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

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

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

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

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Expert Advice for AML Patients When Making Treatment Choices

Transcript:

Katherine:

Hello, and welcome. I’m Katherine Banwell, your host for today’s program. Today we’re going to discuss how to access the most personalized AML therapy for your individual disease, and why it’s essential to insist on key testing. Before we meet our guest, let’s review a few important details. The reminder email you received about this program, contains a link to program materials. If you haven’t already, click that link to access information to follow along during this webinar. 

Finally, please remember that this program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you. All right, let’s meet our guest today. Joining me is Dr. Ellen Ritchie. Dr. Ritchie, would you please introduce yourself? 

Dr. Ritchie:

Hello, my name is Dr. Ellen Ritchie, and I am an attending with a Leukemia service, and an assistant director since, for the last 15 years. 

And I treat mainly Acute Myeloid Leukemia, Myelodysplastic syndromes, which are kind of a pre-Leukemia; and Myeloproliferative diseases. And have a particular interest in the treatment of older patients with AML.  

Katherine:

Excellent, well thank you so much for joining us today. As we begin to talk about personalized therapy and AML, let’s start with the basics. How would you define personalized medicine? 

Dr. Ritchie:

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

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

Katherine:

Well, it sounds like, each person’s AML is unique. So, let’s help our audience be clear about basic testing. What tests are necessary to help understand a patient’s specific disease at diagnosis? 

 Dr. Ritchie:

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

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

So that’s the initial work up for any AML patient. 

Katherine:

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

Dr. Ritchie:

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

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

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

Katherine:

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

Dr. Ritchie:

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

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

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

Katherine:

Okay, that’s really great advice, thank you. How do the results of these tests affect prognosis and treatment? 

Dr. Ritchie:

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

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

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

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

Katherine:

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

Dr. Ritchie:

So, you want to choose whether a patient is most likely to benefit from intense induction chemotherapy. With strong chemotherapies where the backbone of those therapies would be an anthracycline, like Daunorubicin or Cytarabine, or Daunorubicin or Idarubicin, together with Cytarabine. And these are intensive chemotherapies. Versus, non-intensive chemotherapy which is able to be done as an outpatient, more frequently. And it is something that is gentler for a patient, they’re less likely to have severe toxicity. And the backbone of those regimens is using a drug called Azacitidine or Decitabine, together with a second drug called Venetoclax. 

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

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

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

Katherine:

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

Dr. Ritchie:

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

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

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

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

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

So, there are – there is therapy available for that type of mutation that was not available before through the clinical trials. And I expect in coming years that we’re gonna see more and more targeted therapies develop in AML which can be used potentially in combination with what we’re already using as backbones to enhance the outcome of patients with this disease. 

Katherine:

Well, how do targeted therapies work? 

Dr. Ritchie:

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

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

Katherine:

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

Dr. Ritchie:

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

So, it’s intensive chemotherapy because it has to be given in a hospital setting and requires intensive supportive care. Low-intensity therapy can be given in the outpatient setting. So, at the present time you can get a drug like Azacitidine, for example, which is an injection that you get seven days in a row. Unfortunately, you have to come to the doctor’s office every day for those injections, but once you’ve had the injection, you can go home. Combined with Venetoclax which is an oral agent. So, an oral agent can be given at home. You need close supervision in the physician’s office when you’re on this type of therapy, but you don’t need the constant support that you need if you are getting intensive chemotherapy. So, it can be done, in the comfort really of your home and with your family. You will have to come in and have transfusions potentially as an outpatient, nearly everyone does. And there’s always the risk that you develop a fever and if you do, you have to come into the hospital for IV antibiotics. 

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

Katherine:

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

Dr. Ritchie:

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

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

Katherine:

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

Dr. Ritchie:

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

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

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

Katherine:

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

Dr. Ritchie:

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

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

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

Katherine:

Yeah, those are great suggestions. We have a couple of audience questions. Mike wants to know, what does it mean to have high-risk AML? 

Dr. Ritchie:

High-risk AML means that there is something in your chromosomes that are worrisome and may confer a worse outcome. Or that one of the mutations that you have, or the combination of mutations that you have and the genetic testing are poor risk mutations that are associated with poor outcome. So, high-risk, really means a high risk of progression, or a high risk of – it’s a high risk of not going into remission and not being treatable AML. So, these are AMLs we treat aggressively, and if we get a patient into remission, we generally send high-risk patients to a bone marrow transplant. 

Katherine:

The second question is from Craig, he says; I’m currently undergoing treatment for AML, is the Covid-19 vaccine safe and effective? 

Dr. Ritchie:

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

Katherine:

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

Dr. Ritchie:

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

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

Katherine:

That’s what it sounds like. To close Dr. Ritchie, what would you like to leave the audience with? Are you hopeful about the future of AML treatment? 

Dr. Ritchie:

I’m very hopeful. I’ve worked in this field for 15 years and through the 15 years we have seen a lot of new drugs that have been approved for AML. It’s remarkable, the FLT3 inhibitors, IDH1 and IDH2 inhibitors, new formulations of intensive chemotherapy, like Vyxeos, the Pfizer drug; glasdegib – I can never say that one. And most importantly, venetoclax, which has really revolutionized our treatment of low-risk, or not low-risk, but the low intensity patient. 

I see in the future that there is gonna be more – there’s an emphasis on immunotherapy, so I think we’re gonna see more antibody-based therapy that’s going to be approved by the FDA. Maybe it will be used in combination with the drugs that we are already using. There are all sorts of combinations using all the FDA approved drugs in different ways together. So, we can maybe do better with the drugs that we have. And there’s always new targeted drugs which are being tested in AML. So, I think as time goes on, from a molecular perspective it will be even more targeted. And I’m hoping also that there will be oral formulations of a lot of our drugs. So, it’s kind of exciting that there’s an oral form of Decitabine called Inqovi, which is something that could potentially be given in induction therapy right off the bat with Venetoclax for an all-oral regimen at home. 

All of these things are great advances, in my opinion, and I think that the opportunity to treat patients outside the hospital, with more targeted therapy and immunotherapy is gonna be the future. 

Katherine:

Yeah. And the future sounds promising. Thank you so much for joining us today Dr. Ritchie. 

Dr. Ritchie:

Thank you for having me. 

Katherine:

And thank you to all of our partners.  

To learn more about AML, and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell, thanks for joining us.

NCCN Guidance on Safety and Effectiveness of COVID-19 Vaccines for Cancer Patients

NCCN Guidance on Safety and Effectiveness of COVID-19 Vaccines for Cancer Patients from Patient Empowerment Network on Vimeo.

Is the COVID-19 vaccine recommended for people living with cancer? Dr. Erin Roesch shares recommendations from the National Comprehensive Cancer Network (NCCN) for those undergoing cancer treatment, including guidance on mask wearing and advice for family members.

Dr. Erin Roesch is a breast medical oncologist at the Cleveland Clinic. Learn more about Dr. Roesch here.


Transcript:

Katherine: 

Many cancer patients have questions about the COVID vaccine. Is it safe? Do we need to continue wearing masks? Here to address these questions is cancer expert, Dr. Erin Roesch. Dr. Roesch, would you introduce yourself?

Dr. Roesch: 

Hello. And thank you for inviting me to participate in this very important conversation. My name is Erin Roesch. I am a breast medical oncologist at Cleveland Clinic.

Katherine: 

Excellent. Thank you so much for joining us today. I’d like to run through a list of concerns that cancer patients have about vaccines in general and the COVID vaccine specifically.

So, let’s start with a basic question. Should people get vaccinated if they have cancer?

Dr. Roesch: 

Yes. All individuals diagnosed with cancer should get the COVID-19 vaccine as recommended by the National Comprehensive Cancer Network or NCCN.

An immunocompromised state makes many people with cancer at higher risk of serious COVID-19 illness. Those who are vaccinated are less likely to become sick with COVID-19. And, also, vaccinated people who do get COVID-19 are much less likely to become seriously ill.

I would also mention that those living in the same household as a person diagnosed with cancer and caregivers or other close contacts should also get vaccinated.

Katherine: 

Another common question is whether people with cancer should wait for any reason to get the COVID-19 vaccine.

Dr. Roesch: 

Most people with cancer should get the vaccine as soon as they can with a few exceptions according to NCCN.

People in the process of receiving stem cell transplant or cellular therapy should wait at least three months after they finish treatment to get vaccinated.

Those diagnosed with certain forms of leukemia should also wait a few weeks after receiving treatment to allow their immune system to recover so the vaccine can be effective.

It’s not been clearly defined exactly how chemotherapy affects responses to COVID-19 vaccines. But some data suggests that immune responses may not be as robust. However, it is still recommended that those receiving chemotherapy and also immunotherapy and radiation should get vaccinated whenever they can.

Katherine:

I think a lot of people are concerned too about whether one vaccine is better than another. What would you say to them?

Dr. Roesch:

And that is a common question that I often get in my clinic. And I advise my patients to receive or take whatever vaccine they are offered.

We don’t really have any studies or data at this point suggesting one being better than another in cancer patients.

Katherine: 

Some people are wondering if the vaccine can give a person COVID-19. How would you address that?

Dr. Roesch: 

I would say that as none of the currently available vaccines are made with a live virus, the vaccine itself can’t give a person COVID-19. By getting vaccinated, actually, those who are immunocompromised are really helping society to prevent the spread of COVID-19. Immunocompromised people who get COVID-19 may be more likely to infect others due to prolonged shedding of the virus after infection.

Katherine:

What about side effects? Are the vaccine’s side effects worse for people with cancer?

Dr. Roesch:  

No. Side effects do not appear to be worse for those diagnosed with cancer. Results to date suggest that the vaccine’s side effects in people with and without cancer are really no different.

These side effects, as we have seen, may include arm soreness, rash, fatigue, chills, fever, headache, for example.

Katherine: 

And, finally, can cancer patients stop wearing a mask after they’ve been vaccinated?

Dr. Roesch:

Cancer patients should continue to wear a mask post-vaccination. Many people with cancer may have a harder time actually fighting infections and may not respond as well to vaccines. So, people diagnosed with cancer and their close contacts should get vaccinated and then continue to follow precautions, which include wearing masks, social distancing, hand hygiene.

Katherine:

Is there a certain length of time that people need to continue wearing a mask after being vaccinated?

Dr. Roesch:  

At this time, I would recommend patients continue to follow the CDC guidelines that are currently in place. And at this point, I don’t think we have a projected end time for that yet.

Katherine:    

Is there anything else you’d like to share with cancer patients who may be concerned about vaccinations?

Dr. Roesch:    

I would encourage those diagnosed with cancer to not only themselves get vaccinated but to also really voice and stress the importance of vaccination to those that surround them, including, again, members of their household, close contacts, and even beyond their inner circle.

I would also advise people to try and avoid letting the concern of possible side effects related to the shot deter them from getting it. The symptoms of COVID-19 can be much worse and potentially serious for some compared with the relatively minor side effects that we’ve seen with the vaccine itself.

I also would mention I’ve had personal patients that have expressed concern about functioning of their immune system while receiving chemotherapy and how this might affect their response to the vaccine. I do emphasize to them that even though responses might not be as strong as they may be in the absence of active treatment, I feel like the potential benefits of the vaccine still outweigh the risks in my mind.

Katherine:   

Thanks so much for joining us today, Dr. Roesch.

Dr. Roesch:

Thank you for having me.

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.

See More From Engage AML


Related Resources:

 

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


Related Resources:

 

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


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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.

What You Need to Know Before Choosing a Cancer Treatment

What You Need to Know Before Choosing a Cancer Treatment from Patient Empowerment Network on Vimeo.

Download Guide

Haga clic aquí para ver en español

What steps could help you and your doctor decide on the best treatment path for your specific cancer? This animated video explains how identification of unique features of a specific cancer through biomarker testing could impact prognosis, treatment decisions and enable patients to get the best, most personalized cancer care.


If you are viewing this from outside of the US, please be aware that availability of personalized care and therapy may differ in each country. Please consult with your local healthcare provider for more information.


Related Programs:

 

PEN-Powered Activity Guides

Digitally Empowered™


TRANSCRIPT:

Dr. Jones:

Hi! I’m Dr. Jones and I’m an oncologist and researcher. I specialize in the care and treatment of patients with cancer. 

Today we’re going to talk about the steps to accessing personalized care and the best therapy for YOUR specific cancer. And that begins with something called biomarker testing.

Before we start, I want to remind you that this video is intended to help educate cancer patients and their loved ones and shouldn’t be a replacement for advice from your doctor.

Let’s start with the basics–just like no two fingerprints are exactly alike, no two patients’ cancers are exactly the same. For instance, let’s meet Louis and another patient of mine, Ben. They both have the same type of cancer and were diagnosed around the same time–but when looked at up close, their cancers look very different.  And, therefore, should be treated differently.

We can look more closely at the cancer type using biomarker testing, which checks for specific gene mutations, proteins, chromosomal abnormalities and/or other molecular changes that are unique to an individual’s disease.

Sometimes called molecular testing or genomic testing, biomarker testing can be administered in a number of ways, such as via a blood test or biopsy. The way testing is administered will depend on YOUR specific situation.

The results could help your healthcare team understand how your cancer may behave and to help plan treatment. And, it may indicate whether targeted therapy might be right for you. When deciding whether biomarker testing is necessary, your doctor will also take into consideration the stage of your cancer at diagnosis.

Louis:

Right! My biomarker testing results showed that I had a specific gene mutation and that my cancer may respond well to targeted therapy.

Dr. Jones, Can you explain how targeted therapy is different than chemo?

Dr. Jones:

Great question! Over the past several years, research has advanced quickly in developing targeted therapies, which has led to more effective options and better outcomes for patients.

Chemotherapy is still an important tool for cancer treatment, and it works by affecting a cancer cell’s ability to divide and grow. And, since cancer cells typically grow faster than normal cells, chemotherapy is more likely to kill cancer cells.

Targeted therapy, on the other hand, works by blocking specific mutations and preventing cancer cells from growing and dividing.

These newer therapies are currently being used to treat many blood cancers as well as solid tumor cancers.  As you consider treatments, it’s important to have all of the information about your diagnosis, including biomarker testing results, so that you can discuss your treatment options and goals WITH your healthcare team.

Louis:

Exactly–Dr. Jones made me feel that I had a voice in my treatment decision. We discussed things like potential side effects, what the course of treatment looks like and how it may affect my lifestyle.

When meeting with your healthcare team, insist that all of your questions are answered. Remember, this is YOUR life and it’s important that you feel comfortable and included when making care decisions. 

Dr. Jones:

And, if you don’t feel your voice is being heard, it may be time to consider a second—or third—opinion from a doctor who specializes in the type of cancer you have. 

So how can you use this information to access personalized treatment?

First, remember, no two cancers are the same. What might be right for someone else’s cancer may not work for you.

Next! Be sure to ask if biomarker testing is appropriate for your diagnosis. Then, discuss all test results with your provider before making a treatment decision. And ask whether testing will need to be repeated over time to identify additional biomarkers.

Your treatment choice should be a shared decision with your healthcare team. Discuss what your options and treatment goals are with your doctor.

And, last, but not least, it’s important to inquire about whether a targeted therapy, or a clinical trial, might be appropriate for you. Clinical trials may provide access to promising new treatments.

Louis:

All great points, Dr. Jones! We hope you can put this information to work for you. Visit powerfulpatients.org to learn more tips for advocating for yourself.

Dr. Jones:

Thanks for joining us today. 


This program is supported by Blueprint Medicines, and through generous donations from people like you.

Staying Updated on AML Research News: Advice from an Expert

Staying Updated on AML Research News: Advice from an Expert from Patient Empowerment Network on Vimeo.

Dr. Jeffrey Lancet, an AML expert from Moffitt Cancer Center, shares tips for sifting through research news and encourages communication with your healthcare team about what you’ve learned.

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

See More From INSIST! AML

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Navigating AML Treatment Decisions

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

Transcript:

Katherine:      

Well, patients are often educating themselves about developing research and new treatment options. Do you have advice for patients who, when it comes to talking with their doctors about what they’ve learned?

Dr. Lancet:                   

I think it’s important for patients to speak to their doctors directly and as soon as possible as opposed to going on the internet and doing a Google search for this drug or that because every patient’s situation is unique and how to apply these new drugs is very different amongst patients.

And some patients may qualify for certain approaches and others do not. So, it’s very important to talk to your doctor about how you can individualize your treatment based upon your specific scenario. What type of mutation does a patient have, what is their level of fitness, are they potentially candidates for bone marrow transplant? Those are some of the basic questions that come up all the time to determine what is the best treatment approach.

And as we’re developing new therapies, and more of them, there will be more options for patients and a more personalized approach that can be taken that really can only be decided based upon that individual patient’s unique profile. So, it’s very important to really recognize that one size does not fit all when it comes to treatment of this disease and that certain drugs may be helpful and certain drugs may be unhelpful in that particular site.

Katherine:                   

What would you like to leave patients with today? Are you hopeful about the future of AML treatment and research?

Dr. Lancet:                   

Yes, I’m very hopeful. I think AML is a disease that is really a very diverse and complex one. It doesn’t lend itself well to huge immediate breakthrough therapies that will immediately change the landscape by double digit percentages for example. This is a disease that, again is very complex, and in which advances are made slowly but steadily. And I think we’ve seen that over the past to 5 to 10 years is that we are gradually incorporating new drugs into our treatment regimens with gradually increasing levels of success as we learn more about these drugs starting out as single agents and then beginning to combine them.

I think that we’re learning an awful lot about the molecular landscape about AML and how it impacts treatments and treatment decisions and prognoses. I think our ability now to detect what we recall measurable residual disease is very important. Also, because now we can get a grasp of how well our treatments are working and are we knocking out enough bad cells to expect good outcomes, and if we’re not, then hopefully we can intervene and kind of hit it while it’s down so to speak and use some of these new therapies to knock out what might be left over to give patients better overall long term responses and results.

So, definitely reason to be hopeful, but we have to stay patient as well. It’s difficult because it’s a, it’s a terrible disease but we have to recognize that it’s something that requires very careful research to develop the appropriate clinical trials that will have the highest chance of success.

Katherine:                   

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

Dr. Lancet:                   

Thank you very much for having me. It was good to be with you and I appreciate the opportunity.

Katherine:

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

 

 

 

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

See More From INSIST! AML

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Navigating AML Treatment Decisions

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.

 

Advocating for Key AML Testing: Advice From an Expert

Advocating for Key AML Testing: Advice From an Expert from Patient Empowerment Network on Vimeo.

Dr. Hetty Carraway, an AML specialist at Cleveland Clinic, shares advice on advocating for yourself when diagnosed with AML, underscoring the importance of asking questions, and including your caregiver as part of the conversation.

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.

See More From INSIST! AML

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 Treatment Approaches in AML: Key Testing for Personalized Care

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

 Understanding Risk in AML: How Molecular Testing Affects Treatment Options

Transcript:

Katherine:

What advice do you have for patients when it comes to asking for appropriate testing and speaking up in their own care?

Dr. Carraway:            

This is so important. I think patients are leery to stir the pot or be difficult. I think coming from a place of inquiry, teach me about this, that, or the other thing, help me understand this, that, or the other thing – I would like you to show me why this decision or talk with me about why this decision versus another decision might be better for me compared to somebody else.

I can’t underscore the importance of advocating for yourself and asking questions about why am I getting this drug? What are the side effects to this drug? What is my prognosis? What is different about my case versus somebody else’s situation? How do I best prepare myself in getting ready for the therapy that I’m about to go through?

Those are all important questions that patients should ask. They should certainly have people, if possible in their family be advocates for them. I welcome that, and I think that that’s a really important part of going through this type of therapy for any patient. Your physician should welcome having your involvement in that. Don’t be shy about that. It’s your health, and any investment in that the most important people in that is inclusive of you and your caregivers. They should be a welcome part of the team.

Understanding AML Induction and Consolidation Therapy

Understanding AML Induction and Consolidation Therapy from Patient Empowerment Network on Vimeo.

Dr. Hetty Carraway, an AML specialist at Cleveland Clinic, provides an explanation of the role of induction and consolidation therapy in AML patients.

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.

See More From INSIST! AML

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

 Understanding Risk in AML: How Molecular Testing Affects Treatment Options

Transcript:

Katherine:

Would you define induction therapy and consolidation therapy and tell us what the differences are?

Dr. Carraway:            

For most patients that are diagnosed with an acute myeloid leukemia, over the last 30 to 40 years we’ve used an intensive chemotherapy regimen that we call induction. Induction means that we’re trying to get the leukemia into remission with an intensive chemotherapy regimen. Classically, that has been two agents; one, a cytarabine based regimen along with an anthracycline, either idarubicin, danorubicin, or some anthracycline that’s similar.

Now, the cytarabine based therapy is a continuous infusion over seven days. The anthracycline is given over three days as an intravenous IV push, and so that’s why it’s kind of been nicknamed seven and three – seven days of cytarabine and three days of another anthracycline.

Now, that has constituted the induction intensive regimen in the hospital with the idea that that leukemia gets under control and goes away. More recently for patients, they can receive therapy that is not this inpatient, in-hospital, induction chemotherapy but rather use oral therapy combining with venetoclax, which is a Bcl-2 inhibitor, along with azacitidine, which is either IV or subcutaneous given to patients over seven days. The oral, venetoclax is every day.

That type of induction can also be given and is now an outpatient regimen and more often offered to patients that are older, over the age of 75.

That, too can be considered induction with the idea that once a patient is diagnosed with leukemia this regimen is started, and after one month or even two months on venetoclax plus azacitidine patients’ leukemia can get into what we call remission, where the blast percentages are less than 5 percent. Then, normal hematopoiesis of platelets being greater than 100,000 and a neutrophil count greater than 500 or 1,000, and the patient is then transfusion-independent.

In general, induction chemotherapy is that first round of chemotherapy that’s trying to get the leukemia under control.

Consolidation chemotherapy is when you use subsequent cycles of chemotherapy to keep the leukemia under control because we know that if we don’t continue to give some continuation of therapy that the small, little seeds of leukemia will re-emerge and leukemia will relapse.

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.

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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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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.

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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.

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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.