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Essential Testing for Lung Cancer Patients: How Results Impact Treatment Choices

Essential Testing for Lung Cancer Patients: How Results Impact Treatment Choices from Patient Empowerment Network on Vimeo.

What testing should take place after a lung cancer diagnosis? Dr. Jessica Bauman discusses the various imaging and molecular tests for lung cancer, and how the results may inform treatment choices. 

Dr. Jessica Bauman is assistant professor in the department of hematology/oncology and as associate program director of the hematology/oncology fellowship training program at Fox Chase Cancer Center in Philadelphia. Learn more about Dr. Bauman here.

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What You Should Know When Making a Lung Cancer Treatment Decision


Transcript:

Katherine:               

Dr. Bauman, what testing should take place following a lung cancer diagnosis?

Dr. Bauman:                

So, this very much depends on how the cancer was diagnosed initially. So, some cancers are diagnosed on screening – lung cancer CTs right now – but other cancers are found incidentally, for other reasons. Or there are some that are diagnosed with a scan because somebody’s developing a symptom. So, in general, what I would say is that we always need good imaging essentially of the entire body when a lung cancer is suspected. Often this includes CAT scans, but this very commonly also includes a PET scan. And it will often include a brain MRI as well because the best way to the look at the brain is with an MRI.

Obviously, that can vary a little bit depending on what studies people have already had and what radiologic techniques are most accessible.

Katherine:                   

What about molecular testing and biopsies?

Dr. Bauman:                

So, sorry, I was sort of going on the imaging. But so, of course, you need full imaging. But the first thing you need to do that is paramount is establishing a histologic diagnosis, which goes to this initial thought of, “Is this small cell? Is this non-small cell? What is it?” So, if there is a lung mass that is suspected to be lung cancer, the first thing that happens is a biopsy as well as imaging. The imaging helps us establish, “Has this gone anywhere else? Does it involve the lymph nodes?” and helps us with the initial staging workup. Often there is a biopsy of the mass itself.

But there are often biopsies as well as the lymph nodes that are involved, in particular in the center of the chest called the mediastinum, because that also helps us establish the stage of the cancer.

And then if the cancer does look to have spread to somewhere else, we sometimes biopsy only that area or that area in addition to establish that it, in fact, has spread to a different place such as the liver or the bone. Once that biopsy is done, and once we know what type of lung cancer it is, then we also send more studies on the biopsy itself that help us determine what the best treatments are, in particular when we’re talking about what I call “systemic treatments.”

So, treatments that are going into the body and all over the body that involved immune therapies, chemotherapies, or targeted therapies. So, that extra testing that we do is something that’s called molecular testing.

It’s also called next generation sequencing. There are a bunch of different terminology that we use.

Katherine:                

Okay. Dr. Bauman, would you walk us through how lung cancer is staged? And is it different for small cell vs. non-small cell lung cancer?

Dr. Bauman:                

Absolutely. So, as we talked about, the first thing that we do is we do get a biopsy to establish the diagnosis. The second piece is often if it looks to be a cancer that is only limited to the chest – so there is a mass and maybe some activities in lymph nodes that we’re concerned about but nowhere else – not only do we want to biopsy the mass itself, but we also want to know whether those lymph nodes are involved. So, those are biopsied because that will tell us the stage of the cancer. Staging very much depends on the size of the tumor itself, and then it also depends on, “Has it spread to lymph nodes in the center of the chest, and has it spread outside of the chest to other places?”

And so, early-stage lung cancers are just the primary cancer itself that has not spread anywhere else. More advanced stage lung cancers – things like stage IIs and stage III lung cancers – are ones that also involve the lymph nodes. And then a stage IV lung cancer involves a lung cancer that has spread to somewhere outside of the body. And depending on the stage is really what determines the way we approach treatment for these patients.

Katherine:                  

And that is actually my next question. What do the results of these tests tell us about prognosis and treatment choices?

Dr. Bauman:                

So, they tell us stage, and, ultimately, prognosis and treatment choices are completely linked to the stage of a cancer. So, an early-stage lung cancer, often a stage I or stage II lung cancer, primarily our first choice of treatment is surgery. And if surgery is feasible for the patient – because, of course, it also depends on their other medical comorbidities and whether they can withstand a surgical resection of the cancer.

But usually, early-stage lung cancers we start with surgery. And then depending on what the pathology shows us, we sometimes include a course of chemotherapy afterwards to decrease the risk of the cancer coming back. More advanced lung cancers, so stage III lung cancers, often involved what we call “multiple modalities.” So, for some patients we do a combination of chemotherapy and radiation in an attempt to cure the cancer. Often that is followed by immunotherapy. There are other patients who have stage III lung cancer where we do chemotherapy and radiation and follow that with surgery.

So, it’s a very case-dependent decision algorithm, where it really depends on where the tumor is, the type of tumor, what the surgery would be, what the patient’s underlying health status is, etc.

And then if it is a stage IV cancer, often we are really approaching this with systemic therapies. So, once a cancer has spread outside the lung, we traditionally think of this often as an incurable cancer. And there is a much more limited role of surgery and radiation, though I wouldn’t say that they’re absolutely off the table. Again, we sometimes think of these in sort of a case-by-case scenario. But in general, our approach for a stage IV cancer is with some kind of systemic therapy. And that completely depends on all those special tests that we do that we were talking about that we send on that initial biopsy.

Katherine:                   

What about the significance of chromosomal abnormalities?

Dr. Bauman:                

So, what I would say is, what we do for, in particular, in the setting of a stage IV lung cancer diagnosis right now, is we send molecular testing on the biopsy samples of these patients, in particular if they have adenocarcinoma.

And the reason we do this, what this gives us, is it tells us about the DNA of the tumor, and whether there are genes in the tumor that are changed in some way that are affecting the cancer’s ability to grow. And the reason that’s so important, is there are new treatments that really capitalize on those changes in the tumor to be able to stop the cancer from growing. The best example of this is for people who have something called an EGFR mutation.

And there are multiple different kinds of mutations. I call it “alphabet soup” because there are so many different letters and numbers.

But if people have an EGFR mutation that we think is one of the primary reasons they have this cancer growing, there are pills that target that EGFR protein that stop the cancer from growing. But if they don’t have that mutation, then those pills are not going to do them any good.

And so, that is really where lung cancer treatment and diagnosis has become so personalized based on, of course the person itself, but also the characteristics of their tumor.

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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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 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

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?

AML Treatment Decisions: What’s Right for You Resource Guide

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

Related Resources:

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

See More From The Pro-Active AML Patient Toolki

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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

See More From The Pro-Active AML Patient Toolki

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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.

Shared Decision-Making: The Patient’s Role in Treatment Choices

Shared-Decision Making: The Patient’s Role in Treatment Choices from Patient Empowerment Network on Vimeo.

What is the role of the patient when it comes to treatment choices? Dr. Brady Stein details how he partners with patients in decision-making for their MPN care. 

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.


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Transcript:

Katherine:                  

What do you feel is the patient’s role in the decision for therapy?

Dr. Stein:                   

I think it’s a really important role. I think historically – and, this is decades past; this era should be well over and behind us – this era of authoritative medicine is over.

You can’t just have a doctor walk in the room and say, “This is your treatment, this is what you should do, I’ll see you later.” It’s shared decision-making, and that can be troubling for some patients. But, the idea of shared decision-making is us explaining options informing the patient and making decisions together. That’s really the paradigm for modern contemporary medicine.

Some patients have a harder time with that. A lot of patients say, “Well, doc, this is too overwhelming for me. I just want you to decide for me.” And, we try not to do that. That’s a more uncomfortable type of visit for me when a patient is very deferential and says, “Whatever you say, I’ll do.” That’s not really what we want to hear. I want to know that you feel really informed, that you have a good understanding because each of these treatments – any treatment, any medication has its pros and cons.

There are no real magic bullets, and each upside has an equal downside, so you have to engage and open a dialogue, and what that means is that patients need to read and learn. That’s hard, but patients need to become proactive in their approach to their own illness, and all the patients who are listening now are doing that, trying to get more education about your relatively rare illness that’s going to give you a much better framework to help make decisions together.

Katherine:                  

Absolutely. If a patient isn’t feeling confident with their treatment plan or their care, do you recommend that they maybe consider a second opinion or seek a specialist?

Dr. Stein:                   

Of course, yeah. These are rare diseases, and patients often – I would say that in my clinic, a lot of the patients direct their own second opinions. Oftentimes, it’s coming from the patient more so than their doctor. I think the patient community is very active, the patients are networking, and they’re finding the right specialist to get to.

I think it should be really a team approach. It’s never – it’s usually not very convenient to go to a university unless you live really close, so you want to have someone close to home who can handle the routine, and then, someone who maybe is a little bit further away who can see you once a year, can help with the big decisions, can be part of the healthcare team. So, we generally recommend that you have someone near, and that maybe you have someone far who focuses only on MPNs as part of your team, and now, it’s a little different. Telemedicine is becoming a pretty ingrained part of medicine. It’s a little easier to have those visits with a physician who’s far away because of telemedicine.

Self-Advocacy: Advice for Being a Pro-Active MPN Patient

Self-Advocacy: Advice for Being a Pro-Active MPN Patient from Patient Empowerment Network on Vimeo.

How can myeloproliferative neoplasm (MPN) patients be more pro-active in their care? Dr. Brady Stein shares advice to help patients educate themselves about the disease, while finding the right balance of knowledge to prevent them from feeling overwhelmed. 

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.


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Transcript:

Katherine:

Let’s talk about patient self-advocacy now, Dr. Stein. Patients can sometimes feel like they’re bothering their healthcare team with their comments and questions.

Why is it important for patients to speak up when it comes to symptoms and side effects?

Dr. Stein:                   

I smile a little bit because patients – I get a lot of patient emails by MyChart. That’s our medical record, and it’s a secure patient email, and a lot of patients will start their message by saying, “I’m sorry to bother you.”

And, I always say, “Why do you think that? It’s my job. Please don’t apologize for reaching out to me.” So, that’s kind of the first thing. Don’t feel like you’re bothering your doctor. There are certain things that we won’t know unless you tell us, and so, I think that’s pretty clear. When we’re in a patient room and there might be a husband and wife together, and whether it’s the husband is the patient or the wife is the patient, we might ask a question, and we might get, “No, everything is fine,” but all doctors kind of sneak over to the partner, and the partner may be saying – they’re making gestures to us. There may be nonverbal forms of communication to tell us there’s something much worse than what the patient is telling you.

So, again, “advocate” meaning you have to tell us what’s going on with you. If you’re worried about something, please don’t be stoic about it. These diseases are treated a lot based on your symptoms, and so, if you don’t tell uls about your symptoms, we won’t know.

And, in terms of advocacy, I think one of the things is that these are pretty rare diseases. In an academic center, no, this is our focus, but if you’re in a community practice where the doctor’s seeing 10-15 different things during the course of a day, it’s basically impossible to keep up with myelofibrosis, especially if you have one patient in your whole practice. I can’t do that for diseases that I see that I have only one patient. The medical literature can be overwhelming.

So, patients can quickly outpace their doctor in terms of their knowledge of these diseases, but I think it’s really important to read, to learn, and to think about the illness because you may find out things through your research that your doctor wouldn’t know are available. You may find a clinical trial, a new strategy, or a new test that they simply haven’t had the time to keep up with or learn about. So, that’s what advocacy is about. Reading is really important, but you have to find a balance. I want my patients reading, but you’ve got to find the right amount because there’s a certain amount of reading where the patients start to get overwhelmed.

All patients kind of get to this point. They take it in – like taking it in like a fire hydrant in the beginning of the disease, and it’s overwhelming, and then they start to find their balance. I think there’s a point where the reading becomes anxiety-provoking rather than ameliorating anxiety, and all patients just generally find their balance.

Is My MPN Treatment Working?

Is My MPN Treatment Working? from Patient Empowerment Network on Vimeo.

During myeloproliferative neoplasm (MPN) treatment, specific blood tests and diagnostic measurements help to gauge a patient’s treatment response. Dr. Brady Stein details the criteria he assesses in monitoring the efficacy of a therapy, including patient-reported outcomes.  

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.


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Transcript:

Katherine:                  

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

Dr. Stein:                   

That’s a good question because this is a very unique area. Yes, of course, in some respects, it’s straightforward with ET or PV. If we’re starting a medication to control a blood count in hopes of having lowered the thrombosis risk, you can look objectively at blood counts.

Okay, your hematocrit is at this goal? Yes, therapy’s working. You have not had a blood clot?

Yes, therapy’s working. So, there are some objective things. In myelofibrosis, there are some objective things like measuring the spleen and seeing it reduce. You can feel that with your hands, or you can do an ultrasound. So, there are some objective parameters of success. But, in this area, patient-reported outcomes are really important, and so, a measure of success is really just asking the patient, “Do you feel like your drug is working? Do you feel better?

It’s kind of a simple question, but it’s really important, and it’s what we ask in patients who are on certain therapies. “Do you feel like the net effect of your therapy is still positive? Do you feel like it’s helping?” Seems like a straightforward type of question, but I think the answer is extremely informative. When a patient says, “Yes, definitely, my medication is still helping me,” then I know that I don’t need to change it.

MPN Treatment Choices: Where Do Clinical Trials Fit In?

MPN Treatment Choices: Where Do Clinical Trials Fit In? from Patient Empowerment Network on Vimeo.

When considering MPN treatment approaches, clinical trials are a viable option for care. Dr. Brady Stein discusses clinical trials and factors to keep in mind when considering participation.

Dr. Brady Stein is a hematologist focusing on myeloproliferative neoplasms (MPNs) at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Learn more about Dr. Stein, here.


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Transcript:

Dr. Stein:                   

Clinical trials are always a treatment – always an option for patients with myeloproliferative neoplasms because while we have some standards, we can definitely improve upon those standards for certain. So, clinical trials are always a therapeutic option. I think the one thing is that it may not – it’s not always the most convenient option, but it could be a really important option if available to you.

So, clinical trials basically offer something new or novel that would not otherwise be available to other patients. So, ruxolitinib (Jakafi) was approved around 2011, but the first clinical trials were in 2007, so that’s the example I give to a patient about the benefit of a clinical trial.The patient can get access to a drug that’s effective perhaps three to four years before it’s commercially available.

That’s really the biggest advantage, is you can get early access to something that could really help you. The downsides are that clinical trials are not usually as convenient as regular care, there are often more visits, and there are a lot of unknowns – unknowns about whether it will work. Some side effects are known and expected; there are others that are unknown. So, it’s a lot to think about, but I think it’s always important to consider, especially if your first-line therapy has not been effective, if it’s losing its touch, it’s a good thing to think about for a second line.

Katherine:                  

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

Dr. Stein:                   

Yeah, absolutely. I think the first question – I think patients are often worried that they have a really rare disease, and why would anyone do research in this area, and that’s – the research community is extremely engaged, the productivity is pretty impressive, and there’s a lot of clinical trials in the space, and I think what I try to explain is pharmaceutical companies aren’t just targeting the most common diseases.

They have interests in rare diseases, and findings in rare diseases can be extrapolated to other diseases that you might think are unrelated, but they can share features, so when you find something working in one space, it can have broad applicability. So, there’s an abundance of research in myeloproliferative neoplasms which are emerging?

In PV, I think there’s quite a possibility that there’ll be a drug approval in 2021, a novel type of interferon called ropeginterferon

That is a drug that’s approved abroad; it’s approved in Europe, and I believe it’s approved in Taiwan, and the FDA is looking at it now. So, it’s a possibility that there’ll be a future option for patients with polycythemia vera. So, yes, it’s research now, but it could be available, and so, that’s the drug that I’m starting to talk more and more about for patients with PV.

In myelofibrosis, you have two JAK inhibitors that are approved, ruxolitinib and fedratinib, you have two others in clinical testing, momelotinib and pacritinib, and then you have a whole other class of what we call non-JAK2 type of therapies targeting the vast array of pathway abnormalities in myelofibrosis.

So, there are a number of different clinical trial options, especially in myelofibrosis. I think that’s the disease area where there are the most clinical trials.