell – Thank you, Dr. Daver.

Notable News August 2020

Just when you think science can’t get any cooler, researchers figure out a way to learn more about cancer from dinosaurs. Yes, dinosaurs! Researchers have also found that fewer people are dying from lung cancer, and that the more they learn about immunotherapy the better. There is also a new study warning of a potential cancer risk for older adults.

The study pertains to adults 65 and older and indicates that taking aspirin every day could make cancer worse for older adults, reports medicalnewstoday.com. While past studies have shown that a daily dose of aspirin could protect older adults from cardiovascular issues, and clinical trials have shown that aspirin may reduce the risk of developing cancer in middle-aged adults, a recent study showed that daily aspirin intake may actually increase the risk of being diagnosed with advanced cancer in older adults. There was a 19 percent higher risk of metastatic cancer and a 22 percent higher risk of an advanced cancer diagnosis. People who are already taking a daily dose of aspirin should not stop doing so and should consult their healthcare providers with any concerns about the study before making any changes to their daily aspirin regimen. Learn more about the results of the study here.

Researchers may have discovered why immunotherapy isn’t effective for some patients, says sciencedaily.com. The treatment, which uses a person’s own immune systems to target cancer cells, doesn’t seem to work so well for young and female patients. A study shows that immunotherapy is less effective in young and female patients because they tend to have strong immune systems that are better at getting rid of tumor cells. That sounds like a good thing, but cancer cells that the immune system can’t easily recognize and get rid of often remain in the body, so immunotherapies are less effective at targeting those cells. Now that researchers know why immunotherapy doesn’t work for some patients, they can use the information to determine how to make treatments more effective for every patient. Find more information about the study here.

Researchers have also been looking for ways to increase the effectiveness of immunotherapies to treat blood cancers, says technologynetworks.com. By combining two types of immunotherapies, researchers found that they become more effective in treating blood cancers such as leukemia. There is also evidence that the new combination of therapies could be a safer option. The researchers are using similar technology used in the immunotherapy known as CAR-T therapy in which T cells are modified. In the new combined therapy, instead of modifying T cells, cells known as natural killer (NK) cells are modified. The CAR cells are also modified to help the NK cells find the tumor cells. The process utilizes the benefits of modifying both types of cells and may reduce the potentially life-threatening side effects of the CAR-T cell therapy which would greatly improve immunotherapy treatments. Read more about the combination of the two immunotherapies here.

Improved therapies are also having an impact on lung cancer mortality rates, reports cancer.gov. A new study by researchers at the National Cancer Institute shows that deaths from non-small cell lung cancer, the most common type of lung cancer, have dropped in the United States. The death rates are dropping faster than the cancer is occurring and the extra decline is attributed to therapies, approved in the last ten years, that target the cancer. Find out more about the NSCLC mortality rates here.

We are learning more and more about treating cancer all the time, and sometimes information comes from the most unlikely places, such as dinosaurs, says technologynetworks.com. Researchers have found the malignant bone cancer, osteosarcoma, in the lower leg bone of a Centrosaurus apertus, a plant eating, horned dinosaur that lived in large herds approximately 76 million years ago. The damage to the dinosaur’s bone was first thought to be a fracture, but upon further examination, the cancer was found. Using similar methods to diagnose cancer in humans, researchers discovered that the adult dinosaur had an advanced stage of the cancer and that it may have spread to other parts of the body. However, it is not believed that the cancer is what killed the dinosaur, as evidence showed it died as part of a herd in a flood. Researchers hope that they will be able to better understand diseases today if they are able to understand how they have evolved from the past. Learn more about how the dinosaur’s cancer was diagnosed here. You can even watch a short video about it. It’s super cool!

Is It Time to Treat Your CLL? What You Need to Know

Is It Time to Treat Your CLL? What You Need to Know from Patient Empowerment Network on Vimeo.

When it’s time to move forward with a chronic lymphocytic leukemia (CLL) treatment plan, what determines the best therapy for YOU? In this webinar, Dr. Susan O’Brien, reviews key decision-making factors, current CLL treatments and emerging research.

Dr. Susan O’Brien is the Associate Director for Clinical Science, Chao Family Comprehensive Cancer Center.

Download Program Resource Guide

See More From The Pro-Active CLL Patient Toolkit


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CLL & COVID 19: What Do Patients Need to Know?

 

How to Learn More About Your CLL

Transcript:

Katherine:                  

Hello and welcome to the webinar. I’m Katherine Banwell, your host for today’s program. Today we’ll discuss how you could work with your physician to find the best CLL treatment path for you. Joining me is Dr. Susan O’Brien. Welcome Dr. O’Brien. Would you please introduce yourself?

Dr. O’Brien:                

Sure. I’m Susan O’Brien. I’m the Associate Director for Clinical Sciences at the Chao Family Comprehensive Cancer Center in Orange, California.

Katherine:                  

Excellent. Thank you. And a note before we begin. This program is not a substitute for medical advice. Please refer to your healthcare team. Many CLL patients start in a period called watch and wait. Would you give us a brief overview of this approach?

Dr. O’Brien:                

Sure. The reason that we do watch and wait, or as some patients like to call it, watch and worry, is because many people present asymptomatically. So, for example, it’s very common that a patient might be found to have CLL because they go in for a routine physical and they have a slightly elevated lymphocyte count. So, many people have no symptoms at all. The average age of the disease is about 71.

So, people at the age of 71 often have what we call comorbidities. So, what does that mean? High blood pressure, high lipids, coronary artery disease. So, they also have a lot of comorbidities and even though right now we have great treatments for CLL that are generally well tolerated, all drugs do have side effects. So, if a person feels fine and the disease is not causing any problem in their life, why give them a treatment for it?

Particularly if we think that we don’t have a curative strategy. There may be a cure fraction for a small subset of patients with CLL who are young and have what we call a mutated immunoglobulin gene. But they’re a minority of most patients with CLL. So, what we want to do is keep people alive as long as we can with CLL until they likely die of other causes that people die of as they age. Heart disease, et cetera.

So, if they don’t need any treatment, we don’t want to expose them to the side effects. And some people, if you take all comers, everybody diagnosed with CLL, about a third of people will actually never need treatment for their disease. And so, that’s the idea behind it. That we’re sparing people side effects from treatments when they feel fine and their quality of life is perfectly good.

Katherine:                  

How do you decide when it’s time to treat?

Dr. O’Brien:                

So, it’s very variable because there are different indications from treatment in CLL. When I’m teaching my fellows, what I say to them is you basically treat the disease when it’s causing a problem. There are published guidelines, but they’re guidelines. They’re suggestions about when you might need to treat. But we take into account a number of different things. And in two different people the indications for treatment could be completely different. So, let me give you two examples.

 We could have a patient where they have big lymph nodes maybe in their neck, under their arms, in the groin, in the abdomen. And those nodes are getting bigger and bulkier to the point where they’re really problematic. That could be an indication for treatment. Other people might have very small lymph nodes but have very abnormal blood counts. So, their lymphocyte count could be really high. They could be starting to get anemic where their hemoglobin is dropping.

If you get too anemic, what’s going to happen? You’re gonna be symptomatic with fatigue and shortness of breath. So, we want to intervene not at a time when the disease is not causing any problems, but we also have to kind of find a happy medium. We don’t want to intervene – and wait until the patient is sort of bedridden and then start to do anything about the disease.

So, it’s a little bit of a judgement call. We also take into account the symptoms that the patient might be having. Like, are they having really terrible night sweats and fatigue that’s impacting their daily activities? So, we look at symptoms, we look at blood counts, and we look at lymph nodes or bulk of disease.

Katherine:                  

Where does genetic testing fit into the plan to treat?

Dr. O’Brien:                

There are certain tests that we definitely want to do before treatment. And some people have these tests done at diagnosis. So, the two main tests I would say are FISH, which just stands for fluorescence in situ hybridization, which is a fancy word for looking at chromosome abnormalities inside the CLL cell. The other thing we look at is the immunoglobulin mutation status. So, a patient’s immunoglobulin can be mutated or unmutated.

The immunoglobulin mutation status never changes. So, if a patient has had that test done once, they don’t have to have it repeated. However, the FISH, or the chromosome test, can change. So, it’s very important even if it was done at diagnosis that we repeat it at a time when a patient needs therapy. And why that’s so important is there is a particular chromosome abnormality called a 17p deletion where we know that those patients respond very poorly to chemotherapy.

And so, really should never receive chemotherapy and should receive a targeted therapy if that’s the case. There are other people that still could benefit potentially from chemotherapy, but not if they’re in that 17p deletion group.

Katherine:                  

All right. Dr. O’Brien, once it’s determined that it’s time to move forward with treatment, what do you take into consideration to help guide the treatment choice?

Dr. O’Brien:                

Well, the good news and the bad news are kind of the same. The bad news is it’s a very complicated decision, but the good news is the reason it’s complicated is because we have a lot of good options. So, as I said, there are some people for whom chemotherapy would still be an option. One of the benefits of that is that it’s intravenous, i.e. there’s no copays for the patient. It’s administered over a finite period of time. Generally, six months.

And then, most patients will get several years of remission after that where they don’t have to be on any treatment. However, we now have what we call the small molecules or the targeted therapies and those come in two major categories. One is called BTK inhibitors. And there we have two drugs available in the same family, if you will. One is ibrutinib. One is acalabrutinib

And then we have a different category of oral treatment where we only have one drug, which is a BCL-2 Inhibitor, which is Venetoclax. So, what these drugs do, they’re not chemotherapy, but they interfere with certain proteins in the CLL cell. And by doing that, cause the cell to die off.

Katherine:                  

Okay. What do you feel is the patient’s role in this decision?

Dr. O’Brien:                

Well, I think the patient plays a key role, which they usually do when there’s options because then you have – you with your doctor have to make a choice. So, for example, we talked about chemotherapy is time limited and you generally will be done after six months in contrast, with the BTK inhibitors, those are given indefinitely. They’re pills but given indefinitely for several years.

With Venetoclax it’s given with an antibody, which is given intravenously but the Venetoclax can be stopped after 12 months. So, the side effect profiles are different also. So, we have to take into consideration the duration of the therapy as well as the side effect profiles in determining what might be best for that patient.

Katherine:                  

Well, you talked about chemo and targeted therapies, but where – where’s stem cell treatment fit? Where does – where does stem cell treatment fit in and when is it considered?

Dr. O’Brien:                

So, stem cell treatment – if we’re talking about stem cell transplant, allergenic stem cell transplant is a transplant where you need a donor and you receive stem cells from the donor. And that can be a curative therapy, but it can also be associated with significant risks including risk of dying from the transplant. Because we have so many effective therapies nowadays, we’re generally not needing to use allogenic transplant.

And what I mean by that is if these targeted therapies don’t cure people, and the jury is still out on that I would say, if we can sequence them such that we get five years from one, six years from another, et cetera, we’re going to be able to keep the patient alive long enough until they die of something else. So, where the stem cell transplant comes in is generally much younger patients with CLL.

I mentioned the average age is 71, but we have – all of us int eh field have seen patients, for example, in their 30’s. Well, yes, a sequence of therapies might not get that patient to a normal lifespan, because they’re so young to start. So, really the consideration is pretty much reserved for younger patients where we might need a curative strategy that we might not have otherwise.

But for older patients, we probably have enough active drugs now. We have other categories of drugs that we can use if the disease reoccurs. So, we have enough categories of drugs that I think we can keep most people who are the average at CLL alive for quite a long time.

Katherine:                  

What about CAR-T therapy? Where do we stand on that with that research?

Dr. O’Brien:                

So, my answer is a little bit like allogenic stem cell transplant. CAR-T therapy is also associated with significant risks, but also significant benefit. Up until now, it’s pretty much been reserved because of the risks for patients who, to be frank, their disease has now kind of escaped everything. We don’t feel like we have great options that are similar and easier to use.

So, it can be effective, but it’s not something we do very early on because of the associated risks. If you take patients who go for CAR-T therapy, about 25 to 40% of them will wind up with some stay in the ICU. So, I’m really talking about some serious complications from these therapies.

It’s possible that as we learn how to minimize the toxicities of CAR-Ts, that they might become a more attractive strategy. And so, that could change with time. But the counterpoint to that is we’re having new drugs approved all the time for CLL. So, that gives us also more options before we would need to move to a CAR-T.

Katherine:

Dr. O’Brien, where do clinical trials fit in in all of this? Should patients discuss clinical trials with their physicians?

Dr. O’Brien:                

Absolutely. If we think of these great drugs that we have now, and I’ve mentioned ibrutinib, acalabrutinib, Venetoclax. Before those drugs were available, the only options were chemo. So, that means that people that went on the clinical trial, so let’s say with ibrutinib, have access to a really treatment changing revolutionary drug in CLL years before it was commercially available.

So, clinical trials can be a great way to have access to drugs or combinations. So, for example, right now there are some clinical trials looking at combinations of a BTK inhibitor and a BCL-2 inhibitor. So, the patient might say, “Well, why can’t you give me that combination, doctor?” “Well, technically I could.” If the drug is approved by the FDA, a physician can prescribe it really pretty much anywhere they see fit.

However, does insurance pay for it? That’s the trick. And these are very, very expensive drugs. And so, outside of an FDA approved combination, it probably wouldn’t – I wouldn’t be able to prescribe that combination because it wouldn’t get paid for and it would cost thousands and thousands of dollars. But on a clinical trial in general, the drugs are paid for.

Katherine:                  

Mm-hmm.

Dr. O’Brien:                

And so, clinical trials are testing, for example, combinations now, which are not standard and there are some preliminary data from some of these trials that look really promising, i.e. two drugs may be better than one. There are also patients who, perhaps we’re talking about younger patients now, who have kind of worked their way through the available therapies. And so, they might not have a standard therapy that’s really gonna work for them. And for whatever reason they might not be a good candidate for stem cell transplant.

And so, innovative or totally novel drugs that we don’t have that class of drugs available at all are also being tested in clinical trials and allow people access to them. So, sometimes it’s – I think some people think of it as, well, a last resort if the drugs that are out there don’t work. But don’t think of it that way, because as I mentioned, these combination trials are for people who’ve never had prior therapy, but their disease has progressed enough to need treatment and could potentially offer, at least at a preliminary level, looks like a dynamite combination of drugs.

So, it’s not just for people who failed other drugs or whose disease has failed other drugs. That could be one group that is particularly important for, but even patients who’ve never had treatment, there may be clinical trials that they would be highly interested in participating. And again, it generally has a big financial benefit too, because remember oral drugs have copays for cancer patients.

Katherine:                  

Right. But how is that treatment monitored to evaluate its effectiveness?

Dr. O’Brien:                

Well, generally the things we’re – the same things we’re look – the same things we’re looking at when we treat. Right? So, we’re looking at abnormal blood counts. We’re looking at enlarged lymph nodes or spleen. We have symptoms. So, those three things are looked at when the patient is on the therapy. Are the lymph nodes shrinking? Are the blood counts improving? Are their symptoms getting better?

So, the same way pretty much that we would follow a patient who’s not on a clinical trial is the way we follow them on a clinical trial. Now, if it’s a very new drug which has never been given to humans before, let’s say, those trials probably have more frequent surveillance than we might do with a drug that we are familiar with and know what to expect with it. So, sometimes the trials might have more surveillance, more visits, more tests.

But generally, if those tests or visits are required – are not considered standard of care, the companies pay for them. So, usually what’s billed to the insurance is only what we would do treating any CLL patient with an already available drug.

So, it doesn’t wind up costing the insurance or the patient any more to be on a clinical trial. And they might get actually – there is some data the patients on clinical trials get better care because they’re being monitored very carefully as part of the trial.

Katherine:                  

Let’s turn to patient self-advocacy. How can patients feel confident in speaking up and becoming a partner in their own care?

Dr. O’Brien:                

Yes, obviously for some people that’s going to be a lot harder than others. What I generally advice people is if you’re going in for your physician and you’re diagnosed with CLL, I would say this for any cancer because cancer is obviously a potentially life changing diagnosis, is you probably want to get an opinion with an expert. I would talk to my doctor first, ask them what their plan is so I know, and then see an expert in the field.

Then if the expert in the field says, “I think your doctor’s plan is great.” 1.) you’re now comfortable because you’ve got a second opinion, and 2.) that’s also a way, in my experience, to know if your doctor’s really gonna allow you to have an easy time participating. What I mean by that is that if your doctor is upset or finds it offensive, quite frankly you probably need a new doctor. That’s my take on that. Because that means they’re not going to be too open to your comments or you’re saying, “Well, I would prefer to do this.”

That’s just my quick take on how you can tell if it’s going to be easy or hard. But I think the relationship between the doctor and the patient is very important and you have to establish that relationship early on. If you go to a doctor who – where you start to ask questions and they’re in a hurry or they’re looking at their watch, you know that’s probably not the doctor that you want. I think most doctors realize that if they’re diagnosing a patient with a cancer, that’s going to be a pretty long clinic visit, because any patient is going to have a lot of questions to ask.

I also tell patients when you go to see a specialist or get a second opinion, bring somebody with you. It’s very well known that when patients have just been diagnosed with a cancer, they’re overwhelmed. Their emotional system is overwhelmed. Even if it’s “not a bad cancer”. Maybe early stage CLL. And that makes it very hard to process what a doctor is saying.

Particularly if they’re trying to give you quite a bit of information, which you need because you’ve just been diagnosed, and you need to know what to expect from the disease. So, having a friend or a spouse or a significant other there is really, really helpful.

Katherine:                  

Yeah. Yeah. That’s really good advice. Are there resources to help patients stay informed and educated?

Dr. O’Brien:             

Oh, yes. Our Leukemia and Lymphoma Society is great at that. Lymphoma Research Foundation were two of the big ones. And then there’s patient spots. CLL Society is a very well-known one run by a physician who’s also a CLL patient. I know him very well. And they have online support meetings now.

They used to have them in person, but now they have them online. And those can be really helpful because that allows a patient to talk to another patient who has their same disease. So, there are quite a lot of resources for patients nowadays. Especially in our technology enabled world.

Katherine:                  

That’s great. We have a couple of questions from patients. Patrick asks, “I’ve discussed a treatment plan with my doctor, but I’d like to get a second opinion. What are your thoughts on that?”

Dr. O’Brien:             

I think it’s a great idea. That’s exactly what I would do if I had a cancer. And again, I think Patrick made an important point that I’d like to emphasize. See what your doctor’s plan is first. Because then when you go to see the specialist or the second opinion, you can say, “This is what my doctor’s suggesting.”

And then if the specialist says, “Exactly what I would do.” But if you don’t know what your doctor is going to do – was suggesting to do when you go in to see the second opinion, it’s going to be really hard to make sure –put together that feeling of confidence that you’re on the right track.

Katherine:                  

Right. Right. To judge. A question from Julie. “How do you approach treating a relapse?”

Dr. O’Brien:

So, treating relapse we do the same thing that we do upfront. Namely “watch and wait”. So, for example, if a patient had a treatment on – let’s say they had some chemotherapy. And three or four years alter the lymphocyte count starts to go up, well, that technically would be indicating relapse.

But let’s say for the sake of discussion the person is asymptomatic, they feel fine, and their lymphocyte count is 20,000. Well, why do we need to do anything? So, in most cases we take the same approach of watch and wait when the disease comes back. And the first point at which it comes back is not always the time at which we need to initiate therapy.

Katherine:                  

Right. Right. Another question. Will is wondering, “If inhibitor treatments have to be taken forever?”

Dr. O’Brien:                

Well, it depend on the group – the class. So, for the BTK inhibitors, all the trials so far have given those drugs indefinitely. For the BCL-2 inhibitor, Venetoclax, there are time limited regimens both in the frontline setting and in relapse. But realistically, I have talked to my patients who are going on a BTK inhibitor who say to me, “Do I really have to be on this forever?”

And so, my answer is, “I don’t know what life is forever, so I would never use that word. We generally use the word indefinite.” But what I’ve said to those patients is, “If you’re on the drug for a while – and I’m not talking months. I’m talking say two, three years. And you’re in a really good remission and you think you want to stop treatment. I’m not necessarily opposed to that.”

Because if you’re in a very good remission, even if it’s not complete, but most people who are not in complete remission, meaning they still have a bit of disease left, have very little disease if they’ve been on the BTK inhibitors for a while. So, maybe only some enlarged lymph node on a CAT scan or a little bit of disease in the bone marrow.

But basically, most people after a couple years, they’re blood counts are normal, they feel fine, unless they’re having side effects from the drug and their physical exam is normal. So, I’ve told my patients if you want to go off, I expect you’d probably be off for even a couple years. And then we could always restart therapy potentially with that drug again or with one of the other drugs.

So, I think it’s important to let people know that they have options. But I will say that all of the clinical trials with the BTK inhibitors have given those drugs basically until the patient loses their response or there’s a toxicity where they just don’t want to take the drug anymore.

Katherine:                  

Mm-hmm. Mm-hmm. One last question from Jen. “What should be considered related to side effects when choosing a treatment plan?”

Dr. O’Brien:                

Well, the BTK inhibitors have some side effects. They can cause diarrhea, but that’s usually mild and self-limited. They can sometimes cause joint aches or arthrology. They – the two probably most serious side effects are atrial fibrillations, which is an irregular heart rate. But that generally is not frequent and tends to occur mainly in older men with heart disease.

Katherine:                  

Hmm.

Dr. O’Brien:                

They also are more likely – they impact the platelet function. So, they can more likely cause bleeding, but it’s typically minor bleeding like a bruise. Major bleeding is quite rare. In general, I’m outlining a lot of side effects, but remember not all side effects occur in everybody and there’s some people who don’t have any.

For the BCL-2 inhibitor, Venetoclax, one of the things we have to be very careful of when a person first goes on that and this would be particularly true if they have a very high lymphocyte count or a bulky lymph nodes, it that drug can cause something called tumor lysis. Tumor lysis, lysis is just a fancy word for breakdown, is where the disease responds so rapidly that their lymph nodes shrink very quickly. Lymphocyte comes down, which sound really good.

But what can happen is that breakdown of the cells can release potassium which can cause heart arrythmias. The cells can clog the kidneys and cause kidney failure. So, we have to be very careful about that when we start. And the way that drug is started is it comes with a starter pack actually to help make it easy where you go up, you start at a low dose, and go up weekly until we get to the target dose.

But we have to monitor very carefully during that escalation phase. The other thing that the Venetoclax can cause is neutropenia, meaning low neutrophil counts. What – that’s important because neutrophils are what we use to fight infection. So, if we get low neutrophil counts, the options are either to add a growth factor transiently, in other words a shot to – the subcutaneous injection that stimulates the bone marrow to release neutrophils. Or if it’s really a persistent problem, then we can go down on the dose of Venetoclax.

Katherine:                  

All right. How do you feel – how do you feel about the future of CLL treatment? Are you hopeful?

Dr. O’Brien:                

Absolutely. I think we’ve had something like six drugs approved in the last seven years, which is mindboggling. I think in the 30 years before that, we didn’t even have six drugs approved. That’s how rapidly – it’s mindboggling, really. That’s how rapidly the field is moving forward. And not just CLL, but other cancer fields also are moving at a very dizzying pace.

Which is great because that – anything that gives us more options is wonderful. So, I am very, very optimistic about CLL going forward. And I’m also very hopeful that some of these combination regimens might actually be – small molecules might actually be curative in the long run. But I will say it’s way too early to know that.

Katherine:                  

Are there emerging treatments that patients should know about?

Dr. O’Brien:                

So, one of the categories we haven’t talked about, where there actually are two FDA approved drugs, are PI3K inhibitors – that’s another oral small molecule. They’re not approved for frontline therapy. So, that’s kind of why we weren’t talking about them so much today where we’re talking about making a choice for the first therapy. But they are approved for patients where the disease reoccurs.

And there’s two of those as we mentioned. We have antibodies, which we really haven’t talked about very much, and then there’s new classes of drugs that are being explored in clinical trials. So, for example, there are interesting drugs which are antibodies that bind to the patient’s own T-cells and they also mind the CLL cells and they redirect the T-cells towards the CLL cells.

Kind of like CAR-T but inside the body without having to take out the T-cells. So, those are really interesting class of drugs. None have been yet approved in CLL or lymphoma, but I think those are on the horizon and looking very promising.

Katherine:

Hmm. One last question, Dr. O’Brien. In this uncertain time, do you have any advice related to COVID-19 for CLL patients?

Dr. O’Brien:                

It’s a hard time for everybody and particularly CLL patients because we know that they’re immunocompromised by – and even if you’ve never been treated and you probably never get any infections, which is quite a number of people with CLL, unfortunately you do have to think about yourself as being a high-risk patient.

So, masks are very important. Washing hands. Avoid – social distancing. Avoiding crows. It’s really important for patients with CLL to follow those same guidelines that we’re giving to everybody. But very important for them because they are in a higher risk group.

Katherine:

How do you feel telemedicine is working for CLL patients?

Dr. O’Brien:                

Telemedicine works I’d say better for CLL patients than some other patients, particularly watch and wait patients. Obviously the one thing that we can’t do in telemedicine is a physical exam. But in patient we can get – have patients get their blood counts done and then talk to them and see how symptomatic they are and know what their blood counts indicate anything is changing.

And then what I’ve been doing is, say I have a watch and wait patient – or it also applies let’s say to a patient who’s been on ibrutinib for years now and they’re in remission. There’s probably nothing to exam anyway. Right? So, those patients are good. I think it’s not going to work very well if you’re starting a new treatment. But for people who are watch and wait or have been on established treatments that are doing well, it works really well.

And then you can use the video visit if the patient says, “This is going on.” Whatever it is. “And I think I’m worried about this or I have this pain here.” Or whatever. If that’s an issue, you can always then schedule a regular visit. Right?

But I think that it – because it’s a chronic disease as opposed to acute leukemia where you really can’t do video visits, I think it lends itself to it very well. And my expectation is that moving forward, even after hopefully COVID has died down or we have a vaccine, that video visits are definitely here to stay.

Katherine:                  

Yeah. Yeah. I agree with you. What about patients who are fearful going into a medical center? Do you have any advice for them?

Dr. O’Brien:                

Usually – and it does vary. I also would be nervous if it was a hospital-based place where I had to go for my visit. But for example, where we are in the cancer center, it’s a completely separate building. Everybody is temperature checked before they get in. Everybody has to fill out a questionnaire about their symptoms. If they do have a low-grade temperature, we immediately triage them to another area.

So, actually I think the cancer center is probably a pretty safe place to be. Probably safer than the grocery store in that sense, because of the screening and the testing of the temperature of everybody who comes in there. And, of course, everybody has a mask on.

So, I would be probably a little bit weary in a hospital setting where they may be many sick patients hospitalized with COVID. But I think in a lot of clinic buildings or freestanding buildings, I probably would not be that worried.  

Katherine:                  

Well, Dr. Susan O’Brien, thank you so much for joining us today. And thank you to all of our partners. To learn more about CLL and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell. Thank you so much for joining us.

How Does COVID Impact CLL Patients?

How Does COVID Impact CLL Patients? from Patient Empowerment Network on Vimeo

How has chronic lymphocytic leukemia (CLL) care been impacted in the age of COVID-19? Dr. Phillip Thompson explains how COVID affects CLL patients and the importance of not delaying CLL treatment.
 
Dr. Phillip Thompson is an Assistant Professor in Medicine in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about this expert here.

See More From The Pro-Active CLL Patient Toolkit


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CLL & COVID 19: What Do Patients Need to Know?

 

How to Learn More About Your CLL


Transcript:

Dr. Philip Thompson:

There was a large ISH study published, I think, in Lancet Oncology, recently,  from the UK, where they looked at outcomes for patients with cancer. And of course, it was all patients with cancer, not specifically CLL, specifically blood cancers. But I think there were roughly 200 patients with hematologic malignancies.

And the interesting thing that I noticed, there were that patients who had recent chemotherapy, which I might have expected to be a really high-risk feature for a poor outcome, actually didn’t do any worse than patients who hadn’t recently been treated.

By far, the most important predictors of outcome for patients were whether their cancer was controlled or not, number one. And then other co-morbidities that patients had, like lung disease, advanced age, that sort of thing. So, actually, we need to see more data from more – from datasets that have more patients with CLL. But it seemed like the type of treatment mattered less than whether the disease was controlled and what other problems the patient had in terms of predicting their outcome from COVID.

So, I am taking that information with a – we have to, as I said, see more data. But I’m not going to use COVID as a reason not to patients who need treatment.

We may stretch things out somewhat in people where the decision is really well, and maybe you don’t definitely need to treat. But I don’t want to see people get into really severe trouble from their CLL because we’re trying to delay treatment because of COVID. Because that might actually be counterproductive. Because people with very uncontrolled CLL, if they were to get the infection, may actually have inferior outcomes to people whose disease is controlled.

Partnering With Your Doctor on CLL Treatment Decisions

Partnering With Your Doctor on CLL Treatment Decisions from Patient Empowerment Network on Vimeo.

Which CLL treatment could be right for you? Dr. Steven Coutre, a CLL specialist, reviews current approaches and explains why patients should stay informed about emerging options.

Dr. Steven Coutre is a Professor of Medicine in the Hematology Department at Stanford University Medical Center. Learn more about this expert.

See More From The Pro-Active CLL Patient Toolkit


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Transcript:

Dr. Steven Coutre:

Well, any decision about treatment is, of course, a joint decision between the physician and the patient. It’s our job to really educate each individual patient about their options and also, I think, very importantly, determine what their goals are. You don’t really follow a strict algorithm. It’s really making a decision for each individual patient.

So, of course that takes into account other medical conditions they may have, the nature of their disease, why it is that we’re treating that individual, what we’re trying to accomplish, and very importantly, what the goals of therapy are for that individual. That may be very different, for example, for somebody who’s quite young versus somebody who’s older or who might have significant medical comorbidities.

I think patients are always well served by asking questions about the treatment, side effects of treatment, of course, these days, cost comes into play, so I think we have an obligation to let patients know the differences between the therapies because often we have choices about a therapy. There isn’t any one best therapy, for example. It’s often a number of choices, and sometimes that can be very, in some ways, confusing for patients, because they wanna know, “Well, what’s the best therapy?” and as I mentioned, it’s not so much what’s the best. It’s what’s the best for that patient, and many times that’s choices of treatment.

Some are time limited, for example. Some are continuous therapies. So, there’s plusses and minuses, and again, it all goes back to what’s your goal for that individual patient, what are their preferences in terms of the treatment that they want to receive.

The drugs that I mentioned earlier are Bruton Acalabrutinib, Venetoclax, for example. These are really the first of our new really transformative drugs for CLL. Drugs, along sometimes, with our antibodies, Rituximab and Obinutuzumab, which are really replacing the use of chemotherapy in treating the disease. So, moving forward, we’re looking at combinations of these drugs. Can we drive responses deeper? That would lend itself to stopping therapy, in some case, instead of using continuous daily therapy as we currently do with drugs like Ibrutinib or Acalabrutinib.

So, that’s the major focus right now. There, of course, will be other new drugs. There’s a third drug, Zanubrutinib, which is another BTK inhibitor, so that’ll probably play a role in treating CLL. There may be differences in side effect profiles between these drugs. There isn’t any new drug that we’re looking at currently that’s far enough along to say that it’s gonna be yet another fundamentally different, revolutionary therapy for CLL. But those, of course, can come along as we learn more about the biology of the disease.

You may have heard about CAR T-Cell Therapy, where you’re using your body’s own immune system to try to target the cancer. This has been very successful and is actually approved for use in other diseases, like large cell lymphoma, for example, but it remains very much investigational in CLL. There are also other clever ways of trying to achieve the same, endpoint, that is, using your own immune system to target the cells, that are simpler than CAR T-Cell Therapy and those kinds of approaches are also in clinical trials.

So, when you’re having the discussion about treatment, it’s always good to learn about what the latest therapies may be, even if they are investigational. I think that’s how we move the field forward and, of course, the newer drugs that we have brought forward came from clinical trials that patients greatly benefitted from. So, always ask your physician about clinical trials. Another great source for that, I think, is the Leukemia Lymphoma Society. They’re very patient-focused, they’re very up to date on the latest therapies and the latest trial results. They have a very robust presence, both online, and also, generally locally. There’s local chapters. So, I would encourage you to reach out to them for information.

Comprehensive Hematological Cancer Centers

CLL & COVID 19: What Do Patients Need to Know?

CLL & COVID 19: What Do Patients Need to Know? from Patient Empowerment Network on Vimeo

What do people with chronic lymphocytic leukemia (CLL) need to know about COVID-19 (coronavirus)? CLL expert Dr. Steven Coutre provides guidance for patients during the current global pandemic.
 
Dr. Steven Coutre is a Professor of Medicine in the Hematology Department at Stanford University Medical Center. Learn more about this expert.

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Transcript:

Dr. Steven Coutre:

Well, of course, we are in the COVID era. We don’t know how long this is going to last. And so, a very common questions that comes up from our patients with CLL is what impact does this have on them and are they more susceptible, you know, the natural things that people wanna know. With CLL in general, there probably is some compromise to the immune system, but it’s really hard to measure or quantify. Certainly, individuals who’ve had a lot of chemotherapy in the past, who have advanced disease are more susceptible to infections. In contrast, someone who’s without symptoms, has a low burden of disease probably is close to being like somebody who doesn’t have CLL. So, there’s certainly a spectrum.

Really, we just try to advise following the guidelines that we are all following in terms of social distancing at present, at being aware of being around others too closely, or those who may have symptoms. So, I think, in a way, what everyone is doing now is something that is beneficial to patients with CLL, and certainly other cancers, with respect to infection risk.

Now, what about do we have any information? Is somebody with CLL more susceptible to getting COVID? What if you do get the infection? Is it going to be more severe because you have underlying CLL? And, at least in general terms, the answer seems to be no. That’s really just based on experience, anecdotal experience, certainly in areas like New York City or Italy, for example, where infectious rates have been quite high. Colleagues have commented that their patients don’t seem to be more ill simply because they have the underlying disease or because they’re on a certain treatment, for example.

There’s actually some very interesting data suggesting that perhaps the BTK inhibitors, Ibrutinib, Acalabrutinib, et cetera, might actually confer benefit, might lessen some of the consequences of the infection, and as a result, large clinical trials have started for patients without CLL. Just anyone who has a significant COVID infection who’s hospitalized, they’re testing that hypothesis. So, it’ll be very interesting to see what we learn from this. Perhaps what we’ll learn is that being on a drug like that might actually be beneficial.

It’s certainly natural to be hesitant to come into a healthcare facility because of the risk of infection, and certainly that’s gonna vary quite a bit depending on where you are. At the height of the pandemic in New York City, of course, a lot of concern on the part of patients going into a hospital clinic, for example. Whereas, at our institution, the impact has been quite low. All institutions, of course, have taken any precautions they can to limit exposure, so, I’ve often told my patients that it’s probably safer to come into our clinic and get your blood drawn or see someone if you need to than going to the grocery store, for example, in terms of exposure.

But that’s very different than saying the same thing in the middle of New York City. So, I think you have to deal with each situation as it arises, and one would hope that your physician can give you guidance. And I think, in particular, what we can do is really decide how important it is to see somebody in person or have them come in and get a lab test there. I think in many, many, many cases, that can be avoided for the time being.

And that also is an important point, that we can provide reassurance that you know, you’re used to coming in every four months or every six months and having things checked, and in many cases we can reassure that individual that it’s okay to wait. It’s not critical to get that information right now.

So, remember that what we often emphasize in evaluating someone and making decisions when to treat is three things. It’s how you feel, what your exam is like, and what your blood counts look like. So, of course, you know how you feel. If something changed, you’re having night sweats, or a lot more fatigue, is it significantly different? Of course, you typically know if anything’s changing with your exam. Are your lymph nodes getting enlarged?

Do you notice discomfort in your abdomen because of an enlarging spleen?

And so, two of the three things you can sort of self-assess, in a way, and then based on what your blood counts have been showing over time, your physician can factor that in and decide how important it is to get that test now. And as I mentioned, in many, many cases, it’s perfectly fine to delay that. So, it’s not as difficult as it might seem to you to be able to come up with a reasonable assessment about how somebody might be doing, even in the absence of seeing them and doing an exam in person.

How Can CLL Patients Take Advantage of Telemedicine?

How Can CLL Patients Take Advantage of Telemedicine? from Patient Empowerment Network on Vimeo.

In light of the global pandemic, many providers expanded their telemedicine options so that patients can connect with their physicians virtually and avoid in-person visits. Expert Dr. Steven Coutre explains how this approach could benefit people with chronic lymphocytic leukemia (CLL).

Dr. Steven Coutre is a Professor of Medicine in the Hematology Department at Stanford University Medical Center. Learn more about this expert.

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Transcript:

Dr. Steven Coutre:

Well, we are in a new era, at least temporarily, and, for example, we’ve switched almost exclusively to video visits. This had largely been used for patients who lived in remote areas. They didn’t have good access or ready access to healthcare providers, and so, the government reimbursed for those kinds of visits, but not for somebody who lived close by, for example.

Well, that all changed dramatically with the COVID infections, even for our patients on clinical trials. And we’ve done the grand experiment that never would have been done otherwise, of just suddenly doing all video visits, and I must say, it’s worked out quite well so far. I think patients are quite satisfied with it, by and large. It allows them to have their questions answered and continue to have appropriate monitoring if they’re on therapy, or even if they aren’t. And so, I think, when things improve, this will continue, to some extent. So, right now, I would expect that any CLL patient would have ready access to their hematologist or oncologist via video visit.

And also, I think this whole situation has promoted a lot more video conferencing, educational video conferencing. Not having to physically attend a conference in order to get information. So, I think they’ll see a lot more educational resources out there online for them.

Well, of course, with CLL, we’re also very interested in blood counts, as are our patients, and if we’re doing remote visits, or even if they live fairly close but aren’t coming in, we do try to get the lab work done, but that’s worked out quite well. We’re used to dealing with patients coming from far distances, and so, in the past, if we wanted to get a lab result in between visits, we would simply make those arrangements with their local lab. Everybody tends to have an internist, a family doctor that they see, and so they’re familiar with getting lab tests done near where they live, and in all cases, we’ve been able to accommodate that.

And now with the increasing of electronic medical record usage and interlinking of medical record systems, we can, for example, get lab tests done at a local lab and have those. Actually, those results are directly imported into the medical record. So, they’re easily accessible to us. So, I must say, it’s been a pleasant surprise to see how well this has worked.

A Look at Leukemia

What is Leukemia?

As with many other cancers, leukemia is not a singular disease. There are many types of leukemia, and while it is a common childhood cancer, leukemia actually occurs more often in older adults. Leukemia is the most common cancer in people under the age of 15, but it is most likely to affect people who are 55 or older. There are more than 60,000 cases of adult leukemia diagnosed each year, and it is more common among men than women. 

Leukemia is a broad term that describes cancer of the blood or bone marrow. It starts when the DNA of developing blood cells are damaged and the bone marrow makes abnormal cells. The abnormal blood cells are the leukemia cells which grow and divide uncontrollably. Unlike healthy cells that follow a life cycle, the leukemia cells don’t die when they are supposed to so they continue to build up, eventually overcrowding the blood. They crowd out normal white blood cells, red blood cells, and platelets so those normal cells can’t grow and function. Eventually, there are more cancer cells than healthy cells in the blood. The type of leukemia is determined based on which blood cells are affected by the abnormal cells. Leukemia usually affects the white blood cells, called leukocytes, but can occur in other blood cells. There are four main types of leukemia: chronic, acute, lymphocytic, and myelogenous.

Leukemia that grows slowly is called chronic leukemia. The cancer cells form very slowly so the body can also continue to form healthy cells, but over time the cancer cells continue to grow and the leukemia worsens. 

Acute leukemia grows very quickly and gets worse really fast. It has been identified as the most rapidly progressing cancer, and it can develop and grow in a matter of days or weeks.

Lymphocytic leukemia forms in the part of the bone marrow that makes lymphocytes, which are white blood cells that are also immune cells. Chronic lymphocytic leukemia (CLL) is most common in older adults and makes up about 25 percent of adult leukemia cases. It is more common in men than women and is very rare in children. Acute lymphoblastic leukemia (ALL) also affects older adults, but children younger than five have the highest risk of developing it.

Myelogenous leukemia forms in the bone marrow cells that produce blood cells, rather than forming in the actual blood cells. Chronic myelogenous leukemia (CML) accounts for about 15 percent of all leukemia cases in the United States. CML develops mostly in adults and is very rare in children. Acute myelogenous leukemia (AML) is a rare cancer that develops quickly with symptoms of fever, difficulty breathing, and pain in the joints. It can be caused by environmental factors, and develops more often in adults than children, and more often in men than women.

There are also several less common types of leukemia. Most of these types are chronic, and each year in the United States, about 6,000 cases of these less common leukemias are diagnosed.

  • Chronic myelomonocytic leukemia (CMML) develops from myeloid cells.
  • Juvenile myelomonocytic leukemia (JMML) is typically found in very young children and is another type of myeloid leukemia.
  • Acute promyelocytic leukemia (APL) is a subtype of AML.
  • Hairy cell leukemia is slow growing, chronic, and makes too many B cells that appear hairy wen viewed under a microscope.

Leukemia Possible Risk Factors

There are several risk factors linked to leukemia. There are environmental factors and genetic reasons why some people might develop leukemia. Some of the factors can be controlled while others can not. Age, smoking history, and exposure to hazardous chemicals are all possible risk factors. Other risk factors may include exposure to chemicals or medical treatments, personal health history, and family history. Some of the possible risk factors need more study to determine a definite link to leukemia, but being aware of your potential risk is important.

If you were exposed to chemotherapy or radiation therapy for another cancer you have a higher chance of getting leukemia later in life. Also, children who took medications to suppress their immune systems, such as after an organ transplant, may develop leukemia. Exposure to chemicals such as benzene and formaldehyde, often found in cleaning products, hair dyes, and embalming fluid, may also increase your risk of developing leukemia. Smoking and exposure to workplace chemicals like gasoline, diesel and pesticides could also be a risk factor.

There are several syndromes, conditions, and genetic disorders that can also increase leukemia risk. Li-Fraumeni syndrome, a hereditary disorder, is linked to leukemia, and children with Down syndrome have a two to three percent increased risk of developing acute myeloid or acute lymphocytic leukemia. Other genetic disorders that increase leukemia risk are Fanconi anemia, and dyskeratosis congenita (DKC). The inherited immune system conditions ataxia-telangiectasia, Bloom syndrome, Schwachmai-Diamond syndrome, and Wiskott-Aldrich syndrome also increase the risk of leukemia. Risk is also increased in patients with a history of blood disorders such as myelodysplastic syndrome, myeloproliferative neoplasm, and aplastic anemia. There are also viruses, such as the human T-lymphotropic virus (HTLV-1), linked to leukemia.

Family history can also play a role in the development of leukemia. Having a sibling with leukemia is a risk factor, and having an identical twin with leukemia gives you a one in five chance of developing it yourself.

Preventing Leukemia

There are no known ways to prevent leukemia; however, being aware of risk factors and attempting to reduce them could help. Studies have linked leukemia to smoking and obesity, so quitting smoking and having a healthy body weight could help prevent leukemia. In addition, avoiding heavy exposure to dangerous chemicals might decrease your risk.

Signs and Symptoms

There are no reliable early screening methods for leukemia and, especially in chronic leukemia, the symptoms may not be very noticeable early on. Symptoms such as fatigue and fever may not be alarming at first, and could be mistakenly attributed to other causes. Acute leukemia symptoms come on faster and are typically more noticeable. All types of leukemia can have similar symptoms, but the symptoms each individual patient has can help determine the type of leukemia. Any symptoms should be checked by a doctor.

The most common symptoms of leukemia are:

  • Extreme fatigue that doesn’t respond to a good night sleep
  • Enlarged lymph nodes that are swollen and tender as a result of leukemia cells building up
  • Unexplained fever higher than 101 degrees that occurs frequently or lasts more than three weeks with no explanation
  • Night sweats that can also occur during the day, and can drench the sheets through to the mattress
  • Bruising and excess bleeding such as frequent nose bleeds caused by poor blood clotting which is also a symptom
  • Poor blood clotting is apparent when small red or purple spots, called petechiae, appear
  • Abdominal pain occurs when white blood cells accumulate in the liver or spleen
  • Bone and joint pain usually occurs in the hips or sternum where there is a lot of bone marrow that is being crowded by abnormal cells
  • Headaches and other neurological symptoms such as seizures, dizziness, visual changes, nausea, vomiting can occur due to leukemia cells in the fluid around the brain and spinal cord
  • Unintentional weight loss of five percent or more of your body weight in 12 months or less. Weight loss can sometimes be a result of having a swollen liver or spleen which can lead to loss of appetite
  • Frequent infections occur because white blood cells aren’t working properly to fight infections
  • Anemia, or iron deficiency, occurs when there is a lack of hemoglobin in the blood to transport iron in the body. Iron deficiency can cause labored breathing and pale skin. Symptoms of anemia are nausea, fever, chills, night sweats, flu-like symptoms, weight loss, bone pain, and tiredness

Complications from Leukemia

Leukemia can cause several serious complications due to the nature of the disease and treatment. Complications such as life-threatening infections can occur when white blood cells are damaged or reduced. When white blood cells aren’t fully functioning, the body can’t properly fight infections, so any infections a leukemia patient gets, such as urinary tract infections or pneumonia, can become very serious. Low platelet counts make bleeding in areas such as the brain, the lungs, and the stomach or intestines very dangerous, while high white blood cell counts can cause leukemia cells to spill over from the blood into other organs possibly causing respiratory failure, stroke, or heart attack.

There are other complications that are related to specific types of leukemia. Notably, the development of secondary cancers and blood cancers are more likely in CLL patients. Another complication of CLL is called a Richter transformation in which the cells can transform into an aggressive form of lymphoma. Kidney failure can be a treatment-related complication of AML or ALL.

Leukemia Diagnosis 

Leukemia can’t be diagnosed based solely on symptoms, but if leukemia is suspected, in a general exam, the doctor will look for an enlarged spleen or liver and take a blood sample. Further diagnostic testing may include a bone marrow test where a long needle is used to extract marrow from the center of a bone (usually the hip). The bone marrow test will help determine if the patient has leukemia and the type of leukemia.

Staging Leukemia

Staging is used to identify the size and location of cancer in the body. Typically cancers have four stages with Stage I usually indicating the cancer is in one location and is not very large. Stage IV indicates the cancer has grown large and spread far from the original location. Most leukemias aren’t usually staged because they are in the blood and therefore have already spread throughout the body. Instead, leukemia can be considered untreated, active, in remission, or recurrent. The exception is CLL, which can spread through the lymph nodes or the blood or bone marrow, so it does have three stages.

Treatment

The earlier treatment starts for leukemia, the better chance of remission. However, thanks to some exceptional advancements in leukemia treatment medications, doctors are often able to take the time they need to come up with the best treatment plan for each individual with leukemia, even in cases of acute leukemia if life-threatening complications are not present. When coming up with a treatment plan, doctors consider the patient’s age, overall health, and most importantly, the type of leukemia the patient has.

Leukemia treatment options vary for each type of cancer:

Watchful Waiting is used when treatment for slower growing leukemias, such as CLL, may not be necessary;

Chemotherapy is the primary treatment for AML, and sometimes a bone marrow transplant is needed;

Targeted therapies are medications that are tyrosine kinase inhibitors which target cancer cells, but don’t affect healthy cells. Targeted therapies have less side effects. Many CML patients have a gene mutation that responds very well to targeted therapy;

Interferon therapy is a drug that acts similar to a naturally occurring immune response which slows and then stops the leukemia cells. This therapy can cause severe side effects;

Radiation therapy is often used in ALL to kill bone marrow tissue before a transplant is done;

Surgery to remove the spleen may be necessary, depending on the type of leukemia;

Stem cell transplant is effective in treating CML and is usually more successful in younger patients. After chemotherapy or radiation or both are used to destroy the bone marrow, new stem cells are implanted into the bone marrow so noncancerous cells can grow.

Treatment for acute leukemia can take up to two years. It is usually done in phases. In the first phase the goal is to use chemotherapy for several weeks to kill the cancer cells and put the patient in remission. The second phase is designed to kill any remaining cancer cells using chemotherapy or stem cell transplant or both. The treatments and their side effects can be pretty harsh for older patients so researchers have been focusing on finding targeted therapies for acute leukemia, which have fewer side effects. Researchers are also hoping CAR T-cell therapy, which uses the patient’s own immune system to treat cancer, could be an eventual replacement for stem cell replacement therapy in older ALL patients. AML is more aggressive and often harder to treat, but several new targeted medications have been approved to treat AML. Researchers continue to look at other targeted therapy options and other drugs for AML.

In some cases of chronic leukemia, a stem cell transplant might be required, but the main treatment is oral medications that patients will probably take for the rest of their lives. Some research is investigating whether or not patients could potentially stop taking the medications at a certain point. 

CML treatments have really advanced and there are now several drugs that target the abnormal protein that causes CML. Thanks to these targeted medications CML patients now have a close to normal life expectancy and a 90 percent five-year survival rate. Clinical trials are looking at using targeted therapies to treat CLL as well and CAR T-cell therapies are also being considered for CLL treatment.

Recovery and Survival

Leukemia represents 3.5 percent of all new cancer cases in the United States, and it is the seventh leading cause of cancer death. The outlook for leukemia patients depends on which type of leukemia they have, their overall health, and their age. Leukemia is more likely to be fatal in older patients. The average age of those who die from leukemia is 75. However, the many advances in treatment options and medications, such as targeted therapies, have created a better prognosis for many. Leukemia has a 62.7 percent five-year survival rate, and some people with leukemia can now achieve complete remission.


Sources

Felman, Adam. “What to Know About Leukemia” Medical News Today, medically reviewed August 28, 2019, https://www.medicalnewstoday.com/articles/142595. Accessed March 9, 2020.

Raymaakers, Karen. “Symptoms of Leukemia” Verywell Health, medically reviewed November 1, 2019, https://www.verywellhealth.com/leukemia-signs-and-symptoms-2252435. Accessed March 9, 2020.

“Adult Leukemia: What You Need to Know” Dana-Farber Cancer Institute, updated December 5, 2019, https://blog.dana-farber.org/insight/2019/11/adult-leukemia-five-things-you-need-to-know/. Accessed March 9, 2020.

Wang, Eunice. “How Fast Does Leukemia Develop” Roswell Park Comprehensive Cancer Center, October 4, 2018, https://www.roswellpark.org/cancertalk/201810/how-fast-does-leukemia-develop. Accessed March 9, 2020.

“Reducing Your Risk for Leukemia” Canadian Cancer Societyhttps://www.cancer.ca/en/cancer-information/cancer-type/leukemia/risks/reducing-your-risk/?region=on. Accessed March 9, 2020.

“Risk Factors for Leukemia” Canadian Cancer Societyhttps://www.cancer.ca/en/cancer-information/cancer-type/leukemia/risks/?region=on. Accessed March 9, 2020.

Stöppler, Melissa Conrad. “Leukemia” MedicineNet, medically reviewed September 11, 2019, https://www.medicinenet.com/leukemia/article.htm. Accessed March 9, 2020.

“Leukemia Screening” Moffitt Cancer Centerhttps://moffitt.org/cancers/leukemia/diagnosis/screening/. Accessed March 9, 2020.

“Leukemia — Patient Version” National Cancer Institutehttps://www.cancer.gov/types/leukemia. Accessed March 9, 2020.

“Cancer Stat Facts — Leukemia” National Cancer Institute Surveillance, Epidemiology, and End Results Programhttps://seer.cancer.gov/statfacts/html/leuks.html. Accessed March 9, 2020.

“Advances in Leukemia Research” National Cancer Institute, June 25, 2019,https://www.cancer.gov/types/leukemia/research. Accessed March 9, 2020.

Patient Profile: Perseverance and Positive Thinking Helped This Young Mother

Cancer is a stealthy assailant. Symptoms can be nonexistent or masquerade as some other ailment. When a medical professional utters the “C” word, the shock can be intense.

“I had no idea,” said Lindsay Hutchings of the softball-sized tumor that had been growing in her chest behind her breastbone. “I just knew I didn’t feel right.”

Lindsay was 34 at the time of her diagnosis. A mother of two young children, she never suspected cancer when she started feeling unwell. It was October. Time for picking Halloween costumes and the season when colds spread like wildfire through schools.  A mom with young kids feeling fatigued and achy was nothing to be alarmed about.

Lindsay went to a walk-in clinic. When she didn’t improve, she went to her primary care doctor. She was given antibiotics. She was tested for the flu and then mono. Allergies were blamed and antihistamines suggested. Every week she was back in either the walk-in clinic or her primary care doctor’s office.  Until one morning she woke up with a swollen neck and jaw.  She knew this was not just a stubborn cold. She knew it wasn’t allergies.

“This time I was diagnosed with a sinus infection and referred to an Ear, Nose & Throat (ENT) physician. It was frustrating because I knew it wasn’t a sinus infection. I just didn’t have any idea what it could be.”

By this point, Lindsay’s husband, Jake, was going to appointments with her in hopes he might think of some question or detail she had missed. The ENT doctor examined Lindsay and listened to the path that had brought her and her husband to see him. He scoped Lindsay’s sinuses and found nothing.

Then he ordered a CAT scan and posed the possibility that Lindsay’s symptoms might not be related to a virus, allergy, or superbug. It might be cancer.

She was told to expect to wait three weeks for the results of her CAT scan because of the Christmas and New Year’s holidays, but the ENT called her after a few days with the results.  He suspected lymphoma and referred her to an oncologist.

Lindsay started the New Year off by having two biopsies and a PET scan to confirm what the ENT had suspected.  Four months after she first began feeling off, Lindsay had an answer. It was Stage IIB Hodgkin Lymphoma.

About Hodgkin Lymphoma

Cancers that start in white blood cells—also called lymphocytes–are categorized as lymphomas. The two main types of lymphomas are Non-Hodgkin Lymphoma and Hodgkin Lymphoma. Hodgkin Lymphoma (HL) can start in any lymphoid tissue in the body, such as the spleen, bone marrow, thymus, adenoids or tonsils. However, it most often starts in lymph nodes in the upper part of the body. Lymph nodes are bean-sized collections of lymphocytes and other immune system cells and are located throughout the body.

The causes and triggers for HL are unknown. Children and adults can develop Hodgkin Lymphoma. The average age at the time of diagnosis is 39. Although there is a higher rate of lymphomas in people with immune disorders, there is usually no known risk factor or cause for people diagnosed with HL.

There are four subtypes of classic HL and a rarer form of HL called nodular lymphocyte-predominant Hodgkin lymphoma (NLPHL). Treatment for the disease varies depending on what type the patient has, what stage the disease is in (I, II, III, or IV), and whether certain other symptoms are present (called B symptoms).

Cancer’s Emotional Side Effects: Shock, Optimism & Guilt

From the first mention of lymphoma by the ENT physician Lindsay began researching the disease online. She went to her first post-diagnosis oncology appointment armed with questions. Her oncologist patiently answered her questions and laid out a treatment plan he felt confident was the right one. He explained the survival rates were high and the rates of recurrence of HL were low. He assured Lindsay and Jake her prognosis was good.

“I was in shock. I had no idea what I was doing but he was responsive and reassuring. I would call or email my oncologist between appointments with questions and he always responded. It really helped me be positive and more confident,” Lindsay explained.

She needed that positive confidence to help with the first challenge that followed on the heels or her diagnosis. She and Jake had to sit down with their daughters—who were just four and seven—to explain their mommy had cancer and what that meant for their family.

Lindsay’s mother had passed away from lung cancer just two years prior.  It was hard for seven-year-old Delaney to understand that this cancer was different from her grandmother’s.  She became anxious after the effects of treatment began taking their toll that Lindsay might die just as her grandmother had.

Her younger daughter had a hard time distinguishing the kind of sick her mommy was from the everyday illnesses she and her friends might have.

And of course, once treatment began, there was a lot Lindsay couldn’t do for her children anymore. Some days she couldn’t get out of bed. Often Lindsay was unable to take them to school or help with homework. She stopped volunteering at their schools. A low point for their family came early in Lindsey’s treatment when Delaney came home from school sick Lindsay had to avoid being near her. Meanwhile, her husband worked from home as much as he could or took time off to care for Lindsay and their daughters.

Lindsay admits, “I still feel guilt for the burden I put on my family.”

The Cancer Journey Continues

Lindsay’s cancer was treated with chemotherapy and radiation. There were side effects, of course:  exhaustion, chemobrain, nausea, constipation, blood clots.  She had to get daily shots of blood thinners in the doctor’s office, which brought other risks and complications. There were moments of panic when it seemed the tumor had stopped responding and additional biopsies followed.  But in the end, nearly a year after Lindsay first began experiencing symptoms, she was declared cancer-free.

There is a sense of victory from beating cancer. But like many other cancer survivors, Lindsay can’t say her cancer journey has truly concluded.

“At this point I am cancer-free, but I’m paranoid. It [the tumor] got so big without me knowing! I feel like some part of me will always worry.”

Ten to 30 percent of HL patients experience recurrence of the disease, with recurrence being lowest for those who are treated in the early stages of the disease. However, rates of developing a second cancer are higher for HL survivors than the general public regardless of whether the lymphoma returns.

Lindsay will have quarterly follow-up visits and two scans this year to screen for recurrence. As long as her results remain normal, she can scale back to annual screenings the following year. However, because she’s now at higher risks for other medical issues, she needs annual screenings by a cardiologist and pulmonologist. She will have annual mammograms now, instead of waiting until age 40 or 45. She has a roster of doctors at a time of life when many of her peers are in peak health.

The upside, Lindsay says, is that if any of her friends or family ever need a recommendation for a specialist in town, she has her list ready!

In addition to the health concerns she will carry, Lindsay also continues to deal with fatigue. Fortunately, while undergoing treatment Lindsay was able to connect with, Brittany, a two-time survivor of Hodgkin Lymphoma.  Brittany used to teach at Delaney’s school and when she heard about Lindsay’s diagnosis, she tracked down her phone number from a friend because she knew from experience how helpful it is to hear perspective and encouragement from someone who has been there before.

“When I went to my appointments, I was usually the youngest person in the office by decades,” Lindsay explained. Talking to someone closer to her own age has helped.

In addition to finding a connection with a fellow survivor, Lindsay is also grateful for the support she and her family received from friends and family in their community. During her treatment, Jake and Lindsay’s family helped as they could.  But there was also an outpouring of support from friends and acquaintances who helped with meals, gift cards, and donating to a GoFundMe account for the family.

“If I could give one piece of advice,” Lindsay says, “it would be to build your community. If we didn’t have friends and family to help get us through, I don’t know what we would’ve done. You can’t be involved in your community or volunteer or even go to church when you are sick. But I am so grateful that we were involved and active before the diagnosis.”

At a time when she saw few people and did very little outside of her home, she felt buoyed by the care and concern of from relationships she’d established before her illness. So, along with the guilt and worry she may carry forward from her cancer journey, she will also carry an abiding appreciation for the value of a supportive community.

Life for the Hutchings family is gradually settling back into normalcy, with family vacations and school schedules supplanting doctors’ appointments and treatments. Lindsay can begin to enjoy her new mantle of ‘cancer survivor’ with increasing confidence. But rather than consign the experience to something that “happened” to her, she is sharing what’s she learned from the experience. She would not claim to be an expert in cancer and its treatment options. But she does advocate for the power of building relationships and positive thinking. These are lessons that can help others whether they are confronting cancer or any other life-altering ordeal.

ASH 2019: Timely Myeloma Care Makes a World of Difference; Experts Prioritize Addressing Race-Associated Risks

Diverse Health Hub and the Patient Empowerment Network will partner to produce ongoing educational programs beginning in 2020. These programs identify demographic disparities found in existing diagnostic and treatment practices for multiple myeloma. Program content and educational resources will supply actionable and meaningful material tailored to healthcare providers, patients, and patient care teams. When patients feel heard and understood by their healthcare providers, they are more likely to participate in clinical trials and advocate confidently for treatment options. Our joint goal is to empower a targeted and unique population of myeloma patients to spark life-saving conversations with their providers. Be sure to sign up for PEN’s newsletters to learn more.


Onsite at ASH 2019, Diverse Health Hub interviewed prominent myeloma researchers, including questions from our members.

Is earlier effective treatment for a deeper response keeping myeloma at bay? Yes. According to new evidence around timing of treating myeloma presented at ASH 2019, immunotherapy drug daratumumab (DARZALEX) demonstrated it could repeatedly attack marker CD38 – a game changer. Dr. Sikander Ailawadhi sheds light on these new findings: “In the past the thought was that once the patient was treated by a drug that targets one particular marker that whole pathway or that mechanism of action is gone, but there was data presented at ASH, which we are all very encouraged about. Patients who have let’s say been treated with daratumumab (DARZALEX)—so one drug affecting that pathway – when they had disease progression at some point, they were treated with a brand-new drug going in for that pathway and the patients got very good deep responses.Watch the complete interview below.

  • Myeloma Treatment: Earlier effective treatment for a deeper response to keep disease quiet
  • New Drugs: 2020 to be a big year for myeloma, drug approval buzz
  • Encouraging Data: News at ASH 2019 reveals CD38 marker can be targeted repeatedly

Are disparities shortening the lifespan of a subset of myeloma patients? Yes. Several published papers indicate that the burden of disease was higher for a subset of myeloma patients as a result of socioeconomic status, age, race, lack of resources, access, and insurance type. Dr. Ailawadhi identifies the need for programs that educate both patients and providers to mitigate underlying disparities. Watch the complete interview below.

  • Access to Care: Significant number of minority patients unaware of medical record access
  • Burden of Disease: African Americans and Hispanics get treatment later than whites; costs tend to be higher for minority patients
  • Observation: More frequently diagnosed with myeloma later stage, at a younger age
  • Need: Educate patients, educate providers. Patients need to be their own advocates and direct the conversation with their providers in order to get to the right expert care

What role does education and awareness play in the diagnosis of ethnic myeloma patient populations? Despite advances in the treatment of multiple myeloma, Dr. Ajay Kumar Nooka identifies a gap between patient education and awareness of current therapeutic options. Dr. Nooka discusses how myeloma presents in various ethnic groups, and identifies disparities in access to initial treatment for African Americans and Hispanic populations. Nooka says, “education and awareness is the biggest gap we tend to see.” Watch the complete interview below.

  • Good news: “Really good time in myeloma, more therapeutic options”
  • Need Improvement: Education and awareness gaps still need to be filled; disparities among people of color, long road to diagnosis, delays and access to drugs
  • Clinical Trials: Lack of minority awareness and participation in clinical trials contributes to treatment disparity

About Diverse Health Hub:

Diverse Health Hub is a health equity education and awareness channel producing educational content for both patients and providers in order to bridge the gaps between healthcare practices and the needs of multicultural communities.  Diverse Health Hub works directly with a diverse patient and respected provider population in multiple therapeutic areas to promote cultural competence in healthcare. The organization believes access to these diverse perspectives cultivates culturally competent communities.