Tag Archive for: hematology

How Does Essential Testing Affect Myeloma Care and Treatment?

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How Does Essential Testing Affect Myeloma Care and Treatment? from Patient Empowerment Network on Vimeo.

 Why is it important to ask about essential testing for your myeloma? Find out how test results could reveal more about your myeloma and may help determine the most effective care for your individual disease.

See More From INSIST! Myeloma

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What Should You Ask Your Doctor About Myeloma Testing?

Transcript:

Why should you ask your doctor about essential myeloma testing?

When a patient is diagnosed with myeloma, they typically undergo a series of tests that aid in diagnosing and staging their individual disease. The standard tests include:

  • Blood Test
  • Urine Test
  • Bone Marrow Biopsy, and
  • Imaging

As research in the field evolves, genetic profiling via more in-depth cytogenetic testing is increasingly common to further classify your myeloma. This testing often identifies unique biomarkers of the myeloma, such as translocations or changes in chromosomes.

So why do the results of these tests matter?

  • The presence of certain biomarkers can indicate a patient is low-risk, which can suggest a more positive prognosis.
  • There are certain biomarkers that indicate high-risk myeloma, meaning an aggressive treatment approach may be more effective.

Knowing your risk in myeloma is useful to your healthcare team when choosing a treatment approach or may help in determining if a clinical trial might be right for you.

How can you Insist on the best care for YOUR myeloma?

  • First, always speak up and ask questions. Remember, you have a voice in YOUR myeloma care. Your doctor is expecting you to ask questions and should be able to answer them.
  • Ask your doctor if you have had or will receive genetic testing for risk stratification and how the results may impact your care and treatment plan. Be sure to ask for paper or electronic copies of your important test results.
  • And finally, bring a friend or a loved one to your appointments to help you process information and to take notes.

To learn more about your myeloma and access tools for self-advocacy, visit powerfulpatients.org/myeloma 

Which Myeloma Treatment Is Right for You? What You Need to Know

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Which Myeloma Treatment Is Right for You? What You Need to Know from Patient Empowerment Network on Vimeo.

What should you know before deciding which treatment is best for YOUR myeloma? Myeloma expert Dr. Saad Usmani reviews essential testing that may help guide treatment decisions, and discusses the impact of risk stratification on myeloma care. Dr. Usmani also provides an overview of treatments in development, the importance of clinical trials, and shares why he’s hopeful about the future of myeloma research.

Dr. Saad Usmani is the Chief of Myeloma Service at Memorial Sloan Kettering Cancer Center in New York City. Learn more about Dr. Usmani, here.

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See More From INSIST! Myeloma

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Essential Tests & Imaging After a Myeloma Diagnosis

Lab Tests in Myeloma: Key Results to Monitor

Myeloma Targeted Therapy: Why Identifying Chromosomal Abnormalities is Key

Transcript:

Katherine:

Hello. And welcome. I’m Katherine Banwell, your host for today’s program. Today, we’re going to discuss how to access the most personalized care for your myeloma and why you should insist on essential testing. Before we get into the discussion, please remember that this program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you. Okay. Let’s met our guest today. Joining me is Dr. Saad Usmani. Dr. Usmani, would you introduce yourself please?

Dr. Usmani:

Certainly. Thank you for inviting me, Katherine. I’m Saad Usmani. I’m the incoming chief of myeloma at the Memorial Sloan Kettering Cancer Center in New York.

Katherine:

Excellent. Thank you for taking the time out of your schedule to join us today. Before we delve into the discussion, let’s start by defining a term that we’re hearing more frequently. What is personalized medicine?

Dr. Usmani:

Personalized medicine is a fancy term to examine different aspects of a patient’s health outside of their cancer diagnosis. And also, the cancer itself – factors that are associated with good response to treatment or an early relapse from treatment. So, it’s a holistic kind of an approach that looks at all of these factors together. Also, looks at the patient’s mental and social well-being and comes up with a game plan for them.

So, I would probably divide the various factors that kind of come into play with the personalized medicine or personalized approach to cancer treatment by taking into account factors that are patient-related, factors that are cancer- or disease-related, and then factors that are related to treatments that they maybe receiving.

So, these three kinds of combined together to form a plan that is unique to that individual patient.

Katherine:

Right. What tests are necessary to help understand a patient’s specific disease both at diagnosis and prior to treatment?

Dr. Usmani:

So, the testing includes – what’s the objective of testing – we do tests to help in diagnosis to assess how much of cancer we’re dealing with and then what kind of cancer we’re dealing with. Even within a given cancer, how much cancer you have and what kind you have is important. Folks can have a little bit of cancer in

terms of burden. But it can be aggressive in its nature. So, you can have King Kong at your door, or it could be the green giant just trying to serve up veggies. Whereas King Kong will bite your head off.

So, with that in mind, there are things that we do such as blood tests to see effects on blood counts, kidneys, liver. We also do certain blood tests to identify what kind of multiple myeloma a patient may have as an example. So, the kind of myeloma protein they’re secreting. The kind of light chain they’re secreting. Then urine tests are done to see if there are any proteins that are leaking through the kidneys if there is kidney damage. Then bone marrow biopsy to a) look at how much myeloma and b) what kind by specific testing that we do on the bone marrow biopsy. And then imaging to see what parts of the bone’s affected.

Katherine:

Great. I’m assuming that these tests will help with the opening of the stages of myeloma.

So, how is myeloma staged?

Dr. Usmani:

So, the staging of myeloma is still a work in progress. The reason why I say that is we have a good way of accessing how much myeloma a patient may have. But if we don’t combine it well with what kind or how aggressive it may be. So, staging in myeloma relies on two blood tests that are serum albumin and serum beta-2 macroglobulin.

And they help us give a good assessment of how much myeloma patients have. And maybe a little bit of information about whether patients may have a bit more aggressive kind. But then you overlay that with cytogenetic information from the myeloma cells that are from the biopsy as well as another blood test called LDH.

If patients have any of the quote unquote high risk features, they are – along with a high level of beta 2 microglobulin, you stage them as stage three. If they don’t have them, they’re stage one. If they have some of the features, they’re kind of in between in stage two. And that’s how we stage multiple myeloma.

Katherine:

You mentioned cytogenetics. What testing is involved in that?

Dr. Usmani:

So, bone marrow biopsy – it’s very broad. But there are two parts to it.

One part is getting the bone marrow aspirated where we insert a needle into the pelvic bone and get parts of the bone marrow – the blood inside the bones out. And look at how much percentage of plasma cells are there. What kind of surface markers or features they have.

And then we look at if those cancer cells have any chromosome abnormalities that are unique to myeloma. And some chromosome abnormalities can be high-risk.

What does high-risk mean? High-risk means if you treat patients in a certain fashion, they have a higher chance of relapsing or a higher chance of the myeloma coming back out of remission. So, we identify those features by way of looking at cytogenetics. And there are different techniques in which we can take a look at that.

Katherine:

And what are those techniques? There’s something called FISH, right?

Dr. Usmani:

Yes.

Katherine:

And flow cytometry and also next generation sequencing?

Dr. Usmani:

Yes. So, and there is also conventional cytogenetics. So, flow cytometry looks at the different proteins that are part of the surface of any cell – any blood cell for that matter. It could also be any other cell as well, not just blood cells.

But in this particular case when we do flow on the blood marrow aspirate, we’re looking for unique features of those myeloma cells. But that does not tell us anything about the chromosomes. Conventional cytogenetics is the old fashion way. It’s a 40 – 50-year-old technique in which you make the cells in a test tube. You make those cells go through cell division. Each human cell has 46 chromosomes or 23 pairs. And when the cells are dividing, those chromosomes kind of line up in the center.

And the old fashion technique of conventional cytogenetics was take a look at the cells when those cells – when the chromosomes are aligned, and see if some parts of the chromosomes are missing or one chunk of one chromosome has attached to the other. That’s the old fashion way. The FISH technique, what it does is it’s geared toward identifying specific abnormalities.

And one part of that particular protein or molecule that goes and attaches to that chromosome has a color-coded probe. So, you can see within a cell different colors light up. And based on those unique features, you can identify “Okay. This cell over here is missing a part of chromosome 17. Or this part of chromosome 14 is attached to chromosome 4.” That’s FISH. So, FISH is very specific. Conventional cytogenetics is not. Next-generation sequencing, there are – that’s a broad term. You can measure different types of nucleic acids: RNA versus DNA. And those different techniques identify specific – they can identify specific mutations in a cancer cell.

So, each of these techniques provide different layers of information for our myeloma patients.

Katherine:

Thank you for that explanation. I appreciate it. How can the results of these tests affect prognosis and treatment?

Dr. Usmani:

So, currently for the most part, we’re treating myeloma patients in a similar fashion. Except for some tweaking based on these quote unquote high-risk features. So, there are certain chromosomes abnormalities that tell us that a patient has a higher chance of relapsing early even if they get the standard of care treatment. So, we try to enroll those patients into a clinical trial or have better optimization of their induction treatment and their maintenance strategy.

So, identifying these high-risk abnormalities is important because our treatment decisions may be modified for that patient’s disease. Or we might be able to get them to a clinical trial sooner than later.

Katherine:

Right. What is risk stratification? And how is it used in patient care?

Dr. Usmani:

So, risk stratification helps us identify people who are going to do well in terms of getting to a good response and maintaining that response and maintaining being progression free or being disease free versus those folks who maybe relapsing sooner. And that’s called risk stratification. So, you are essentially identifying and dividing patients into two different buckets saying, “All right. I have to pay attention to this person a bit more because they can relapse soon. So, I’m going to be keeping an eye on their labs and such very much, much closely.”

Katherine:

Let’s talk about therapy for myeloma patients. How are low-risk patients treated?

Dr. Usmani:

So, typically, the low or standard risk patients are treated with at least a three-drug induction treatment at the time of diagnosis. Or sometimes with four-drugs if you combine an antibody treatment. There are various regimens but the standard of care is at least three drugs. Then for patients who may be eligible for a stem cell transplant, they go on to receive autologus stem cell transplant.

Once they’ve recovered from the stem cell transplant, they go on to maintenance treatment.

And the idea is that the induction along with stem cell transplant for those patients who are eligible gets patients to as deep as a response as possible. And the concept of maintenance is you maintain them in that response and delay the disease from coming back.

Katherine:

Right. And then what about high-risk patients? How are they treated?

Dr. Usmani:

So, for high-risk patients, we typically prefer using a four-drug regimen. Either daratumumab (Daralex) RVd or carfilzomib (Kyprolis) with len dex or KRd as induction treatment for high-risk patients. After the stem cell transplant, most patients would continue both the lenalidomide as maintenance along with the proteasome inhibitor. If f patients had low or standard risk disease, they would only be getting lenalidomide as maintenance. So, here for high-risk patients, you’re adding a proteasome inhibitor.

Katherine:

Right. I see. Okay. And where do clinical trials fit into treatment?

Dr. Usmani:

So, as a clinical researcher, I’m a big proponent of telling my patients that if there’s a clinical trial that’s available to you, it doesn’t matter which stage of disease you’re at. Whether you’re newly diagnosed, or another myeloma has come back. Consider a clinical trial as your first and best option. Talk to physicians about both the standard of care options as well as clinical trial options.

Most clinical trials in myeloma are not someone getting treatment and the other person not getting anything. The trials that we’re doing, patients are getting at the very least the standard of care treatment. So, I would say that the – yeah. I mean, the clinical trials end up being the best option for majority of patients instead of standard of care.

Katherine:

Who is stem cell transplant right for?

Dr. Usmani:

So, stem cell transplant are kind of a misnomer. There is nothing magical about getting your own – collecting your stem cells and giving them back to you. I think the stems cells are – the way that – what they’re really doing is helping the patients bone marrow recover from the melphalan chemotherapy that’s given as part of the stem cell transplant because it’s melphalan, which was our first anti-myeloma medicine discovered back in the ‘50s and early ‘60s. That has been a mainstay of treatment of myeloma for six, seven decades now.

But if you give high doses of melphalan, there’s certain side effects. It can damage the stem cells and delay blood count recovery. So, that’s why patients get stem cells. So, in the body of evidence we have, most myeloma patients would be eligible for a stem cell transplant either at the time of diagnosis or if they decide to collect their stem cells and hold it back for the first relapse. That would be the other setting. But age is not a barrier. It’s more about how fit a patient is. And this is where a comprehensive myeloma geriatric assessment becomes important because an eyeball test is not good enough. You need to have more complex assessment of patients. So –

Katherine:

So, this is looking at comorbidities.

Dr. Usmani:

It is looking at comorbidities.

It’s looking at performance status. It’s looking at cardiopulmonary reserve. It’s looking at cognition and mental health as well. So, all of those factors. And obviously besides that, if you don’t have good social support, then going through a stem cell transplant becomes a challenge as well. So, there’s all these factors that kind of come into play together.

Katherine:

Yeah. Dr. Usmani, how is immunotherapy advancing in this field?

Dr. Usmani:

I think that’s the big area of research and clinical therapeutics over the past five or six years is immunotherapies. And it’s a broad umbrella. There are a few things that kind of fall under it – under that category.

So, it includes antibody-based treatments, includes CAR T-cell therapies. Yeah. I mean, it’s a very active area. Again, we can have a one-day seminar just talking about all the advances that are happening in that specific space. But that’s the new frontier. I think that’s the immunotherapies play a big role in finding a cure for myeloma.

Katherine:

You mentioned CAR T-cell therapy. Is it showing a lot of promise in myeloma care and treatment?

Dr. Usmani:

It is in the relapse refractory as in the advance refractory patients as well as in early relapse patients. And we are just starting to do clinical trials in newly diagnosed, high-risk patients. So, yes. It’s showing good promise. One advantage of CAR T-cell therapy is once you get the CAR T-cell therapy, it’s a one and done deal.

You just get CAR T-cell therapy and there’s no maintenance. So, patients really enjoyed that part of being off of therapy. They go into remission and then they don’t have to take anything for months or even a few years. So, I think that’s the biggest excitement about CAR Ts.

Katherine:

Yeah. Once a patient begins therapy, how do you monitor whether a treatment is working?

Dr. Usmani:

So, as part of the diagnostic work up, we typically have identified in the blood using serum protein electrophoresis and serum free light chains. What kind of myeloma proteins these – that particular patient’s myeloma cells are making. And we can monitor them every cycle of treatment. So, every three or four weeks.

And that’s the most noninvasive way of seeing if the treatment is working. The second obviously important thing is if someone has symptoms. If they have kidney damage, if they have bone pain, all of those things start improving as you’re getting treatment. And then in some patients, we’re also looking at imaging like PET CT scans at certain time points. And at some point, we do also look at the bone marrow biopsies to see what’s really going on in the factory.

Katherine:

We often hear the term MRD, or minimal residual disease used in the myeloma space. So, what is it exactly and how is it used in patient care?

Dr. Usmani:

So, minimal residual disease is a way to measure how much myeloma is left over in a given patient.

And historically, we were simply looking at the serum proteins and the light chain levels along with just the morphology of the bone marrow to see if – kind of determine a response. But we can have a much deeper assessment of how many cancer cells as a leftover from a bone marrow biopsy by different measurements. Someone can be in a complete response with M-Spike is gone. The light chains have normalized.

Yet they can still have 10,000 – 100,000 myeloma cells still in the bone marrow. And just using the bone marrow biopsy the way that we used to, we won’t be able to see them. We’ll just see, “Oh, these look like normal plasma cells.” So, using next generation sequencing and flow cytometry, we can look at normal myeloma cells at a very deep level – one out of one million.

But these tests are highly specialized. And especially the flow cytometry requires a lot of expertise. The NGS requires good sampling at the time of diagnosis as well as subsequent specimen.

Katherine:

Here’s a question we received from a viewer before the program. Mary writes: “I was just diagnosed with MGUS, and I’m obviously very concerned. What should I be looking for and how often should I check in with my doctor?”

Dr. Usmani: That is a very good question. MGUS is a precursor disease to myeloma and other class cell muscle disorders. And based on the original homestead county data from the mayo clinic, if there were 100 folks who had MGUS, one out of 100 every year would – there’d be one percent likelihood of them progressing to myeloma or some other plasma cell disorder.

So, the overall risk say in the next 20 years for a given patient is fairly low. And what we look at when we’re determining how frequently to check the blood or see the patient is the value of that M-spike.

If it’s a high value, if it’s two or three, we’ll be checking the labs more frequently every three months or so. Maybe seeing them every six months for the first year or two. If the M-spike value is very low, it’s one gram or less, we might be just checking labs once or twice a year and seeing patients once a year. But I would highly recommend in addition to seeing your regular hematologist who diagnosed you with this MGUS to do seek an opinion at a myeloma center of excellence.

Katherine:

Okay. If a patient is interested in participating in a clinical trial, what question should they ask their doctor?

Dr. Usmani:

The question that they should ask each time when you’re at that fork is can you please share with me what clinical trial options I have and compare them. Give me more information about “How do they compare with the standard of care treatments that are being offered?” And if you do not have any clinical trial options, would it be worthwhile, to again seek an opinion at a myeloma center of excellence to see if there are clinical trials available.

And in today’s day and age, you can have a virtual consult with a myeloma center of excellence. You don’t have to even go in. You can just chat with an expert on video and see if a clinical trial maybe right for you.

Katherine:

Are there common misconceptions you hear from patients concerning clinical trials?

Dr. Usmani:

Yeah. I think the most common perception patients have is “Oh, I’m going to be used a Guinea pig for something that hasn’t been used in humans before.”

Katherine:

In a human before. Exactly.

Dr. Usmani:

So, most of the clinical trials are not first in human trials. Yes. We do have first in human trials where we are using novel treatments in some instances.

But there is strong rational and safety guardrails built around that. And if you’re participating in a first in human study, it’s highly likely that the other treatments have stopped working and there might not be other options. However, majority of trials that patients end up participating in are getting at least the standard of care treatment. So, I think it’s very clear to kind of communicate this to patients that, “Hey, you are going to be getting a standard of care treatment even if you go on the quote unquote control arm. It’s not that you’re getting placebo.”

So, I think clarifying what the protocol is, giving patients information kind of alleviates some of those concerns. But that’s the most common misconception people have.

Katherine:

If patients are concerned about voicing their concerns and I think many of us are, why should they feel like they’re a partner in their care?

Dr. Usmani:

Well, that’s the only way that they will feel empowered. And we have to remember why we’re doing this, right? So, we’re doing this so that we can alleviate the burden of this disease from our patients and give them as good of quality of life as possible. And it’s a partnership. And in that partnership, the patient is the most important partner. Everyone else – it’s like you’re the main character.

The patient’s the main character in the movie. And all of us are supporting cast around them. I think that’s how you have to approach it. That’s how – that’s why it’s very important. And of course, patients – we’re not expecting our patients to read the papers and be knowledgeable about everything. But have a general sense of what to expect and it will be – so, having a more educated patient helps them deal with treatments better and have realistic expectations of what’s to come.

Katherine:

Right. As I mentioned at the start of this program, Dr. Usmani, patients should insist on essential myeloma testing prior to choosing a treatment. As we conclude, I think it’s important to point out that some patients may not know if that can even receive these important tests. So, what key question should they ask their physician about them?

Dr. Usmani:

So, you should be asking your physician about what kind of myeloma you have? What stage of myeloma you have? How much involvement in the bones you have? Do you have any chromosome abnormalities or any features of disease that put you at a higher chance of the myeloma coming back?

As you ask these questions, your physician will be prompted to think about “Okay. Am I missing something in my work?” And you can always ask is there anything else you need to do in terms of testing to give you a better idea of how best to approach my treatment and follow up.

Katherine:

I’d like to close by asking about developments in myeloma research and treatment.

What’s new that you feel patients should know about?

Dr. Usmani:

Oh, my. We can spend a long time with this answer. I would say that we understand what’s driving myeloma as a disease. We have a better understanding of what’s going on with the rest of the immune system and the bone marrow microenvironment where the myeloma cells live. So, the treatments that are being developed right now are trying to combine different ways in which you can shut the myeloma cell down by targeting those abnormalities or those abnormal pathways. And also, to harness the patient’s immune system to go after the cancer cells. So, combining what we’re calling immunotherapy with small molecule or more cancer directed treatments.

So, I think that’s kind of where the field is headed. And it’s – these are smarter strategies, smarter treatments. And we’re moving away from old fashioned conventional chemotherapies.

Katherine:

Dr. Usmani, thank you so much for joining us today. It’s just been a pleasure.

Dr. Usmani:

It’s been my privilege. Thank you so much for inviting me to this.

Katherine:

Thank you. And thank you to all of our partners.

To learn more about myeloma and to access tools to help you become a more proactive patient, visit PowerfulPatients.org. I’m Katherine Banwell. Thanks for joining us today.

 

Which Tests Do You Need Before Deciding on an AML Treatment Path?

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Which Tests Do You Need Before Deciding on an AML Treatment Path? from Patient Empowerment Network on Vimeo.

 Why is it important to ask about biomarker testing for your AML? Find out how test results could reveal more about your AML and may help determine the most effective treatment approach for your individual disease.

See More From INSIST! AML

Related Resources:

AML Targeted Therapies, What’s Available and How Do They Work

 Factors to Consider When Choosing an AML Treatment

 Understanding Key Tests That Affect AML Treatment Choices

Transcript:

Why do you need biomarker testing before deciding on a treatment plan for your acute myeloid leukemia—also known as AML?

The results may predict how your AML will behave and could indicate that one type of treatment may be more effective than another.

Biomarker testing—also referred to as risk stratification, genetic testing, or molecular testing—identifies specific gene mutations, proteins, chromosomal abnormalities and/or other molecular changes that are unique to your AML.

The results of these tests are used to determine if you have low-risk or high-risk AML to help guide prognosis and to evaluate the goals of treatment.

There are certain biomarkers—such as the FLT3, IDH1 and IDH2 mutations—that could indicate that your AML may respond well to a targeted therapy. There are several FDA-approved targeted therapies—known as inhibitor therapies—which treat patients with these mutations.

Additionally, the identification of other biomarkers—such as TP53, NPM1, or CEBPA mutations, to name a few—may aid in assessing your prognosis, determining a treatment course, or may identify if an allogeneic stem cell transplant may be appropriate. Results of these tests may also suggest that a clinical trial is your best treatment option.

So, how can you Insist on the best care for YOUR AML?

• First, always bring a friend or a loved one to your appointments to help you process information and to take notes.

• Ask your doctor if you have had, or will receive, biomarker testing and how the results may impact your care and treatment plan. Be sure to ask for paper or electronic copies of your important test results.

• Finally, always speak up and ask questions. It’s important that you understand all of the information that you want to know about your AML to help make the best treatment decisions for you. You are your own best advocate, and treating AML is a team approach.

To learn more about your AML and to access tools for self-advocacy, visit powerfulpatients.org/AML. 

How Is AML Treatment Effectiveness Monitored?

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How Is AML Treatment Effectiveness Monitored? from Patient Empowerment Network on Vimeo.

How can acute myeloid leukemia (AML) treatment effectiveness be monitored over time? Expert Dr. Ellen Ritchie explains when testing is typically done following AML treatment, which methods are used for monitoring, and when retesting may be appropriate.

Dr. Ellen K. Ritchie is assistant professor of medicine and a member of the Leukemia Program at the Weill Cornell Medical College of Cornell University and the New York Presbyterian Hospital. More about Dr. Ritchie, here.

See More From INSIST! AML

Related Resources:

Key AML Testing for Better Care: Understanding Prognosis and Treatment Choices

Transcript:

Katherine:

Once a patient has begun treatment, how do you monitor whether it’s working?

Dr. Ritchie:

So, one of the more frustrating things about being an AML patient, is you don’t know right off the bat whether or not that you have gone into remission. So, what happens is you receive the chemotherapy, and the day you start chemotherapy is really day one. And somewhere around day 14, you’re at your lowest point. So, your blood counts are low, and you often feel really terrible, and you really wonder, is this working? But unfortunately, I can’t really tell you. Some institutions do bone marrow biopsies if you have intensive chemotherapy on day 14, or if you’re getting venetoclax (Venclexta) therapy somewhere around day 21 to look and see whether they still see leukemia cells, but the utility of that is different per institution.

The real test of whether chemotherapy x, is at the end of about 28-35 days, are your blood counts coming up, and are you making normal blood cells. Are you making platelets, which are the part of the blood that clots the blood? Or are you making neutrophils, which are the important cells needed to help you fight infection. So, the real proof of a remission, is are your platelets over 100,000? Is your neutrophil count over 1,000? And when we look in the bone marrow around that time, do we see normal cells developing and no leukemia?

Katherine:

How often should testing take place? And should patients be retested over time?

Dr. Ritchie:

So, the bone marrow biopsy is done frequently once you have a diagnosis of acute leukemia. So certainly, it’s done upon diagnosis of the disease.

And as I mentioned earlier in certain institutions, about halfway through your chemotherapy cycle, they’ll do a bone marrow biopsy to see whether or not they see any residual leukemia cells. That’s not done everywhere, and it’s done differently depending upon institutions sometimes. At the end of the chemotherapy treatment, if you recover your blood counts, we do a bone marrow biopsy to confirm a remission. If by day 35, we haven’t seen that your blood counts are recovering, we may do a bone marrow biopsy to see whether or not we see leukemia cells in there, or early recovery. So, you’re definitely going to have bone marrows at those time points. If you’ve gone into remission, it depends on what we’d do next as to when you would have another bone marrow biopsy. So, if you’re going to bone marrow transplant you may have one more biopsy, just prior to going into transplant, and another biopsy at the end of the first month after transplant.

If you’re going to have what we call ongoing therapy, roughly every three or four months, we may do a bone marrow biopsy to determine whether or not the remission is holding. If during ongoing therapy, we see that there is blood count abnormalities that we weren’t expecting, that might be a reason that we would do a bone marrow biopsy. And that’s unpredictable as to when that would be.

Is the COVID-19 Vaccine Safe and Effective for AML Patients?

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Is the COVID-19 Vaccine Safe and Effective for AML Patients? from Patient Empowerment Network on Vimeo.

What should acute myeloid leukemia (AML) patients know about COVID-19 vaccines? Expert Dr. Ellen Ritchie shares information about COVID-19 vaccine safety and effectiveness for AML patients and reviews side effects that may follow vaccination.

Dr. Ellen K. Ritchie is assistant professor of medicine and a member of the Leukemia Program at the Weill Cornell Medical College of Cornell University and the New York Presbyterian Hospital. More about Dr. Ritchie, here.

See More From INSIST! AML

Related Resources:

Transcript:

Katherine:

The second question is from Craig, he says, “I’m currently undergoing treatment for AML. Is the COVID-19 vaccine safe and effective?”

Dr. Ritchie:

I recommend the COVID-19 vaccine to everyone, all my patients. A little immunity is better than none. And there is preliminary data, looking at patients with myeloid malignancies, not lymphoid, but myeloid malignancies, where it appears there is an immune response to the COVID-19 vaccine. So, I would suggest that you get the COVID-19 vaccine. Any of them that are available, are good. Whether it’s Moderna, or Pfizer, or Johnson & Johnson. Whatever is available to you, you should go ahead and get.

Katherine:

Are there any symptoms or issues that AML patients should be looking for post-vaccine?

Dr. Ritchie:

Post-vaccine, there’s a lot of symptoms that people have. And they can be similar among myeloid patients. Some of my patients have had no reaction whatsoever, some people have had a really sore arm.

Some patients are incredibly tired after the vaccine; some patients develop a low-grade fever for a couple of days. Those are really what we watch for. Sometimes when there’s a reaction, we’re hopeful that there’s an antibody being made, or an immune response that’s developing. So, it’s not always a bad thing if you have a reaction. But I don’t think that the reactions of patients of myeloid malignancies is any different than that of the general public.

How to Be a Partner in Your AML Care

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How to Be a Partner in Your AML Care from Patient Empowerment Network on Vimeo.

How can acute myeloid leukemia (AML) patients take a proactive approach to their care? Expert Dr. Ellen Ritchie shares advice for qualities to look for in your AML care provider and how to ensure all your questions are answered by your healthcare team.

Dr. Ellen K. Ritchie is assistant professor of medicine and a member of the Leukemia Program at the Weill Cornell Medical College of Cornell University and the New York Presbyterian Hospital. More about Dr. Ritchie, here.

See More From INSIST! AML

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Being Pro-Active in Your Care: Key AML Testing to Advocate For

Advocating for Key AML Testing: Advice From an Expert

 Advocating for Key AML Testing: Advice From an Expert

Transcript:

Katherine:

Dr. Ritchie, what advice do you have for patients to help them feel more confident in speaking up and advocating, being a partner in their care?

Dr. Ritchie:

Well, when you choose a leukemia doctor, you need to choose someone that you can actually communicate with. Someone who you feel is not allowing you to ask questions, or is not curious about what your life is like, you may want to think, I want to check out somebody else.

Because it’s really important you like the person who’s your doctor, and that you have a trust relationship together. So, it’s really – I tell some patients it’s a marriage of convenience that we have. And that you really have to think of it that way. If someone doesn’t allow you to ask questions or if they are not fully answering your questions in a way that you understand, try and speak up for yourself and make sure that the doctor tries to address that. And if the doctor won’t address those things for you, or you feel like you don’t understand what is being explained to you, then you can think about trying to see someone else. I think it’s really important if you can, to write down as many questions as you have about your disease before you come in.

Because often what happens is you get there, you’re stunned by the amount of information, and the questions you wanted to ask, you forget. And the next day, you’re like, “Ugh, I didn’t ask these questions.” So, before you come in, if you write questions. Questions about insurance coverage, that may not be something that we go over. Or questions about toxicities, or questions, “If I’m going to lose my hair, do you have the name of a wig facility?” All these questions that you might have, put them on a piece of paper, so that they can be addressed when you’re with the doctor. And other things will come up, you’ll have other questions when you’re there, but make sure your fundamental questions are answered.

What Is Low-Intensity AML Therapy?

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What Is Low-Intensity AML Therapy? from Patient Empowerment Network on Vimeo.

How does low-intensity AML therapy differ from high-intensity AML therapy? Expert Dr. Ellen Ritchie provides a comparison of the administration methods, side effects and reviews which AML patients low-intensity and high-intensity therapy are right for.

Dr. Ellen K. Ritchie is assistant professor of medicine and a member of the Leukemia Program at the Weill Cornell Medical College of Cornell University and the New York Presbyterian Hospital. More about Dr. Ritchie, here.

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Transcript:

Katherine:

You mentioned earlier, Dr. Ritchie, low-intensity therapy. Could you tell us about the types of treatment options?

Dr. Ritchie:

So, I’ll go – high-intensity therapy or intense chemotherapy always has to be given really in a hospital. And if you don’t start it – if you can start certain intensive chemotherapies, like daunorubicin and cytarabine (Vyxeos), which is also intensive, in the outpatient setting, but by day seven or eight, you end up in the hospital. And in intensive chemotherapies, you lose your hair, there’s GI toxicities, you’re at high risk of developing infections and you need a lot of transfusion. And for even young people, it’s a difficult therapy for which you’re in the hospital, and 90-some percent of patients are on IV antibiotics.

So, it’s intensive chemotherapy because it has to be given in a hospital setting and requires intensive supportive care. Low-intensity therapy can be given in the outpatient setting. So, at the present time you can get a drug like azacitidine (Vidaza), for example, which is an injection that you get seven days in a row.

Unfortunately, you have to come to the doctor’s office every day for those injections, but once you’ve had the injection, you can go home. Combined with venetoclax (Venclexta) which is an oral agent. So, an oral agent can be given at home.

You need close supervision in the physician’s office when you’re on this type of therapy, but you don’t need the constant support that you need if you are getting intensive chemotherapy. So, it can be done, in the comfort really of your home and with your family. You will have to come in and have transfusions potentially as an outpatient, nearly everyone does. And there’s always the risk that you develop a fever and if you do, you have to come into the hospital for IV antibiotics.

But in general, low-intensity means not so much support needed in a hospitalized setting, and the tolerability of this particular chemotherapy in the outpatient setting.

AML Targeted Therapies, What’s Available and How Do They Work?

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AML Targeted Therapies, What’s Available and How Do They Work? from Patient Empowerment Network on Vimeo.

There are several targeted therapies approved for the treatment of acute myeloid leukemia (AML). Expert Dr. Ellen Ritchie provides insight about recent approvals, how these therapies work, and shares details about newer therapies currently being studied for the treatment of AML.

Dr. Ellen K. Ritchie is assistant professor of medicine and a member of the Leukemia Program at the Weill Cornell Medical College of Cornell University and the New York Presbyterian Hospital. More about Dr. Ritchie, here.

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Transcript:

Katherine:

You touched upon this earlier, but what targeted therapies or treatments are available for AML patients?

Dr. Ritchie:

So, there have been many recent FDA approvals of drugs that are targeted. One, is the FLT3 inhibitors. And the two that are available are Midostaurin, which is most commonly – was the first drug that was really added to intensive chemotherapy.

And clinical trials show that in those FLT3-positive population that patients had an overall better outcome if midostaurin (Rydapt) were added to intensive chemotherapy. There’s also a drug called gilteritinib (Xospata), and this drug is also a FLT3 inhibitor that was tested in patients who had refractory leukemia. They could either get real chemotherapy regimen or they could get gilteritinib. And it turns out in the FLT3-positive patients, the gilteritinib was superior to the strong chemotherapy. So that’s been approved for patients who have refractory, or disease that didn’t really respond to initial therapy, that is IDH – or is FLT3-positive.

Then there’s the IDH1 and IDH2 inhibitors that have also been approved, and a small proportion of AML patients will be positive for IDH1 or IDH2 mutations.

The IDH1 inhibitor ivosidenib (Tibsovo), is available and can be used to treat patients if you know up front, they have an IDH1 inhibitor. So, that’s a regimen where the single agent can be used to treat an IDH1 mutated patient who’s newly diagnosed. Those patients are also eligible for many clinical trials now, where they’re combining that particular drug with other agents, in an effort to improve outcome. For IDH2 positive patients, there’s a drug called enasidenib (Idhifa). And this drug is used mainly in patients in the second line setting. But it specifically targets IDH2. And patients go into remission sometimes for a prolonged period of time. So, these drugs are FDA approved, and they’re treating targetable mutations.

TP53 mutations are a particularly bothersome mutation because it confers a poor outcome. And I’m happy to say that we have clinical trials now that are available that actually target TP53 mutations.

So, there are – there is therapy available for that type of mutation that was

not available before through the clinical trials. And I expect in coming years that we’re going to see more and more targeted therapies develop in AML which can be used potentially in combination with what we’re already using as backbones to enhance the outcome of patients with this disease.

Katherine:

Well, how do targeted therapies work?

Dr. Ritchie:

So, targeted therapies work on – it’s sort of complicated. The targets which are available, IDH or the FLT3 is really on the outside of the cell and it is a drug which is targeted directly to the FLT3 on the outside of the cell.

It works quite well in the peripheral blood, where you see the blast oftentimes disappear. The big concern always is how well it’s working getting deep into the marrow. But it’s looking at the target on the outside of the cell. IDH1 and IDH2 inhibitors work on particular chemicals which are involved in the kreb cycle, and those of you that took high school chemistry may have memories buried in the deep parts of your brain of learning the kreb cycle. And this is a fundamental metabolic cycle inside cells, and if you have a mutation, an IDH1 or IDH2, you’re unable to go through that full kreb cycle in the appropriate way. And that is something that leads to you having a cancer, in this case AML. So, these drugs actually interfere with what’s happening in that kreb cycle, and allow you to make more normal cells.

Factors to Consider When Choosing an AML Treatment

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Factors to Consider When Choosing an AML Treatment from Patient Empowerment Network on Vimeo.

What test results and factors should be considered when choosing an acute myeloid leukemia (AML) treatment? Expert Dr. Ellen Ritchie explains how test results impact AML prognosis and treatment – and other factors that come into play when determining a treatment approach.

Dr. Ellen K. Ritchie is assistant professor of medicine and a member of the Leukemia Program at the Weill Cornell Medical College of Cornell University and the New York Presbyterian Hospital. More about Dr. Ritchie, here.

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Transcript:

Katherine:

How do the results of these tests affect prognosis and treatment?

Dr. Ritchie:

Well, when a patient has AML, if they are a fit patient, if it will help us determine after initial induction, whether to cure the patient we need to do a bone marrow transplant, or we can just continue with chemotherapy.

And those are really important things to determine. So, if you have a good prognosis AML, if you have an AML that has certain translocations like inversion 16 or 821, or if you have a CEPBA mutation or you have an NPM1 mutation, and that’s all you have, you may do particularly well with chemotherapy treatment alone. And you won’t need to have a bone marrow transplant.

If you have certain other mutations, we know that the only way that we’re going to cure you, is with a bone marrow transplant. And if you are fit, when we finish induction and even as we’re doing induction, we’re preparing you for a bone marrow transplant down the line.

One disadvantage, just to mention about the molecular testing, is it doesn’t come back as quickly as some of the other testing. So that you will have already started induction chemotherapy most generally before the mutational testing comes back. Which can be anywhere – depending upon the institution, between seven and 21 days. So, it takes time for those results to be available.

Katherine:

Outside of test results, Dr. Ritchie, what other factors should be considered when choosing treatment?

Dr. Ritchie:

So, you want to choose whether a patient is most likely to benefit from intense induction chemotherapy. With strong chemotherapies where the backbone of those therapies would be an anthracycline, like daunorubicin (Cerubidine) or cytarabine (Liposomal), or daunorubicin or idarubicin (Idamycin PFS), together with cytarabine. And these are intensive chemotherapies. Versus, non-intensive chemotherapy which is able to be done as an outpatient, more frequently. And it is something that is gentler for a patient, they’re less likely to have severe toxicity. And the backbone of those regimens is using a drug called azacitidine (Vidaza) or decitabine (Inqovi), together with a second drug called venetoclax (Venclexta).

So, these are the two backbones, there may be clinical trials or there may be targetable aspects of your leukemia, which drugs would be added to either of those backbones. But those are the two backbones. And I also like to identify those patients that may not benefit from chemotherapy at all. And so, it’s very important, I think to really get to know your patient. And I spend time with my patient, particularly on the first visit, to understand not only their physical health, but their mental health. How good is their cognition, what is their mood, are they depressed, or are they happy people? And what is their circumstance? Do they have people to support them? Do they live close to family? Is a caregiver able to come, with an elderly patient for example, to visits?

Those, and whether or not they’re living alone and need tremendous support. So that’s really important to determine and helps me to choose what the best therapy might be. And also, concurrently what I can do to shore up the patient to do better with whatever therapy that I’m giving them. I.e., if you’re depressed, let’s work on that, or if your blood pressure is too high, or if you are – your diabetes is out of control at the same time that I’m seeing you, to try and fix those particular problems. In older patients I often do sort of a miniature version of the geriatric assessment. And in trials that have been so far, the most important aspects of the geriatric assessment, are really what is your cognitive function? Do have a mild dementia, or do you not have a mild dementia? Because dementia may be or mild dementia may be associated with poorer outcome.

The other is, are you able to do what we call the incidental tasks of daily life. So, you know fundamental tasks are really brushing your teeth and combing your hair, and dressing yourself. But are you able to do your cooking and your shopping and your banking and those things? Patients who have trouble doing their cooking and shopping and banking, and those types of activities, that also has been associated with a poor overall survival in AML. So, it’s really important to determine all of those aspects and if there are any deficiencies, to really know that the only therapeutic choice for that particular patient would be a low-intensity therapy.

Understanding Key Tests That Affect AML Treatment Choices

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Understanding Key Tests That Affect AML Treatment Choices from Patient Empowerment Network on Vimeo.

For acute myeloid leukemia (AML), test results play a vital role in determining the most appropriate treatment option. Expert Dr. Ellen Ritchie reviews key tests used to pinpoint a patient’s specific AML, how the test results are utilized, and important questions patients should ask their doctor about AML testing. 

Dr. Ellen K. Ritchie is assistant professor of medicine and a member of the Leukemia Program at the Weill Cornell Medical College of Cornell University and the New York Presbyterian Hospital. More about Dr. Ritchie, here.

See More From INSIST! AML

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 Essential Testing in AML: How Results Impact Care & Treatment Choices

Transcript:

Katherine:

So, let’s help our audience be clear about basic testing. What tests are necessary to help understand a patient’s specific disease at diagnosis?

Dr. Ritchie:

I mean certainly it’s important to do a physical exam and to find out what the general health of the patient is. In order to evaluate an AML, or any other leukemia, I look at the peripheral blood smear. To look at what I think the type of leukemia might be that I am dealing with. There are some leukemias that have particular way that they look like acute promyelocytic leukemia for which there is a designated therapy which works.

And you can tell that just by looking at a peripheral blood slide. The next test is always a bone marrow biopsy. Patients are not delighted that that is a test, but it is a test that can be done in the office, usually within 15 to 20 minutes. And that test gives us a lot of information. It gives us information about what type of AML it is, what are the markers on the outside of the cell, it gives us information about the chromosomes inside the leukemia cell. Are there missing chromosomes, or rearranged chromosomes? And if there are, that can be very relevant to the prognosis. And lastly, it’s sent for a particular mutations or markers. So, we look for IDH3 mutations, we look for FLT3 mutations, we look for IDH1 and IDH2 mutations, and we do an entire myeloid panel. Which is about 44/45 genes that are most commonly mutated in patients with AML. So that’s the initial work-up for any AML patient.

Katherine:

You mentioned markers, Dr. Ritchie. What is genomic, or biomarker testing?

Dr. Ritchie:

So, we’re looking really at most specifically at mutations inside individual genes that might be in your leukemia cell. So, there are some mutations actually that confer a better prognosis. Like NPM1 or CEPBA, those can be more positive type of prognosis than some of the others.

But we’re also looking for markers that might be targetable with certain therapies that we have. So, if you have a FLT3 ITD or TKD, we actually have particular drugs which can target those particular mutations. There are also drugs that are FDA approved to treat IDH1 and IDH2 mutations.

There are certain mutations that have a relatively poor prognosis, like TP53 for which there are clinical trials which are available, which specifically are meant to target patients who have those sorts of mutations. And there’re other clinical trials using the FDA-approved drugs that I just mentioned, for FLT3, for IDH1 and IDH2 and combining it with other agents to try and improve outcome in AML patients.

Katherine:

Some patients may not know if they’ve received these important tests, so what key questions should they be asking their physician about testing?

Dr. Ritchie:

So, physicians, they – financial coverage of the mutational testing is not uniform across the country and across insurances. So, Medicare and different Medicare insurances and some of the private insurances all vary in their coverage.

So, in my clinic, I am asking – I prefer the test that we do in house at Cornell. But it’s important that I ask, what will their insurance cover. And make sure that I send the appropriate testing that will be covered by insurance. There are some insurances that will not cover this type of testing. So, it is a real question for the patient, when you go to see the doctor to say, are you going to do mutational NGS testing?

And, will my insurance cover this? Hopefully most – if Medicare adopts the coverage of these types of mutational testing, it’s often true that private insurance will eventually pick this up. But it’s a murky field, and it’s really important to talk to your doctor about this. The cost of the bone marrow biopsy, and the chromosomal evaluation is nearly always covered by insurance.

Understanding Personalized Medicine for AML

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Understanding Personalized Medicine for AML from Patient Empowerment Network on Vimeo.

What should you know before deciding on treatment for YOUR AML? AML specialist Dr. Ellen Ritchie reviews key factors that guide treatment choices, including biomarker testing results, and shares advice for partnering with your team to advocate for the best care.

Dr. Ellen K. Ritchie is assistant professor of medicine and a member of the Leukemia Program at the Weill Cornell Medical College of Cornell University and the New York Presbyterian Hospital. More about Dr. Ritchie, here.

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Transcript:

Katherine:

As we begin to talk about personalized therapy and AML, let’s start with the basics. How would you define personalized medicine?

Dr. Ritchie:

Personalized medicine, to me is really, it’s a difficult question. It’s trying to find the best treatment for a particular patient. And it’s looking at biologic issues, what kind of cancer, what type of AML is it, what are the specific mutations or chromosomal abnormalities. But it’s also looking at the person. Is the patient active or not active? Do they have lots of other diseases like diabetes and coronary artery disease? Or pulmonary disease, or are they completely healthy?

Or, do they have support at home? If they’re sick at home is there someone who can take care of them, versus a situation where you’re older and alone and you have no real family member to rely on. So, all of these things are very important in making a personalized decision as to how you treat a patient.

Where Do Clinical Trials Fit Into an AML Treatment Plan?

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Where Do Clinical Trials Fit Into an AML Treatment Plan? from Patient Empowerment Network on Vimeo.

AML expert Dr. Eunice Wang discusses the role that clinical trials play in advancing research, the benefits of participation in research, and explains why she recommends trials for AML patients. 

Dr. Eunice Wang is the Chief of the Leukemia Service and Professor of Oncology at the Roswell Park Comprehensive Cancer Center in Buffalo, New York. Learn more about Dr. Wang, here.

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Transcript:

Katherine:

Where do clinical trials fit in when it comes to choosing treatment?

Dr. Wang:

Clinical trials are the mainstay of everything that we do in cancer care. Every single cancer drug that we’ve developed dating back into the 1970s at the National Institute of Health is the result of some patients and some doctors designing a clinical trial. These FLT3 inhibitors were developed over the last several years, so when I first came out of fellowship and started my training, we didn’t have these targeted therapies. Since 2017, in four years, we’ve had nine different drugs approved.

So, clinical trials are the way that we go from a finding in the laboratory to somebody having an extra birthday or going to their son or daughter’s wedding. That’s really how important it is, and those brave individuals who participate in clinical trials are helping not only themselves, but helping other people. I can’t tell you how many patients I enroll in clinical trials for AML, and I have told them – I said, “These nine drugs that we approved were because of nine different clinical trials which demonstrated benefit involving hundreds of thousands of patients.”

I can’t tell you how many times I’ve had a patient say to me, “Look, doctor, I’m going to participate in this clinical trial so that even if I’m not helped, you could learn something from me that could help the next person with their disease.” People are incredibly unselfish when it comes to clinical trials. I recommend a clinical trial for all my patients because I feel like that’s the cutting-edge clinical care.

I had patients here who I had on clinical trial drugs, and I was able to go to them and say, “Good news: Your drug has now been approved.” And, they say, “Doctor, why? I’ve been on this drug for a year.” And, I said, “That’s right, because you were part of that clinical trial, and you’re here now because of that drug, and now, a year or two later, that drug’s potency has been recognized, and now, the fact that you were in that trial has really helped us get this approval, which is going to help every other patient with that disease going down the line.” So, very important.

Shared Decision-Making, Advice for Partnering With Your AML Team

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Shared Decision-Making, Advice for Partnering With Your AML from Patient Empowerment Network on Vimeo.

AML expert Dr. Eunice Wang reviews how shared decision-making impacts overall care by keeping the individual patient and their unique circumstance in mind when determining a treatment path. Dr. Wang discusses the importance of reviewing clinical factors as well as having honest conversations, giving the patient a voice in their care. 

Dr. Eunice Wang is the Chief of the Leukemia Service and Professor of Oncology at the Roswell Park Comprehensive Cancer Center in Buffalo, New York. Learn more about Dr. Wang, here.

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Transcript:

Katherine:

We’ve been hearing a lot lately about shared decision-making. In your opinion, how is this concept best put into practice?

Dr. Wang:

So, shared decision-making is the process where the physician is no longer dictating the care, and telling patients, “This is the best therapy for you,” and just plowing forward with it. Shared decision-making is really what we want in all of our relationships in our lives, which is sitting down and incorporating many points of view, including both the patient’s wishes and desires as well as those that he or she feels is important to his or her care.

It involves time. It does – it may involve multiple clinic visits. It involves sitting back and having the physician say, “This is the information, this is the data. What is important to you? What is going to work with your particular home situation and family situation and dynamic?”, and then, together, coming up with a decision about care that is individualized for the patient. We talked about individualizing the targeted therapy for the biology of the disease.

Shared decision-making is individualizing the treatment decision for the individual patient and their particular circumstance, and that is best done by sitting down with the patient, looking them in the face, not by looking at your phone, or staring at that computer screen, or reading off some diagnosis from a piece of paper. It’s really involving having those honest conversations.

That’s how things used to always be in medicine, is that it always used to be a decision where the doctor and you would talk and come to a decision, potentially. We’ve kind of gotten away from that with all the electronics and technology, and I think the shared decision-making is a conscious effort by individuals and groups to bring that back in case. It’s very important for AML. AML is a disease that affects largely older individuals, so if you’re in your 60s and 70s and 80s, I can tell you right now that each one of those individuals who have lived decades of life have a certain way that they want to live whatever time they have left.

Katherine:

Of course. Well, when considering a treatment plan, what key questions should patients be asking?

Dr. Wang:

They should be asking – it should be – they should be asking, “How is this going to affect my daily life?” They should be asking questions – “Do I have to be in the hospital? How – do I need to come to the clinic? If I have to come to the clinic, how many times do I have to come to the clinic?”

In my part of the world, it – sometimes even the season in which they’re being diagnosed can impact what disease treatment they want because certain times of the year, travel back and forth in different weather conditions can be difficult. They need to be asking not the question of – that we get asked a lot like, “What would you do if this was your father or your mother?”, but I wouldn’t know.

I turn that around and I say, “But, you’re not my father and you’re not my mother, and if you were my father or my mother, I would ask my father or my mother, ‘What is going to work for you? What are your goals? Do you want aggressive therapy? Do you want to go for high risk/high benefit, or do you want something that’s just going to make you be able to be outpatient for longer, and really what is the most important thing for you and your family right now when we look ahead as to the treatment path?’”

Katherine:

Why is it important for patients to feel like they have a voice in their treatment decisions?

Dr. Wang:

It’s important for them to have a voice in their treatment decision because it is their – first of all, it’s their life, it’s their body. They are the ones that are going to be getting the therapy, suffering the consequences, and making the decisions that can impact not only them, but their loved ones, so – and, I find that the more they understand the disease process, the more they understand and can communicate to me their wishes, the more satisfied we are in care. I’ve had individuals tell me early on in the process where maybe, in a different patient, I would have suggested a second or third treatment – I’ve had them say to me, “I’m done. I’m not – thank you very much.” And, we all have to respect that.

It makes people more satisfied with their care. It makes people feel like they are making – they are guiding the path. They’re not just doing what their husband wants or what their doctor wants. I never want to have a patient say, “Well, I went and got chemo, Dr. Wang, because you wanted me to get chemo.” I don’t want you to get chemo, and I feel like if you have that understanding, I think patients are much more likely to pursue therapy and for the therapy, I think, to be successful or not. But, regardless of whether it’s successful medically, it needs to be successful emotionally for that patient and for that family.

AML Research and Emerging Treatment Options: An Expert’s Perspective

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AML Research and Emerging Treatment Options: An Expert’s Perspective from Patient Empowerment Network on Vimeo.

AML expert Dr. Eunice Wang shares exciting advances in the field of AML research, particularly in targeted therapies related to the TP53 and NPM1 mutations. 

Dr. Eunice Wang is the Chief of the Leukemia Service and Professor of Oncology at the Roswell Park Comprehensive Cancer Center in Buffalo, New York. Learn more about Dr. Wang, here.

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Transcript:

Katherine:

What specifically are you excited about in terms of AML research and emerging treatment options?

Dr. Wang:

I am really excited about the advent of newer targeted therapies. Right now, we only have targeted therapies for probably about three mutations out of the many, many mutations that we know exist in AML. So, we know that there certainly are patients that have specific mutations, such as TP53 mutations, or patients who have very complicated series of DNA damage, that just don’t do well with any of our therapies.

I’m looking forward to another bunch of targeted therapies – these inhibitors called menin inhibitors – that might be useful for treating patients that have mutations in NPM1 gene or other chromosome abnormalities.

I’m also really looking forward to us being able to finally unleash the power of the immune system for treatment of AML with a few novel agents coming down the pike which have, for the first time, started to show that immune modulation can work in AML patients.

What Key Tests Do You Need Before Choosing an AML Treatment?

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What Key Tests Do You Need Before Choosing an AML Treatment? from Patient Empowerment Network on Vimeo.

How do test results influence treatment choices for AML? Dr. Eunice Wang shares information about essential testing and explains how results aid in determining the best personalized treatment option for each patient.

Dr. Eunice Wang is the Chief of the Leukemia Service and Professor of Oncology at the Roswell Park Comprehensive Cancer Center in Buffalo, New York. Learn more about Dr. Wang, here.

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Transcript:

Katherine:

What is the role of testing when deciding on treatment for AML?

Dr. Wang:

Testing is essential in us selecting and determining the best personalized treatment option for each individual patient. As you know, AML is an aggressive hematologic malignancy and can be devastating, both in its life-threatening nature and in its rapidity and the need for a rapid diagnosis. Testing, including both pathology results as well as protein marker testing, and, importantly in this day and age, DNA and RNA testing is essential because we have numerous different treatment options that could be available to the patient if their particular disease biology matches with the targeted therapies that we have.

So, as you may or may not know, since 2017, we’ve had eight or nine different therapies approved for AML, and this is a bonanza of options, some of which are only for specific biological subsets, and some even for specific patients, such as those above the age of 75. So, doing that testing, particularly that genetic testing, is important both in establishing the diagnosis and determining whether there is less toxic, more targeted, personalized treatment approaches, some of which involve low-dose chemo or even pills available to the individual patient.

Katherine:

You’ve answered this, in part, but which tests are essential following an AML diagnosis?

Dr. Wang:

I think all of them are essential, but in this day and age, for the selection of targeted therapy, it really is the mutational testing, which is looking at the RNA of the tumor cells and determining whether that has been altered in allowing the cells to express abnormal proteins. For standard chemotherapy, we also use DNA testing, which is looking at the different chromosomes and seeing whether there’s breakages or what we call translocations, pieces of chromosomes that have been swapped. That DNA chromosome information can give us some insight into prognosis and therapy response.

So, nowadays, it’s not just determining that you have acute leukemia, but looking at the specific DNA and RNA changes, and I have to say that this is a disease that we’re really not seeing any RNA or mutational changes occurring in more than 20 percent or 30 percent of patients. So all of the mutations that we see that could be impactful really don’t occur in more than 20 percent or 30 percent, and could only occur in five or one percent.

So, really, personalizing an individual patient’s disease, both for the disease biology as well as the person that’s getting the chemotherapy or the diagnosis, is really, really important.

Katherine:

Yeah. Let’s define a few terms that are often confusing for patients. What are biomarkers?

Dr. Wang:

Biomarkers are either proteins or expression levels on the cancer cells that can serve to tell us information about the biology of the disease. Okay, so, for example, if you have evidence of residual tumor proteins in your blood, that could be a marker, for example, of minimal residual disease, okay? And, that can tell you maybe one in a million cells have that biomarker, and then you can tell that those one-in-a-million cells are leukemia cells.

So, they’re any marker that we’re using that’s specific for the tumor that can help us in predicting or finding or locating or determining if a tumor would respond to a certain therapy.

Katherine:

What is biomarker testing?

Dr. Wang:

Biomarker testing can be done in many ways. For example, biomarker testing is drawing a sample from the patient and evaluating a marker that we think is going to predict for the disease type.

So, for example, in some cancers, we don’t want to biopsy the lung mass or the tumor mass every single time to see whether it’s shrinking, or getting smaller, or responding. So, in those patients, sometimes we’ll draw a blood sample, and we’ll look for a surrogate marker – some protein that’s expressed in the blood or some DNA or RNA in the blood that is a surrogate or a marker of the tumor so you don’t have to directly biopsy it.

In acute myeloid leukemia, we are looking for – like I said – particular cells in the blood that have particular proteins, and we measure those rather than going ahead and doing that bone marrow biopsy or biopsying those tumors. So, generally, in leukemia, it involves drawing blood samples – that’s the most common; it is a bloodborne disease.

Sometimes, we actually have to go into the bone marrow and do a bone marrow sample, but those biomarkers, as I said, can really improve our ability to detect very, very low levels of disease. So, for example, using a conventional bone marrow biopsy, we can only really detect 1 out of 200 cancer cells by normal – just by visual looking at, but by measuring biomarkers and mutations and other abnormal proteins, we can improve that to 1 in 100,000 cells.

So, really, these biomarkers are very sensitive and important because we want to detect the disease at a point where it’s very, very low. We don’t want to wait until the disease gets very advanced, in which case we think our therapies are less effective.

Katherine:

What is a genetic mutation?

Dr. Wang:

A genetic mutation is a mutation that occurs in the RNA of a cancer cell. That RNA dam – RNA aberration or abnormality does lead to different RNA – what we call transcript levels that lead to abnormal proteins.

Those proteins function in the cells to make a cell a cancer cell, okay? So, all cancer cells start out as normal cells, and as they acquire a mutation, they become a little less normal, and they start acquiring multiple mutations, and some of these mutations occur without DNA changes, some of them occur with DNA changes. And as these abnormalities occur, the cell gets more and more dysfunctional, and eventually, it starts becoming almost evil-ish.

It starts acquiring behaviors that are not normal, and then it starts to grow out of control, and that unchecked growth really is the end result of potentially many mutations occurring over time to drive that cell into becoming a cancer cell, and we call that process transformation, transforming from a normal, healthy-looking cell into almost a monstrous, cancer-like cell.

Katherine:

How do biomarkers affect AML treatment choices?

Dr. Wang:

So, those biomarkers, as I talked about, those mutations can determine what type of therapy patients can have. For example, up to 25 percent or 37 percent of newly diagnosed AML patients will have leukemia cells that carry the biomarker or the mutation in a gene called FLT3, or “flit.”

Those FLT3 cells can be inhibited by specific targeted therapies, including a drug called gilteritinib (Xospata), which is a pill which blocks mutant FLT3 expressed by AML cells. So, we’ve demonstrated, actually, in a randomized clinical trial that patients who have relapsed or recurrent AML who carry cells that have that biomarker – that FLT3 mutation – will actually do better if they take a daily pill – a FLT3 inhibitor – every single day for treatment of their aggressive acute myeloid leukemia than if we gave them low- or even high-dose chemotherapy in the hospital for four to six weeks.

So, that’s the power of those targeted therapies. Because the biomarker is telling you that there’s a sensitivity of that cancer cell to a specific blockage of that pathway, that can really dramatically change the course.

That is where the importance and the power of those biomarkers really goes into play. In the past, patients who had acute myeloid leukemia with FLT3 mutations did poorly with chemotherapy and had disease that came back even after multiple rounds of that intensive chemotherapy. The fact that we can give a pill and people could do better or even go to a bone marrow transplant off treatment with the pill is pretty remarkable.