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Expert Advice for AML Patients When Making Treatment Choices

Expert Advice for AML Patients When Making Treatment Choices from Patient Empowerment Network on Vimeo.

What are key factors to consider for acute myeloid leukemia (AML) patients when making treatment decisions? Dr. David Sallman reviews important considerations and their impact on treatment choices, and shares questions patients should ask their doctor to receive optimal care. 

Dr. David Sallman is an Assistant Member in the Department of Malignant Hematology at Moffitt Cancer Center where he specializes in myelodysplastic syndromes (MDS), acute myeloid leukemia (AML) and myeloproliferative neoplasms (MPN). Learn more about Dr. Sallman, here.

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How Molecular Testing Has Transformed AML Treatment Options


Transcript:

Katherine:

When making a treatment choice, what are three key considerations for AML patients?

Dr. Sallman:

Yeah, so I think the initial probably two main questions are is the patient fit or non-fit, and that’s really an evolving definition. I think historically, we had this magical age if you’re less than 60 or less than 65 years of age, but we’ve really gone past that significantly. So, does a patient have significant medical problems, decreased performance status that we would not think about intensive therapy is one of the main questions. I think what feeds into that. And the other big question is what is the underlying mutations that the patient has which really gives us a prognostic risk from a disease perspective.

With certain mutations and subgroups being much more sensitive to intensive chemotherapy and other groups really where that option is poor irrespective of age. So, I think the most important thing is how does the patient look, what is their fitness level, and what are the underlying cytogenetic and molecular changes that impact their disease.

I think third, of course, is really involving the patient in their preferences, because I think some of these can really be a decision between several options.

Katherine:

What’s the role of the patient in making treatment decisions?

Dr. Sallman:

Yeah, the patient has to be central. I’m really hoping that we’ve moved a long way from the paternalistic practices in the past.

I think there are still many instances where there’s sort of a clear best option from a medical perspective, but there’s a lot of social logistics. If you’re getting intensive therapy, as an example, you’re going to be in the hospital four to five weeks, what’s your support system? What financial, other impact factors, all of these things come into play. I think it’s a tough group. I think the patients that are, let’s say, 60 to 70, because responses are somewhat similar across non-intensive and intensive options, I think there’s the question of is the goal long-term, is the goal quality of life, and I think all of those really are impactful.

I think it can be very challenging to go through all of the specific numbers and how a patient comprehends that or not, but really trying to draw out is their goal long-term, is their goal quality of life, give them the pros and cons of the potential options in that setting, and then real-time discuss that as we go. I think when they have that buy-in from their goals, it’s important.

These are complicated regimens and patient compliance and follow-up and all that are really critical to the overall safety and good outcomes of these patients.

Katherine:

Are there questions that patients should ask in their proposed treatment plan?

Dr. Sallman:

Yeah. I think it’s always important to discuss what options. I think any time there’s a one-option, if there is a one-option, why? Maybe because standard of care in this group is so good that it’s not really reasonable to necessarily offer a main alternative regimen. I think it’s important to understand as much of the disease as possible. If you’re choosing this regimen, why are you doing it? I think asking about the mutations is important, although that’s a very complicated thing to explain. Some patients like it and some patients don’t, and I think you have to do that in your team-based relationship.

I think always asking about clinical trials is an important question to ask. Should they be getting a second opinion? These are overall very rare diseases, and we highly favor an initial consultation at an academic center that specializes in this. I’d say a majority of my patients are ultimately treated in the community. But especially given that the regimens are becoming much more complicated, the intensity of watching their counts, managing side effects, titrating medications, it’s really great to have a team-based model between academic and community centers and that can’t really ever happen if they never come to us. As much as possible for that to occur I think is important as well.

How Molecular Testing Has Transformed AML Treatment Options

How Molecular Testing Has Transformed AML Treatment Options from Patient Empowerment Network on Vimeo.

How has molecular testing impacted approaches to acute myeloid leukemia (AML) therapy? Dr. David Sallman explains how molecular testing has transformed AML care, including a discussion of risk assessment and the role of next-generation sequencing (NGS) in tailoring care for each patient. 

Dr. David Sallman is an Assistant Member in the Department of Malignant Hematology at Moffitt Cancer Center where he specializes in myelodysplastic syndromes (MDS), acute myeloid leukemia (AML) and myeloproliferative neoplasms (MPN). Learn more about Dr. Sallman, here.

See More From INSIST! AML


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Understanding AML Induction and Consolidation Therapy

Transcript:

Katherine:

How has molecular testing changed the landscape of therapy for AML?

Dr. Sallman:

Yeah, it’s really transformed it, and it’s really a constantly evolving paradigm. We have updated classifications; most people utilizing the ELN system.

So, based on both cytogenetic and molecular factors, you can ultimately go into good risk, intermediate risk, adverse risk. In general, for fit patients for good risk, we focus on curative intent, ideally with chemotherapy alone. For intermediate and adverse, typically we’re incorporating allogeneic stem cell transplant. So, that’s one of the main things that really guides treatment really from the beginning and throughout.

Then, I think really where it’s evolving is personalized therapy. So, it’s really not a one-size-fits-all treatment paradigm, it’s you have mutation A, B, you’re this age, this fitness, and we put all those things together to ideally come up with the best treatment plan for the patient.

Katherine:

Is molecular testing standard following an AML diagnosis or is this something that patients should ask for?

Dr. Sallman:

It definitely should be standard and I think the challenge is when you say the word “molecular,” it means lots of things to different people. I think in the community, as targeted medications were first approved, so this was with FLT3 inhibitors, subsequently IDH1 and IDH2 inhibitors, I think people are realizing yes, we have to send these sequencing panels, but there’s a potpourri of choices from a lot of different commercial vendors.

Really the key and one of the main messages we try to get across is you really have to assess for both FLT3 as well as really a comprehensive next-gen sequencing panel in order to cover all of the relevant genes at diagnosis and likely at other time points such as relapsed or refractory disease.

So, there’s no question, it’s standard, although unfortunately, it’s still not uncommon where the comprehensive panels are not sent and you’re left with somewhat not a complete picture for your patients. Since we’re personalizing everything, it’s really quite critical to have these data.

Katherine:

Yeah. How does inhibitor therapy work to treat AML?

Dr. Sallman:

So, you have a gene that turns on and turns off as we go, but with the mutation, it’s basically turned on all the time. Then, you can have targeted pills that basically turn it off. Most commonly this is done, there’s the active

or energy site for these different genes, and so these therapies can really specifically block that. I wouldn’t say that’s the only mechanism. There are IDH1 and IDH2 inhibitors and they’re very specific for those mutations. Each mutation may have a little bit different end biology. In general, you have mutation A, and we’re going to turn it off with drug that inhibits A.

Treatment Advances for Aging AML Patients

Treatment Advances for Aging AML Patients from Patient Empowerment Network on Vimeo.

What are the latest acute myeloid leukemia (AML) treatment advances for elderly patients? Dr. David Sallman shares details about new therapies that he’s excited about and their impact on care for all AML patient groups.

Dr. David Sallman is an Assistant Member in the Department of Malignant Hematology at Moffitt Cancer Center where he specializes in myelodysplastic syndromes (MDS), acute myeloid leukemia (AML) and myeloproliferative neoplasms (MPN). Learn more about Dr. Sallman, here.

See More From Engage AML

Related Resources:

How Molecular Testing Has Transformed AML Treatment Options


Transcript:

Katherine:

Okay. When it comes to AML research and emerging treatment options, what specifically are you excited about?

Dr. Sallman:

Yeah. So, I think probably the most exciting changes have really been in the overall elderly AML setting, although I think are really broadly impactful across patients.

So, the standard has been hypomethylating agents for a long time. This paradigm has recently changed with the FDA approval and now full approval of venetoclax in combination with hypomethylating agents, but we’re still talking about immediate overall survival of 14 months in the Phase III setting.

There are lots of exciting drugs, and I think this is really where the spectrum of myelodysplastic syndrome and acute myeloid leukemia comes into play.

So, I really think in elderly AML, we’re moving towards more triplet type combinations to really ideally move the field forward. That adds levels of complexity, toxicity from additional therapies, but we’re really hoping to truly move that survival curve even more.

There’s a lot of HMA, doublet, triplet combinations that are exciting and I think that’s really where the field is going.

I think at the same time in the failure setting, particularly, let’s say, in the HMA venetoclax failure setting, there’s really a lack of almost any effective therapies. We’re really hoping that novel cellular and immunotherapies will hold significant promise in this group. There are numerous trials that are being considered in this space, but I’m hopeful for it.

What AML Patients Should Know About the COVID-19 Vaccines

What AML Patients Should Know About the COVID-19 Vaccines from Patient Empowerment Network on Vimeo.

What are some key points for acute myeloid leukemia (AML) patients to understand about the COVID-19 vaccines? Dr. David Sallman shares advice for patients who are considering the COVID-19 vaccine.

Dr. David Sallman is an Assistant Member in the Department of Malignant Hematology at Moffitt Cancer Center where he specializes in myelodysplastic syndromes (MDS), acute myeloid leukemia (AML) and myeloproliferative neoplasms (MPN). Learn more about Dr. Sallman, here.

See More From Engage AML


Related Resources:

 

Understanding AML Induction and Consolidation Therapy

 


Transcript:

Katherine:

Are the COVID-19 vaccines safe for AML patients, and how does the vaccine affect treatment, if at all?

Dr. Sallman:

Yeah, I think that’s a great question. I think it’s really a rapidly evolving day-by-day update. For example, at our center, we vaccinated a high number of patients and we’re actually in a study trying to understand what their antibody production. So, I think the question is less ‘is it safe or not safe,’ but more is it as effective or worthwhile based on patients that have low blood counts.

I think, in general, if a patient is in remission, either post-therapy or on maintenance-type therapy that has a relatively preserved white count and is it’s very reasonable to utilize it, I think we still have the caveat of is it as effective, of course we don’t know that clearly since all the large trials, these patients weren’t really included. But in general, if you’re not severely leukopenic, we are vaccinating a high percentage of patients that we’re monitoring closely, but anecdotally, we’ve not had significant different adverse events from our perspective.

AML Research Updates: News from ASH 2020

AML Research Updates: News from ASH 2020 from Patient Empowerment Network on Vimeo.

AML expert Dr. Jeffrey Lancet shares the latest news from the 2020 American Society of Hematology (ASH) annual meeting. Dr. Lancet sheds light on headlines from the meeting including FLT3 inhibitor research, combination therapies with venetoclax, a promising inhibitor therapy, and shares his optimism about the future of AML treatment.

Dr. Jeffrey Lancet is Chair and Program Lead in the Department of Malignant Hematology at Moffitt Cancer Center in Tampa, FL. He is nationally and internationally recognized for his clinical research in the field of acute leukemias. Learn more about Dr. Lancet, here.

Download Program Resource Guide

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New AML Therapies vs. Traditional Chemotherapy: What’s the Difference?

Transcript:

Katherine:      

Hello, and welcome. I’m Katherine Banwell. Today we’ll discuss the latest news from ASH 2020 and how AML patients can advocate for personalized care. Joining me is Dr. Jeffrey Lancet. Welcome. Would you please introduce yourself?

Dr. Lancet:                   

Hi, sure. My name is Dr. Jeff Lancet. I’m at the Moffitt Cancer Center in Tampa, Florida, where I am the Chair of the Malignant Hematology Department. We spend a lot of time treating patients and conducting clinical trials of Acute Myelogenous Leukemia.

Katherine:                   

Okay. Thank you. Dr. Lancet, the American Society of Hematology Annual Meeting just closed. What are the AML headlines from this year’s meeting?

Dr. Lancet:                   

Yeah, so as usual, AML was a very busy area for clinical presentations this year at the ASH meeting focusing largely on novel and targeted therapies.

I don’t believe that there were many practice changing developments per se, but rather discussions about many promising therapeutic strategies that are still under development and moving forward rapidly largely in the areas of targeted therapy, low intensity therapy, measurable residual disease and things of that nature.

Katherine:                   

What does this research news mean for patients?

Dr. Lancet:                   

Well, I think that there’s a lot to be encouraged about and maybe I’ll take the time to review some of the highlights in what was presented with respect to some of the novel therapeutic approaches that many of our patients can look forward to receiving in the not too distant future.

So, we often talk about you know, targeted therapies and, of course, one of the major targets over the years has been that of mutated FLT3 which is one of the most common mutations in AML.

And at this meeting we saw several presentations on clinical trials resolved to utilizing inhibitors of FLT3, with some emphasis on the most recently approved second generation drug called gilteritinib.

There were I thought three major presentations focusing on gilteritinib and one was an update on a randomized Phase III trial comparing gilteritinib plus azacitidine versus azacitidine alone in newly diagnosed unfit for induction chemotherapy patients with FLT3 mutations, preliminarily showing good tolerability and high composite complete response rates in the combination on.

There was another trial of gilteritinib plus venetoclax in relapsed and refractory FLT3 mutated AML.

And what was interesting was that a very high percentage of patients achieved response with this combination of gilteritinib plus venetoclax, many of whom were heavily pretreated previously and many of whom had also gotten prior FLT3 inhibitor therapy during an early stage of the disease. So, the combination of gilteritinib and venetoclax and this more refractive study, it was encouraging to see these promising responses.

And then we saw some data reporting the effects of gilteritinib in combination with more traditional chemotherapy induction with a couple of studies demonstrating both a high complete response rates as well as high rates of mutation clearance of the FLT3 mutation.

So, those were very encouraging data that were presented with respect to the FLT3 mutated AML population.      

So, another very important drug that reached the marketplace for AML recently is a drug called venetoclax, which is an inhibitor of a protein called BCL2.

And this drug was recently FDA approved for use in combination with low intensity chemotherapy drugs such as azacitidine or decitabine.

And it seems as though the combination of venetoclax plus one of these hypomethylating agent drugs, azacitidine or decitabine has resulted in very, very strong efficacy signals as recently published in a New England Journal of Medicine paper that reported on the results of the Phase III trial of venetoclax plus azacitidine.

So, that has now become standard of care for older less fit adults with newly diagnosed AML; the combination of venetoclax plus a hypomethylating agent such as azacitidine.

And naturally, there’s been interest in really kind of taking it several steps further to advance the role of these combinations and to also look at additional drugs in combination with venetoclax plus hypomethylating agent therapy.

So, we saw some of that at the ASH meeting this year. One approach would be to take venetoclax and then to combine it with more intensive chemotherapy for perhaps more fit patients or younger patients that could undergo a more intensive program.

So, we saw presentations of venetoclax being combined with a drug called CPX-351, which is a novel liposome formulation of two common chemotherapy drugs that had been approved a few years ago for secondary AML. And we also saw a combination strategy with venetoclax, and a regimen known as FLAG-IDA, which is a commonly used induction regimen in acute myeloid leukemia.

And I think it’s important to recognize that although these trials that combine the venetoclax with more intensive chemotherapy showed signs of good efficacy with good response rates, there were definitely signals of increased toxicity, hematologic toxicity primarily, which is not completely unexpected with venetoclax knowing that it can cause significant lowering of white blood cells and platelets and hemoglobin.

And then finally, there is a lot of interest in, you know, doing these types of combinations with venetoclax in different subsets of AML and one subset of AML that has been very important recently is that of the IDH mutated AML population of patients.

IDH is a fairly common mutation that occurs either in the Isoform of IDH1 or IDH2 and there’s about a 15 to 20 percent incidence of IDH mutations in AML.

Now we do have an inhibitor for both of these types of mutations: ivosidenib for IDH1 and enasidenib for IDH2, but there also appears to be a strong role for venetoclax plus azacitidine in IDH mutated AML.

We saw from a series of patients presented by a physician at MD Anderson looking at outcomes with venetoclax plus azacitidine in IDH mutated AML. And the response rates were very high when you give HMA plus venetoclax to these patients with IDH mutated AML.

But I think more importantly was that there were what we call high intro patient response rates when switching between venetoclax and HMA therapy with an IDH inhibitor containing regimen.

In other words, a patient would have a good chance of responding to the initial therapy and then if or when that therapy stops working, having a good effect from a salvage therapy with the other regimen. So, when you see initially azacitidine plus venetoclax and then had a relapse, the IDH inhibitors worked well and vice versa if you had received an IDH inhibitor and then subsequently received HMA-venetoclax at a later time point that also worked well.

So, it’s encouraging to see that you can potentially sequence these drugs and get continued responses along the way and ultimately we think will help a survivor and keep patients in a better state of health even longer.               

So, I just wanted to take a few minutes also and discuss some of the newer more novel therapies that are really hitting or approaching the landscape right now. One of these is called CC486, also known as oral azacitidine or ONUREG. And this drug was shown in recent literature to prolong overall survival in patients who are in first remission from their AML who had received induction chemotherapy.

So, this drug was used as maintenance therapy after a variable number of consolidation regimens. And people who got this ONUREG or oral azacitidine drug as maintenance therapy, it resulted in longer survival compared to those who had received placebo.

And this was presented at last year’s ASH meeting, but this year’s ASH meeting provided an update, a very important update, showing that the overall survival advantage from this drug, this oral azacitidine drug, when used as maintenance was independent of whether a patient had measurable residual disease at the time that they went onto the maintenance therapy.

In other words, whether you had MRD, measurable residual disease or not at the time of the study entry, your responses were still more favorable, your outcomes were more favorable, if you received this oral azacitidine drug.

So, this was FDA approved earlier this year for patients in the maintenance phase of therapy for AML who had gotten prior reduction chemotherapy.

And importantly, this drug was also shown to be able to convert about 25% of patients who were positive for measurable residual disease; convert them from positive to negative. So, even though they were in remission, they had measurable residual disease and this drug in about 25 percent of the cases converted that from positive to negative. So, that’s a very important finding as well.

Another important drug that I think you should keep your eye on is a drug called magrolimab. This is an antibody against a certain type of protein that is present on the immune system cell called the macrophage, and when this magrolimab drug was combined with azacitidine in a recent clinical trial, it was demonstrated very high response rates of over 65 percent.

And, in particular, in patients with P53 mutations, which is a very bad mutation to have in most cancers, including AML, in patients with this high-risk mutation, the combination of magrolimab with azacitidine appears to be effective based upon the early data that we have with high response rates.

And then finally, I just wanted to make mention of another important area in, not really just AML, but in all cancer and that’s  outcomes disparities between different races and ethnic groups. And we saw a very important presentation at the plenary session this year where the authors reported outcomes amongst younger patients with AML who were African American compared with Caucasian.

And the data clearly indicated a worse overall survival amongst Black patients compared with white patients under age 60. And this included patients who were enrolled in clinical trials. So, that it appeared that African American patients have a worse outcome than Caucasian patients with acute myeloid leukemia highlighting the need to better understand various risk factors and other factors that play into these disparate outcomes between our Black American population and a white American population, which I think could shed light on additional disease characteristics that may help everybody as well.

 

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

Essential Testing in AML: How Results Impact Care & Treatment Choices from Patient Empowerment Network on Vimeo.

What tests should follow an AML diagnosis and why? Dr. Hetty Carraway, an AML specialist of Cleveland Clinic, reviews the essential testing for patients with AML and explains how those test results may inform treatment decisions.

Dr. Hetty Carraway is Director of the Leukemia Program at Cleveland Clinic. Dr. Carraway cares for patients with acute leukemia and bone marrow failure states. Learn more about Dr. Carraway, here.

Download Program Resource Guide

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What Is the Patient’s Role in Making AML Treatment Decisions?

Navigating AML Treatment Decisions

Insist! AML Resource Guide

Transcript:

Katherine:      

Hello, and welcome. I’m Katherine Banwell. Today, we’ll discuss how you can be proactive by insisting on better AML care and personalized treatment options. Joining me is Dr. Hetty Carraway.

Welcome, Dr. Carraway. Would you please introduce yourself?

Dr. Carraway:            

Hi. My name is Dr. Hetty Carraway. I’m one of the physicians at the Cleveland Clinic. I work as the Director of the Leukemia Program, and I spend most of my time caring for patients with acute leukemia and bone marrow failure states.

Katherine:                  

Thank you.  Let’s start with the basics. What essential testing should AML patients undergo following a diagnosis?

Dr. Carraway:            

This is a pretty standard workup for patients that have this diagnosis of acute leukemia.

For most of our patients we always evaluate with a peripheral blood count including a complete blood count with differential, typically a comprehensive metabolic panel, and looking at a test called a uric acid, which looks at the cell turnover and the cellular debris in terms of the burden on the kidney. We often will get a bone marrow biopsy with aspirate for patients, and in the diagnosis of leukemia typically that’s already been done.

There are tests that are sent off of that aspirate called a test for chromosomes, whether it’s comprehensive cytogenetics or FISH, for fluorescence in situ hybridization. We’re often testing using a study called NGS or next generation sequencing looking for specific mutations of genes known to be important in the pathogenesis of leukemia.

Furthermore, we often get a test called flow cytometry from that aspirate looking at the markers on top of the leukemia cells that help us to identify the blast population. So, I would say those by and large are the tests in the bone marrow biopsy that we get, which are innumerable and detailed.

They often take some time to get back, so at the time of the diagnosis patients know that they have a diagnosis of leukemia, but those additional chromosome tests or mutation testing that can take up to two weeks if not longer to get back. And so, it’s important to follow up on that information later on and say, has that testing come back? If so, how does that change any of what the decisions are moving forward?

Katherine:                  

Genetic testing can often be confused with molecular testing. What’s the difference between the two, and why should patients undergo the testing?

Dr. Carraway:            

The chromosome testing and the mutational testing help us to really classify the risk in terms of the leukemia itself, whether or not that leukemia is responsive to chemotherapy alone, or if it means that there’s a higher likelihood of that leukemia not being controlled with leukemia only.

In that setting, we often then move towards transplant for curative intent in addition to the chemotherapy. The reasons to get the information is to really help us better tailor the therapy for each individual patient. That information really does help us guide not only the upfront therapy for some patients but even the long-term therapy. It can be incredibly overwhelming to have too much information at the get-go, so in some senses it’s better to have these pieces as they unfold over time.

For other patients, they want to know what exactly the plan is going to be A to Z from day one. That is of course more challenging now that it just takes time to get this information. I think what they need to know is that we’re working hard to get that information.

As soon as we get it, we don’t hold back. We reveal and share that information and come together to say, this is what this data or information means, and these are some of the choices that we either recommend that you consider, and these are the risks and benefits to those considerations.

Katherine:                  

Let’s look at something that is similar to what you’ve just been talking about. How do test results impact treatment and overall care?

Dr. Carraway:            

They really can. When you asked me how come chromosome or genetic information is different than mutational information, the chromosomes can help us to figure out where patients land in terms of prognosis. That information is different than the mutational testing. Both of those pieces can help us figure that out.

The mutational test, I will tell you, does help us figure out are there targets on the leukemia that allow us to use therapy that’s directed to that mutation. The key example I’ll give is a mutation in a gene called FLT3. That particular mutation has an agent now that is F.D.A. approved called Midostaurin, and so once we know that a leukemia harbors a FLT3 mutation we often add a drug called Midostaurin to the backbone therapy that is used for patients.

Now, that’s important, and now there are more and more genes that when mutated we have novel therapies that direct against that specific tag that’s on the leukemia and helps to improve eradication of the disease or control of the disease if you will.

That’s different than the genetic information when we’re looking at chromosomal changes that may allow us to say in the rare instances of  favorable cytogenetics like a translocation of chromosome 15 and 17 consistent with APL, the treatment for that type of leukemia,  acute promyelocytic leukemia, is very different than what we do for the majority of other leukemias.  

The prognosis for that leukemia is also very different. It helps to tailor the regimens, and it helps to select specific therapy that may be helpful to each individual patient.

Katherine:                  

Dr. Carraway, you just mentioned FLT3. Would you tell us about the common mutations in AML and how these may impact treatment options?

Dr. Carraway:            

There’s a multitude of mutations that we’re now following in patients. The way that we follow them is by doing this next generation sequencing test at the upfront time at diagnosis.

The reason why we’re doing that is because those mutations can regress with therapy, or they can progress where you gain additional mutations that happen as the disease progresses. Even if it’s responding to therapy or as it loses response to therapy and reemerges, it may reemerge with different mutations. As a result of that, it may change what therapy we select. Our ability at this point in being to recommend exactly at what time points we are checking the next generation sequencing we’re still learning right now as to what are the key times to do that testing.

In general, most institutions are doing that next generation sequencing at the time of diagnosis, and then also for some patients before they go to bone marrow transplant and even after bone marrow transplant.

For some of those patients that unfortunately relapse, we’re also making sure to retest the next generation sequencing mutation testing to see are there new mutations that have come about that weren’t there before?

Katherine:                 

I understand there’s something called IDH. 

Dr. Carraway:            

You were also asking about what other mutations besides FLT3 happen in patients with AML. FLT3 is one such mutation. NPM1 is another mutation that often it frequents patients that have AML. Those two mutations happen in about 30 percent of patients with AML. There are other mutations such as DNMT3A, ASXL1, and TET2 that we typically see in patients with MDS or even a pre-leukemia state called CHIP. For other patients, we have mutations that are targetable like IDH1 or IDH2.

Those two mutations happen in probably 10 percent to 15 percent of patients diagnosed with AML. Why are those important? They’re important because we have oral medications that are pills that patients can take. In the relapse setting for many patients after induction or intensive chemotherapy, they can use these oral therapies to try and control their leukemia. These are pretty exciting. 

All of these oral therapies have been approved in the last two to three years in the space of leukemia, so it’s been a game-changer in terms of identifying these mutations and then identifying drugs that target those mutations. It’s really changed the landscape for patients with AML. It’s new information, and that’s why as patients you want to hear about this so you know what questions to ask and you know, can you tell me, am I a candidate for one of these oral medications that is now available for patients with AML?

Katherine:                  

Dr. Carraway, thanks so much for joining us today.

Dr. Carraway:            

Thank you for the opportunity to be here. 

Katherine:                  

And thank you to our audience. I’m Katherine Banwell.

Navigating AML Treatment Decisions

Navigating AML Treatment Decisions from Patient Empowerment Network on Vimeo.

What factors can help determine the best treatment path for your AML? This animated video walks through important considerations that may help in navigating treatment decisions, including how genetic testing results, treatment goals and patient preference can impact your choice.

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Effective AML Combination Treatment

Transcript:

Hi, I’m Gina. I’m a nurse practitioner and I specialize in acute myeloid leukemia, or AML.

When diagnosed with AML, it’s important to take steps to get a deeper understanding of your disease, and the available treatment options, so that you can feel confident in your care decisions.

Before we walk through the important steps to decide on a treatment path, I want to remind you that this video is intended to help educate AML patients and their loved ones and shouldn’t be a replacement for advice from your doctor.

OK, let’s get started.

The first step is to understand your diagnosis, so that you can find out what treatments are available to you. Unlike solid tumor cancers, such as lung or breast cancer, AML is not staged. Instead, your physician will use lab testing, including blood and bone marrow tests, to determine the subtype of your AML and if you have any chromosomal abnormalities to determine if your AML is low, intermediate or high-risk.

Knowing your risk can impact your prognosis and help establish the best treatment option for you. If you don’t know your subtype, ask your doctor for the information and if you may need further testing to reach a more accurate diagnosis.

Testing that identifies characteristics unique to YOUR AML can impact your treatment options and determine if a targeted therapy or immunotherapy might be more effective. These tests include:

  • Molecular testing
  • Cytogenetic analysis (or karyotyping), and
  • Fluorescence in situ hybridization also known as a FISH test

Before you start any treatment, it’s essential to insist that you have had relevant testing.

Next, you should understand treatment goals. The first goal of AML therapy is to get into remission. The second goal is to maintain that remission.

Induction therapy, or the first phase of treatment, is meant to induce remission. This first-line treatment kills as much of the disease as possible and returns blood counts back to normal.

Consolidation treatment, also referred to as post-remission therapy, is used to prevent leukemia cells from returning and maintain remission. In some patients, stem cell transplant acts as a consolidation therapy. In others, additional treatment options to maintain remission can be explored.

The next step is to consider your treatment options with your doctor. It’s important to understand the approaches available for YOUR individual disease. AML treatments can include:

  • Chemotherapy
  • Targeted therapy
  • Stem cell transplant
  • Immunotherapy
  • Clinical trials, which may provide access to treatments that are not yet approved.

Or, you may receive a combination of one or more of these treatments.

Once you understand the therapies that are available to you, it’s time to talk to your doctor about the risks and benefits of each option. Your doctor will also consider your age, overall health, and existing conditions before suggesting a treatment course.

So, what questions should you address when discussing your treatment goals with your doctor? Consider asking:

  • Is stem cell transplant a viable option for you?
  • Can you tolerate high-intensity therapy or is low-intensity therapy better for you?
  • How will the treatment impact your quality of life and lifestyle?
  • Are there short or long-term treatment side effects that may occur after you have completed treatment?
  • What is the plan if the first approach to treatment isn’t effective?
  • Is there a clinical trial that might be right for you?
  • Is there a member of the team, such as a social worker, that can help you understand the potential treatment costs? And is there access to financial resources that can help you if needed?

Remember that you have a role in making decisions regarding your care. Insist that all of your questions are answered when making a decision with your healthcare team. If you don’t feel supported or you don’t feel heard by your healthcare team, then it is always best to seek a second opinion.

Finally, once you have gathered all the information, it may be helpful to talk it out with people you trust, such as a partner, friend or family member, to help you make a decision that you feel confident about.

Now, how can you put this information to work for you?

  • Ensure that you have an accurate understanding of your diagnosis.
  • Make sure you have had appropriate testing to establish your subtype and risk.
  • Understand your treatment options and talk with your doctor about what’s best for YOUR AML.
  • Remember, you are a partner in your care and have an active voice in finding the best treatment for you.

Visit powerfulpatients.org/aml to learn more about AML.

How Will I Know if My AML Treatment is Working?

How Will I Know if My AML Treatment is Working? from Patient Empowerment Network on Vimeo.

During acute myeloid leukemia (AML) treatment, specific tests help to gauge a patient’s treatment response. Dr. Pinkal Desai details how diagnostic tests are used in monitoring the efficacy of an AML therapy

Dr. Pinkal Desai is Assistant Professor of Medicine at Weill Cornell Medical College and a hematologist specializing in acute myeloid leukemia (AML) at Weill Cornell Medicine. Learn more about Dr. Desai, here.

Download Program Resource Guide

See More From The Pro-Active AML Patient Toolki

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What Are the Goals of AML Treatment?

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Choosing an AML Treatment Path: What Should You Consider?

What Is the Patient’s Role in Making AML Treatment Decisions?

What Is the Patient’s Role in Making AML Treatment Decisions?

Transcript:

Katherine:                  

Once a patient has started treatment, how do you know if it’s working? How do you gauge that?

Dr. Desai:                   

When a patient begins treatment, whatever their regimen is, for the most part, it takes about a month to get into remission. So, initially, with any treatment we would use, the blood counts will actually go down. Everything is down, down, down. That’s important, and it’s good, actually, because if we can’t wipe out these cells, then we’re not going to. The patient’s not going to go into remission. It’s good that these blood counts drop and they keep like that for a month.

After a month, generally, is the first look on an average to see where it is, and that kind of depends on the regimen. For intensive chemotherapy, we take a look in the middle, like Day 14, to see did we wipe out all the leukemia? And can we modify treatment so that whatever might be left behind will clean out? For lower intensity treatments, it’s about a month. So, that’s the first sort of real look at whether a patient is in remission.

And again, when I say, remission is a morphologic criteria that we see the blast count are less than 5 percent, and the cells are – the normal cells are back to what is considered within normal limits or normal for that person’s age. And the idea, at that time, is to not only just confirm remission, but like I was saying, how good is the remission.

So, that’s where MRD testing comes into play. You want to see what you want to find, even if it’s by small numbers, what is the percentage of leukemia that’s left behind. 0.01 percent, 0.001 percent. This is important.

The goal is to ultimately get that down to zero, and that’s how we use it during induction, even when they’re going through consolidation, we’re episodically monitoring with bone marrow or blood testing for some of these molecular mutations that is there continued response from where we started off? And once the treatment is done, we are still, we’re seeing these patients on a regular basis, sometimes doing bone marrow biopsies at regular intervals, to again make sure that there is continued response. And can we see something different, or is there an emerging population of cells that are worrisome, and how do we modify our treatments to try to kill these cells?

What Could Emerging AML Treatment Approaches Mean for You?

What Could Emerging AML Treatment Approaches Mean for You? from Patient Empowerment Network on Vimeo.

In the changing landscape of acute myeloid leukemia (AML) research, how could emerging treatments impact care for patients? Dr. Pinkal Desai shares information about combination therapies, immunotherapy, and clinical trials, and explains the value of MRD in tracking AML response.

Dr. Pinkal Desai is Assistant Professor of Medicine at Weill Cornell Medical College and a hematologist specializing in acute myeloid leukemia (AML) at Weill Cornell Medicine. Learn more about Dr. Desai, here.

Download Program Resource Guide

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Transcript:

Katherine:                  

Are there emerging approaches for treating AML that patients should know about?

Dr. Desai:                   

So, there are several, and this is where there’s lots of lots of new drugs that have been approved. A lot of drugs in the pipeline. And within the categories, you can divide up where the advances are being made in several categories. So, the first one is, can you make a better induction regimen? So, how can you combine chemotherapy or hypomethylating agent plus venetoclax combination?

Can you add more targeted agents to these bad points to improve the chances of remission and to keep the patients in remission? So, that’s one aspect of it, that this is important.

There’s obviously this whole concept of immunotherapy of AML, where there’s a lot of antibodies treatment or drugs that affect the immune modulation that are being used both in up-front leukemia, in many times in the older patients, itself. There are clinical trials, obviously.

And also, in the relapse setting, there are CAR-T cells being used in leukemia therapy in the relapse setting. This is important, and a lot of new drugs are being used in the relapse setting. So, there’s this whole new sort of portfolio of clinical trials and treatment options for patients.

And the third aspect, which is, I would say, very important and as important as using better drugs, is to be able to quantify how the patients are responding to these treatments. Because we don’t want to start treatment, and then be blind about the kind of responses they’re getting.

There’s a whole new concept, what we call MRD measurements, or minimal residual disease, or measurable residual disease, MRD monitoring. That’s very important. So, when a patient starts with chemotherapy, and then you have subsequent bone marrows, even if they’re in remission, the quality of remission matters. The amount of MRD or amount of leukemia that’s left behind matters. And how do we direct our treatments to clean up that MRD? And how do we monitor this MRD, so that we can see what happens in the future? Many times, MRD can tell us that a patient’s going to relapse six months later. And how do we use that information?

So, these are very important aspects of monitoring of treatment that is important, and to measure MRD, not just by looking at the cells themselves, but using the patient’s own signature of molecular mutations that we found at baseline at the time of diagnosis. And how do we keep an eye on that?

This is another new world and new ways to figure out how best to use new drugs, maintenance approaches, better consolidation approaches, and how do we use MRD to mix all of these together to get the best possible outcome for these patients.

I think we’ve seen tremendous progress in leukemia, just over the last five years. We went from pretty much having two drugs to treat leukemia, chemotherapy, 7 and 3, and some hypomethylating agents, to a flurry of 15 new approvals. We now have targeted therapies. We have new clinical trials. I’m very hopeful that the combination of all of the things that we’re talking about, how to monitor patients, how to best utilize stem cell transplants. We’re entering a new age in leukemia, and I’m hopeful that with the advent of all of these drugs and what we know about leukemia, we can actually have a very good shot now to improve cure rates in leukemia.

AML Treatment Approaches: What You Should Know About Your Options

AML Treatment Approaches: What You Should Know About Your Options from Patient Empowerment Network on Vimeo.

What should acute myeloid leukemia (AML) patients and care partners know about treatment options? Dr. Pinkal Desai shares information about frontline treatments, targeted therapies, combination therapies, and clinical trials, and explains an important clarification regarding a newly approved oral hypomethylating agent.

Dr. Pinkal Desai is Assistant Professor of Medicine at Weill Cornell Medical College and a hematologist specializing in acute myeloid leukemia (AML) at Weill Cornell Medicine. Learn more about Dr. Desai, here.

Download Program Resource Guide

See More From The Pro-Active AML Patient Toolki

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What Are the Goals of AML Treatment?

What Are the Goals of AML Treatment?

Choosing an AML Treatment Path: What Should You Consider?

Choosing an AML Treatment Path: What Should You Consider?

Understanding Risk in AML: How Molecular Testing Affects Treatment Option

Transcript:

Katherine:                  

So, in looking at a treatment plan, we’ve discussed the factors that go into that choice. And then, you’ve also just covered some treatment approaches and who they might be right for. So, you’ve talked about chemotherapy. You’ve talked about stem cell transplant. What about targeted therapies and also clinical trials? Where do they fit in?

Dr. Desai:                   

Right now, if somebody’s diagnosed with new AML or newly diagnosed leukemia, and they are eligible for intensive chemotherapy of the approved agents, the one targeted therapy that does make a difference is midostaurin, which is a FLT3 inhibitor.

And patients who do have a FLT3 mutated leukemia, the standard of care is treatment with intensive chemotherapy in combination with midostaurin. So, this is where chemotherapy’s combined with the backbone of the targeted therapy.

There are clinical trials of other targeted therapies that are being combined with frontline treatment. That frontline treatment might be intensive chemotherapy or more of the hypomethylating-based therapy, which is what we call lower intensity therapy. So, these are where the clinical trials are asking the question that can be just how midostaurin was combined with chemotherapy.

Can we combine other targeted therapies with the backbones that currently exist? Chemotherapy or lower intensity hypomethylating agents. And can we combine them to improve the chances of going into remission and staying in remission?

I would say clinical trials are extremely important. Almost any stage of leukemia, whether it’s a new diagnosis, whether it’s second-line or relapse, it’s important, because these questions that are being asked are very relevant. How do we improve upon the existing known remission rates and survival in leukemia?

There are targeted therapies available for IDH inhibitors that are being combined. There is also a newly approved BCL2 inhibitor, venetoclax, which is used in combination with hypomethylating agents, that have shown survival advantage over single agent.

Hypomethylating agents, anybody who’s older, we are now combining the venetoclax with hypomethylating agents for what we call lower intensity induction treatment. And there are several others in the making. We have TP53 inhibitors.

As we talked about this, that leukemia is not one diagnosis, really. AML has several, several, several subtypes, and once we find out what makes that particular patient’s leukemia tick, and if you have a targeted inhibitor towards it, it’s logical that you would want to combine it with what the backbone of treatment is, and that’s where clinical trials are extremely important in asking most relevant questions and improving patient survival. 

Katherine:

Dr. Desai, I learned that oral azacitidine was recently FDA approved. What does that approval mean for patients and who is it right for?

Dr. Desai:                   

So, oral… So, azacitidine. For patients who may or may not know this, azacitidine has been approved in the IV or subcutaneous formulation for treatment of myelodysplastic syndrome and leukemia.

And this is, when I was saying that there is a lower intensity treatment of hypomethylating agents, that’s one of the drugs, azacitidine. And we use it for induction treatment in patients who do not qualify for intensive chemotherapy in AML.

So, oral azacitidine has been currently approved for older patients who have gone through intensive chemotherapy.

The trial was done in patients who did not have prior hypomethylating exposure of any kind, so people who had not seen any IV or subcutaneous azacitidine, they had leukemia, they get the intensive chemotherapy, finish the induction part, and the, what we call, consolidation part, which is the cleaning up with more additional cycles of chemotherapy.

Once that is done, the old standard of care was to not do anything, so these are obviously for patients who are not transplanted. So, once somebody, just to give a background on this, if somebody’s in remission and they’re transplant eligible, we make a decision whether they should go for transplant or they should get some more chemotherapy rounds. Both are consolidation of some kind, transplant or chemotherapy.

So, let’s say somebody went through induction, got into remission, and it was decided that they’re not candidates for transplant, or the patient didn’t want to go through a transplant, and you go for the consolidation. And the old standard was, after that, to do nothing. And oral azacitidine was tested in this situation, where half the patients got oral azacitidine as maintenance. It was given as pills, to take it for two weeks out of a 28-day cycle.

So, every month, you take it for 14 days. And half of them didn’t get the drug, oral azacitidine. And the drug was recently approved for FDA for having a survival advantage over the standard of care, which is to do nothing after consolidation is over.

So, in other words, this is currently available for patients, older patients, who’ve gone through induction chemotherapy, and/or consolidation, and then finished it. Then, you start this oral azacitidine for keeping this remission going on longer. And that’s where the niche of this drug is.

It is very, very important to understand that oral azacitidine has a very different kinetic in the body than IV azacitidine. So, I think people, many times, get confused between is IV the same as oral? They are totally different drugs and have a different way it affects the bone marrow.

So, they’re not to be interchanged for that indication. Oral azacitidine has been strictly approved for maintenance of remission, post-chemotherapy.

What Is the Patient’s Role in Making AML Treatment Decisions?

What Is the Patient’s Role in Making AML Treatment Decisions? from Patient Empowerment Network on Vimeo.

What role do acute myeloid leukemia (AML) patients have in their treatment decisions? Dr. Pinkal Desai explains factors that go into decision-making and how patients may help guide the treatment option that’s best for them.

Dr. Pinkal Desai is Assistant Professor of Medicine at Weill Cornell Medical College and a hematologist specializing in acute myeloid leukemia (AML) at Weill Cornell Medicine. Learn more about Dr. Desai, here.

Download Program Resource Guide

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What Are the Goals of AML Treatment?

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

Transcript:

Katherine:                  

What is the patient’s role in this decision?

Dr. Desai:                   

I think it’s important for patients to understand why the decisions are being made or what goes into the decision-making. Because the patients would appreciate, if they know, that these are the genetic subtypes, and this would be the best sort of approach for them.

So, from a patient’s side, their role is, 1) to understand all the factors that go into the decision-making. And the second aspect, which is important, is their own values and their own decision on what treatment they would like to have. 

So, there are – sometimes, it’s very white and black. There are many times where it’s a gray zone, in the sense that there is a best treatment that’s available, that the oncologist would discuss, but it’s also possible to choose between two different kinds of therapy options.

If the patient is eligible, for example, for both intensive and non-intensive treatment, then what would they prefer based on what’s going on in their life? Whether they want to be hospitalized for 30 days for intensive induction or not? Do they want to do this out-patient? A lot of these things are important, and they have to be involved with this.

The third aspect, which is very important from a patient standpoint, is the need for transplant. So, patients who are younger and transplant eligible for leukemia that has a higher risk of coming back, we do recommend a stem cell transplant, so that the patients have to understand the process of stem cell transplant.

Sometimes, it’s slam dunk that a transplant is needed, but there are certain times where you could or could not go for it, and this is where the patient’s choices and values are extremely important, that once they hear all of this information, they would decide whether they should or should not go for stem cell transplant.

Choosing an AML Treatment Path: What Should You Consider?

Choosing an AML Treatment Path: What Should You Consider? from Patient Empowerment Network on Vimeo.

What should be considered when choosing an acute myeloid leukemia (AML) treatment path? Dr. Pinkal Desai explains the factors that are considered to determine the best treatment for an individual patient.

Dr. Pinkal Desai is Assistant Professor of Medicine at Weill Cornell Medical College and a hematologist specializing in acute myeloid leukemia (AML) at Weill Cornell Medicine. Learn more about Dr. Desai, here.

Download Program Resource Guide

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Transcript:

Dr. Desai: 

Now, in terms of how we decide treatment, so, there is the leukemia aspect of it, of the biologic indicators of leukemia, and there’s obviously the patient. Because everybody is different. There are patients who are coming in at various ages, like you said. Age is a very important thing to look at, because if you’re younger, the patient’s younger, then they’re usually eligible for what we call intensive chemotherapy. And if the patient is older, they may not be able to handle intensive chemotherapy, and in which case, the induction treatment or the first treatment, we call induction treatment, is basically the treatment we give to get you into remission.

So, the induction treatment decision is based largely from a patient aspect on age.

Whether to go with intensive induction chemotherapy, or with lower intensive chemotherapy, depending on the person’s age.

Now, age is… There is a loose definition of what is considered older age, but we generally say over 75, patients cannot handle intensive chemotherapy. Under 75, under 70 for sure, they’re eligible for intensive chemotherapy, but it’s a biological continuum. So, there are patients who are much healthier, even at older ages, and much older at younger ages. So, we take into consideration not just the age, but also what else do they suffer from? Do they have other comorbidities? Is the heart okay? Do they have kidney damage? Do they have lung damage from previous comorbid illness? And that all goes into figuring out what kind of treatments can they handle.

And that’s the patient aspect of it. Then there’s the biologic aspect of the leukemia itself. Leukemia, the chromosome type. There are leukemias that respond extremely well to intensive chemotherapy. So, you’d figure that kind of treatment for it. Within the molecular subclassification, as we said, there are mutations in certain genes, like FLT3 and IDH. There are targeted treatments towards that, so we look at all of these genes to figure out what is the best mix of chemotherapy, targeted therapy, lower intensity therapy, to look at and combine so that we can have the best chance of being in remission, and to continue to be in remission.

What Are the Goals of AML Treatment?

What Are the Goals of AML Treatment? from Patient Empowerment Network on Vimeo.

When it comes to acute myeloid leukemia (AML), what are the goals of treatment? Dr. Pinkal Desai defines the role of remission and the specific goals of treatment for AML patients. 

Dr. Pinkal Desai is Assistant Professor of Medicine at Weill Cornell Medical College and a hematologist specializing in acute myeloid leukemia (AML) at Weill Cornell Medicine. Learn more about Dr. Desai, here.

Download Program Resource Guide

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Transcript:

Katherine:      

Dr. Desai, when deciding on a treatment approach with a patient, I imagine you have to consider a number of factors, like a patient’s age and their overall health. Let’s walk through these considerations, and we’ll start with treatment goals. What does that mean, exactly?

Dr. Desai:                   

So, the first treatment goal is to get into remission. Patients with leukemia will have abnormal blood counts, they don’t feel well, they have a risk of infection, and all of that is only going to get better if you can get into remission.

And remission means that the bone marrow has a blast count less than 5 percent. Now, remember, we talked about if it was over 20, it’s considered diagnosis of AML. So, we want it gone under 5 percent, preferably zero. And we want all the blood counts that are abnormal to normalize back to what it would be for a normal person.

So, that’s the sort of definition of remission, and we want to get there, because ultimately, patients feel extremely good once they go into remission. They feel fine. The risk of infection goes away. It is absolutely important for long-term quality of life and survival. The first goal is to get into remission.

The second goal is to keep that remission going, for as long as possible, and also increase the chances of cure.

So, going into remission does not mean that a patient is cured of leukemia. It means that we’ve taken the first step of knocking the leukemia down to its knees, but there are still a few cells that are hanging out, and they’re still hiding. And the rest of the treatment and approach is to try to kill these cells and improve the chances of cure. So, and generally we say, once you get into remission you stay in remission, and when you’re past that five-year mark, we say leukemia is cured.

So, the first goal is get into remission. Second, keep yourself in remission, and that’s the whole sort of few things that we look at.

Understanding Risk in AML: How Molecular Testing Affects Treatment Options

Understanding Risk in AML: How Molecular Testing Affects Treatment Options from Patient Empowerment Network on Vimeo.

How does molecular testing impact acute myeloid leukemia (AML) treatment options? Dr. Pinkal Desai discusses molecular testing and how results may help determine the best treatment path for patients.

Dr. Pinkal Desai is Assistant Professor of Medicine at Weill Cornell Medical College and a hematologist specializing in acute myeloid leukemia (AML) at Weill Cornell Medicine. Learn more about Dr. Desai, here.

Download Program Resource Guide

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Transcript:

Katherine:      

Dr. Desai, is there a high-risk and a low-risk AML? And if so, what are the indicators?

Dr. Desai:                   

So, in terms of when we talk about risk of leukemia, many patients, when they come, they frequently ask what stage this is, which is generally not how leukemia is categorized, unlike lung cancer, or breast cancer, or any of the solid tumors. Leukemia is in your blood and in your bone marrow, so it’s kind of like all or none to some extent. When we talk about risk in leukemia, we’re talking about what is the chance of this leukemia coming back in the future. So, is the chance high, intermediate, or low?

And that’s how we categorize leukemia, into these three sort of risk categories, low risk, intermediate risk, and high risk. These risk categories are made up.

We decide these based on information from two aspects. One is the chromosomes, which we talked about. There are certain good risks of chromosomal abnormalities as well, where, for example, poor binding factor leukemias, where these leukemias tend to respond very well to chemotherapy. There are some higher risk, that the chances are higher to come back. And then, the middle category of intermediate risk, where it’s sort of in the middle.

The molecular subtype, or the molecular classification of AML is extremely, extremely relevant, because it gives you pretty much your own signature, and the patient sort of specific, personalized risk of whether this is going to have a high, intermediate, or a low risk to come back.

So, it’s a combination of chromosomes, and the molecular subtype, which is extremely important in figuring out the risk category.

Now, in the course of the treatment and decision-making of leukemia, we don’t have – we’ll have the chromosome information quite early, usually within the first two to three days, but the molecular information, some of it comes back pretty fast, like in a couple days from the testing. But many of these tests, the full panel comes back about 14 days after we do the original bone marrow biopsy. Some of these decisions on whether this is high risk or low risk is relevant in the long run. These decisions happen later, and you don’t have to wait for the treatment, obviously. This is more for what happens after a patient goes into remission.

But there are certain molecular genes that are very important in deciding treatment up front, and those we expedite, and they are back usually before treatment decision is made. For example, FLT3 ITD or FLT3 TKB.

These are two genes where the up-front treatment decision changes, depending on the presence or absence of this gene. So, you really, really do want to know this information early on.

Chromosomes you absolutely need it before treatment begins, because there are several options of leukemia treatment that are specific to certain chromosome subtypes. So, that’s like the basic information you need to have before making any treatment plans.

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

Key AML Testing for Better Care: Understanding Prognosis and Treatment Choices from Patient Empowerment Network on Vimeo.

After an acute myeloid leukemia (AML) diagnosis, additional tests must follow to determine prognosis and treatment options. Dr. Pinkal Desai explains key tests that aid in choosing optimal care for each patient. 

Dr. Pinkal Desai is Assistant Professor of Medicine at Weill Cornell Medical College and a hematologist specializing in acute myeloid leukemia (AML) at Weill Cornell Medicine. Learn more about Dr. Desai, here.

Download Program Resource Guide

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Transcript:

Katherine:      

Other than a complete blood count, what additional testing should take place following an AML diagnosis?

Dr. Desai:                

So, a blood count or CBC is just a hint that there might be AML. It’s certainly not diagnostic.

But when you see that there are some abnormalities in blood count, and there might be the presence of these immature cells or blasts in circulation, there is suspicion that this is acute myeloid leukemia. The diagnosis, the gold standard for diagnosis, is a bone marrow biopsy, which is a procedure that can be done out-patient or in the hospital, depending on where the patient is. It takes about 15 minutes, where we take a sample out of the hip bone and look at the cells. This is where bone marrow is being made, so you’re going to exactly where the problem lies, and seeing if the blast count is increased.

So, the diagnosis of AML is established when the blast count is over 20 percent in the bone marrows. And normally, it needs to be less than 5 percent.

And if it’s over 20 percent, that’s the diagnosis of AML. Whether it’s over 20 percent in the bone marrow or in the peripheral blood.

It doesn’t matter, one way or the other. This is a diagnosis of AML, but you do need a bone marrow biopsy to confirm diagnosis of AML.

Katherine:                  

What about genetic or molecular testing? Is that done?

Dr. Desai:                   

AML diagnosis is just one part or the first step of saying somebody has leukemia. There is a slew of other tests that are important, and we generally consider, within the genetic tests, we generally consider two kinds of testing. One is the cytogenetics, or the karyotype analysis, which looks at the chromosomes in our bodies.

So, leukemia can be associated with big chromosomal changes, and that’s important to recognize. And the second one is the molecular testing, and we’ll go over both of them.

The chromosomes, or the karyotypic analysis, the vast majority of leukemia patients have a normal chromosome type, but there are certain recurrent abnormalities in chromosomes that we see in leukemia, and that’s important to know for a variety of reasons: treatment decisions, prognostication.

And the second part of it, the molecular, these are actually genetic routine analysis, and this is not somebody – it doesn’t mean, when we say genetic testing, it’s not the patient’s own normal genetic type. So, we’re not looking for what they have inherited. Most of leukemia is actually a random event, and it’s not inherited. We’re talking about genetic damage that the leukemia cells have within themselves.

It gives us the signature of the leukemia, and it helps us understand what genetic abnormalities are present in the leukemia. There are several panels, 50 to 100 genes, but there’s usually recurrent genetic damage that leukemia cells have.

And you want to know that, because again, like karyotype, this is important in treatment decisions, and also in the prognostication and prediction in the future.