Ask the Expert: Understanding & Managing My AML Diagnosis

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As part of our acute myeloid leukemia (AML) “Ask the Expert” series, patient advocate Carol Preston is joined by leading expert Dr. Gail Roboz, from Weill Cornell Medicine, to answer important questions from the Patient Power community.  Watch the replay of this 30-minute session to learn more about AML genetic research, prognostic factors, treatment options and more.

This program is sponsored by AbbVie, Inc., Genentech, Inc. and Adaptive Biotechnologies. These organizations have no editorial control. It is produced by Patient Power. Patient Power is solely responsible for program content.

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Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

Carol Preston:

Hello, everyone. I’m Carol Preston and welcome to our ongoing discussion and your questions for our Ask the Experts series on AML. Ongoing because our next AML online discussion will happen on October 15th. Please mark your calendars. There also will be lots of news coming out of ASH, the big hematology meeting in early December and of course replays of all of these discussions on Patient Power. Meantime, please email your questions related to AML to aml@patientpower.info. That’s aml@patientpower.info

For those of you who may not know me, I have been living with CLL, chronic lymphocytic leukemia since 2006. But of course today we are focusing on acute myeloid leukemia. Amongst the toughest blood cancers to treat and achieve long-term remission, but things are changing rapidly. A little housekeeping first. First again, we want to thank our sponsors of this program. But again, they in no way have any editorial control and all of that is handled by Patient Power. And please note we always say this too that we can’t provide specific medical advice on this program. It would not be fair to you and we always recommend that you coordinate care with your physician. 

Now, let’s get to AML and our very special guest, Dr. Gail Roboz is professor of medicine and director of the Clinical and Translational Leukemia Programs at Weill Cornell Medicine and New York-Presbyterian Hospital in New York. Dr. Roboz, welcome. So happy to have you here. Before we dive into the questions, can you briefly tell us—now this is a three-parter. What exactly is AML? Let’s start with that one. 

Dr. Roboz:       

So, AML is acute myeloid leukemia. For those of you who are patients and caregivers, you’re going to be confused when you look it up, because it will sometimes say acute myelogenous leukemia. It’s the same thing. It is a disease of the bone marrow—a malignant stem cell disease of the bone marrow. Bone marrow is an important organ. It makes white blood cells that fight infection, red blood cells that carry oxygen and platelets that are little tiny cells that help you clot.

And in AML, for reasons that we usually don’t know and can’t figure out; the bone marrow is taken over by a malignant proliferation of what we call leukemic blasts, which are cells that don’t grow up and do what they’re supposed to do, and instead they crowd out the other ones, prevent the normal functioning of the bone marrow and give the disease called AML.

Carol Preston:

So, it’s one of several blood cancers. I mentioned that I have CLL, the most common and very treatable in the last few years. Why has it been so tough for truly effective and long-lasting treatment in the past 40 years for AML?

Dr. Roboz:       

So, AML affects all ages. There’s AML in children and there’s AML going to age 100. But the median age at diagnosis is in the late 60s, 67 to about 70 years old. And it’s a biologically and molecularly very heterogeneous disease. So, what I say to patients in the waiting room is that if you’re sitting next to someone with AML; it’s probably not anywhere near the same as yours even though the three letters are the same. So, it’s very important to understand that AML is a name, but it actually encompasses a lot of biologically different diseases and the treatment has been very difficult.

The conventional chemotherapy treatments have historically been very difficult for older patients with medical co-morbidities to handle. That’s part of the problem. But things, as you said, are getting better. 

Carol Preston:

So, the good news is that since 2018 and the last couple of years a revolution of sorts. We saw it in CLL about three or four years ago. Multiple myeloma a couple of years, so it seems that scientists are starting to crack the code.

Dr. Roboz:       

Moving in the right direction. You know I have to be careful in answering this because of course, there are significant advances and we are very thrilled about them, but I’m always mindful having come back where there a lot of people still with AML as a very difficult disease to treat that isn’t being helped by current therapies. So, my measured answer is that we are excited and encouraged, and the improvements are definitely significant. We’re going in the right direction, but it’s not cured yet. I still have a job.

Carol Preston:

And you will for a very very long time, and we’re very grateful and appreciative of the work you and your fellow scientists and physicians do well. There are lots of people who have questions on lots of different subjects so let’s get to it. Ann has a question about AML subtype, and she would like to know what testing would she and others need to determine the AML subtype. AS you mentioned, those three letters can stand for many different things.

Dr. Roboz:       

That is a great question and a perfect one to open with because what type of AML do you have. So, patients get immediately confused by going into the literature and looking at something called FAB or French American British subtyping and the often will come in and say, “Do I have M2? Do I have M4?” 

This is kind of old news. This is based on what the cells look like under the microscope. And although interesting, it’s not really what we mean by when we say subtype currently. What you want to be asking your doctor about is cytogenetics for chromosomes and molecular genetics. And molecular genetics refer to mutations that may or may not be present as part of your disease. So, it’s very important to know what chromosome abnormalities and what mutational abnormalities you have, because together that information puts together not only information about prognosis but also potentially information about treatment that may or may not be specific to a subtype that you have.

Also, a PL or acute promyelocytic leukemia is different from all the other subtypes of AML. Different treatment, different disease. So, one of the first questions that you want to know is do I have a PL or do I have regular if you will AML. And then within that, you want to ask for chromosomes and mutations and they are not the same. 

Carolyn Preston:         

So, this is where I always like to put a plug in for making sure either you bring a tape recording, bring paper and pencil, and more important than anything else, bring a friend, a family member to listen. As you know from your many years in practice, people hear the word cancer, they can’t hear anything else. So, for those of you if this is new, if you haven’t before had a friend or a family member accompany you to an appointment, there are a lot of terms here that are going to go in one ear and out the other. And this way if you have someone else hearing them and you’ve written it down or recorded it; it will be a lot easier when you go back to refer. So, I just like to put that plug in because been there done that. 

Dr. Roboz:       

I totally, totally understand that. I will also share from the doctor’s side. I like it better, and I think it’s useful to share with the doc if you like to be recording, because I think sometimes I think patients worry about asking the doctors, and they’re trying to record secretly. And it almost doesn’t work as well as just let us know if you want to record it, because then we can repeat something or clarify it or make sure that it’s serving a good purpose on the recording.

Carol Preston:

Yeah, and when you go back if you’ve gotten the okay and you go back and listen; it’s probably going to generate more questions so great advice and very interesting about the breakdown of the AML. So, not straying too far from that, Mark wanted to know about genetic mutations and what mutations are you looking for and I know from my experience with CLL, mutated was better than unmutated. I don’t know if the same thing applies but what mutations are you looking for?

Dr. Roboz:       

Very different. So, CLL, I have to say it’s a great point to bring up that leukemia, everybody knows that it’s a scary word. But if you talk CLL versus CML versus AML versus ALL, there are lots of different letters going on and it is not all the same thing. So, mutated versus unmutated in CLL is completely different from what we’re talking about in AML. So, there’s been an arms race to sort of figure out well, how many genes can I get onto my panel? And the academic centers will have 50 gene panels or 100 gene panels or 500 gene panels and the revolution in next generation sequencing has resulted in the ability to detect lots of different things that mostly we don’t know what to do with yet.

What you do want to make sure of though is a myeloid mutation panel should be sent, and what you’re specifically looking for initially is a collection of mutations about which we do have some treatments and prognostic information. Things for example like Flip 2, FLT 3, or CEBPA, or IDH1 and 2, or NPM1—this is an alphabet soup for patients to listen to at the moment. But trust me when I tell you that if you look at various patient advocacy groups for example in AML—I have a pan on right now that says know AML, K-N-O-W. What they want you to do is ask the question from the doc, “Hey, what mutations do I have, and which one of these do you know something about? Does it matter for my prognosis? Does it matter for my treatment? Does it matter for both?”

That I think is a very useful conversation as opposed to getting into the weeds on 500 different mutations that we actually don’t know much about yet. There are plenty.

Carol Preston:

Yeah, and so that really slides into Ann’s question who says, “What do I need to know to get educated on my treatment decisions?” You mentioned the alphabet soup of acronyms or initializations and patients have enough to deal with without trying to remember all of that. So, is there a primer as it where for patients?

Dr. Roboz:       

No, I mean I think that the information on the internet is so overwhelming and vast that it is very, very difficult candidly to direct people well, because there’s so much changing information and somehow with this disease; almost invariably in my experience patients are led down a very grim road somehow. I don’t know what it is about the searches but they end up with a lot more scary information at the beginning of the diagnosis than what they should be thinking of. It’s drinking out of a fire hose, and I think it’s very not helpful to immediately go down the road of the worst possible prognostic subtype that you may not have.

Start out by figuring out if you’re in a center that treats a lot of AML or not and then try to establish with your doctor that maybe if you’re acutely ill and if you’re in a small hospital that doesn’t work with a lot of AML, maybe the doctor there could collaborate with a bigger center that treats a lot of AML. Figure out that the right testing has been sent, both cytogenetically and in terms of mutations, and then break it down for you so that what you’re looking up on the internet is what you have. Not what’s theoretically possible for a million patients on Earth, because you want to focus on you? You don’t need to learn about everybody else’s AML.

Carol Preston:

Exactly. Exactly. Yeah, the Internet can be a friend or a foe as you mentioned. And a lot of times people hear the C word and they feel that they have to get the equivalent of a medical degree in about two weeks, and that really is just not the case. So, I urge everybody to please just take a breath and proceed as Dr. Roboz suggests. And then you touched on this a little bit when you mentioned the revolution as it were undergoing since 2018. So, what treatments are available for newly diagnosed AML patients? And again, that’s a very broad-based question because of the subtypes. But tell us about some of the new treatments that you’re hopeful or excited about.

Dr. Roboz:       

So, huge question. Huge. It covers a lot of…

Carol Preston:

…well, let’s break it down and maybe just mention a couple right now.

Dr. Roboz:       

So, conceptually making it very easy. What used to be the case was a combination regiment affectionately known as 7 and 3. 7 days of one drug and 3 days of the other. Cytarabine (Cytosar) and a drug called daunorubicin (Cerubidine) or idarubicin (Idamycin) has been the standard of care for newly diagnosed patients in good shape to handle intensive chemotherapy since the late 1950s. Currently, that regimen can actually be built on by the addition of newer things which might be applicable for some patients. 

For example, 7 plus 3 can be combined with a FLIP 3 inhibitor if you actually have a FLIP 3 mutation. Some patients even today in 2019 will be treated with 7 and 3 and I have to say, more patients have been cured with standard chemotherapy than anything else with AML. So, don’t be put off by the fact that it’s old because it doesn’t mean that it doesn’t work. There are many patients who boast newly enter remission with a regimen that was developed actually before I was born. 

Now, that said what we’re trying to do especially for older and more frail patients is not deploy that particular combination which is often not possible for patients well into their late 70s, 80s, and beyond. And there had historically been low-intensity regimens. Things like azacitidine (Vidaaza), decitabine (Dacogen) and low-dose cytarabine have typically been used. Safer for patients but much lower rates of remission. For older patients who fall into the category of not getting intensively treated, the addition of a drug called benataclax which Carol, you will know a lot about from CLL world. That drug was originally approved in CLL. 

It has excellent activity in AML in combination with azacitidine, discytobine, or low-dose cytarabine that is pushing the complete remission possibilities for older patients, much higher than what we’ve had before. In addition to that, there’s a lot going on with trying to add on specific inhibitors for IDH1, IDH2 and FLIP 3 mutations to learn how to mix and match those novel agents with existing backbones so that patients can be treated with the highest remission and response rates possible. Within all of that though, you’ve got to ask what’s the right one for you, and there are lots of important factors that go into the making of the decision what the choice is but we have a choice now and we haven’t for many years.

In AML, we kind of had two options: less intensive and more intensive and that was the end of the story. Things are better now.

Carol Preston:

So, that is a huge takeaway for those of you listening and watching. There are choices today that did not exist two years ago. The other takeaway that I’m hearing given the level of detail that a specialist like Dr. Roboz can go into is if you possibly can, at least one consultation with somebody who specializes in this disease. If you’re in a regional or a community hospital, see if you can get a referral or visit a major medical center that’s close to you where there is an AML specialist. There’s just so much happening in the field right now. It’s tough for your very fine community oncologists to keep up with the 10, 12 or 15 types of cancers that he may be treating.

So, again lots of choices, second opinion if possible. Let’s talk a little bit about daily life because whenever anybody is going through cancer, there is always that question of what should I do? What can I do? So, big question. How will treatment affect my daily life? Never mind the AML but from Mary, “Will I be able to work? Can I exercise?”

Dr. Roboz:       

So, AML is separated into getting into remission and then what do you do after remission. So, remission is a definition. Remission means you’re reducing the percentage of abnormal leukemia blast cells in the bone marrow to less than five percent and you have normalization of your blood counts to 100,000 platelets and to a neutrophil or infection-fighting white blood cell count of 1,000. Depending on the regimen that you’re getting, most of the time it takes three, four, five weeks to get into remission. And during that period of time, you’re either in the hospital or you’re back and forth very frequently sometimes three times a week to the outpatient office and you are a full-time patient.

Generally, blood counts are low. You’re getting transfusions. You’re getting antibiotics. You might be doing this as an outpatient if you live really close to a center that has the ability to transfuse and see you frequently but otherwise, it’s an inpatient disease. So, that first month what I like to tell patients you’re kind of hostage. You’re not necessarily in the hospital but you are a professional patient. If you get a fever, you’re rushing into the hospital. So, doing things that are normal life kind of things is really hard during that first period of time. Now, the goal is to get you into remission. 

Remission is better. Blood counts are normal. You don’t have to be seen so frequently. How we keep you in remission is either a stem cell transplant or bone marrow transplant or more chemotherapy. So again, the impact to your daily life depends on what we’re going to be treating you with. But AML and the intensive treatment phases is a very difficult disease to kind of live life normally. Coming in, getting a quick chemo, and going back to work definitely in the acute treatment phase is going to be very difficult if not impossible. 

Carol Preston:

Let me ask you this. There’s been so much in the news lately, mainstream news, as well as medical news about MRD, which is minimal residual—I’m blanking. 

Dr. Roboz:       

Minimal residual disease.

Carol Preston:

Yeah, and the question is I actually had a lot of questions about that, minimal residual disease. First of all, someone with AML, when does that testing come into play? And number two, could a patient have MRD not negative meaning no detectable cells and still have a good quality of life or is treatment an absolute must?

Dr. Roboz:       

Okay. So, there’s a lot in there. Let me dial back for a second. The terminology originally started as minimal residual disease, MRD but there’s been a strong movement across the hematological malignancies to change that to measurable residual disease. And the reason is not a trivial one. Doctors and patients can blow off something that’s minimal. “Ah, it’s minimal. It’s not that important.” Measurable residual disease comes into play very strongly because we know that if you use standard looking under the microscope criteria for patients with AML; there are a lot of patients who will have achieved complete remission, normal blood counts, feel good, going to work but who actually relapse.

How is that possible if everything is good? If the counts are normal and you feel good and everything is fine why are you relapsing? The reason is because looking under the microscope is simply not good enough. It is not this century’s technology for looking for evidence of disease that can come back. And for those of us who live in New York, it’s the cockroach analogy. The roaches are meant and designed to stay alive. They’re good at it. They wiggle out from under chemotherapy and they are buried deeply enough into the bone marrow that you can’t see them under the microscope.

So, this is a controversial area in which both next-generation sequencing, flow cytometry and cytogenetic testing are being used to detect evidence of residual disease that is lower than what is seen under the microscope and to make treatment decisions on that basis. MRD is definitely a reason why being seen at an academic center, especially if you’re considering undergoing stem cell transplant which is always done at a major center is important because if you’re detecting residual disease; you may have a great quality of life. You may be running around feeling very good but your chances of relapsing are very high. And the decision tree has to be that, “Well, is a transplant possible? Is there more treatment that is possible that could eradicate that level of residual disease?” And these are complicated decisions that really require a center with some expertise in the whole area of MRD and how to measure it. 

Carol Preston:

Yeah. So, again the takeaway lots of choices. Yeah. If you can even to get one outside consult and then have the specialist who are always happy – listen. I know from experience, specialists don’t want to see every patient under the sun. They want to work with the community oncologists to make sure that you get the right diagnosis and the right treatment. 

Dr. Roboz:       

Carol, let me jump in on that for one second, because you bring up such an important point about the second opinions. I want to just drill into one thing. AML is sometimes a disease that must, must be managed acutely and immediately and I think that there are scenarios in which patients are dangerously ill with AML, were transferred to another center or moving to another center would actually be the wrong thing to do in which case it is a great idea for family members to say, “Listen, can you call someone? Can you reach out?” And I can personally say I get calls at all hours of the night from all over the place saying, “Can I talk to you about a patient?”

So, the hope is that the doc that you’re working with recognizes that you’re really, really sick, and is checking in to see if there’s something that we can do to manage you safely where you are. Don’t run away from a center if the doctors say you’re too critically ill to move because that could be a life-threatening mistake. That said AML is not always an emergency. In fact, it usually isn’t and there are plenty of patients with low blood counts feeling pretty good who do have an opportunity to think about the diagnosis. And we say all the time, “We want to get things right. We don’t want to necessarily get it fast.” 

So, if you have a minute to think if there’s not an acute emergency, your blood counts are low, but you’ve been feeling fine. You were playing golf yesterday, you might have quite a bit of time actually to get your ducks in order, look at the data, get a couple of opinions either by phone or by a video or by checking in with experts via your doctor. You probably have a minute if there’s nothing acute going on to get that additional information.

Carol Preston:

Exactly. And that’s why we encourage people to take a breath especially if you’re feeling all right but encourage your community oncologist to get in touch with someone like Dr. Roboz. It doesn’t have to be her but somebody who is an AML specialist at a major medical center. Believe it or not, we’re almost out of time. This half-hour is just flying by. So, I do want to end with this question briefly if possible and that is from Rose. At what point would a bone marrow or stem cell transplant be the treatment or necessary treatment?

Dr. Roboz:       

Super important. Very basic points. Number one, you have to figure out what are your chances of getting cured with chemo alone versus getting a transplant and the second thing that patients don’t realize is you want to be in remission before getting a transplant. That’s why transplant as an upfront treatment is not the—that’s not done. So, you first get into remission and you ask your doctor, “Can this disease be cured actually with chemotherapy or would I be eligible for a bone marrow transplant?” And that’s when that discussion happens preferably by the way in MRD negativity. So, you’d like to be in remission with no residual disease and then get a transplant.

Carol Preston:

Great advice. And as I said, we hope you’ll come back, Dr. Roboz because our time is up so we want to thank you profusely, Dr. Gail Roboz of Weill Cornell Medicine and New York-Presbyterian Hospital not only for your time and your expertise but the hope that you have offered today and will continue to offer AML patients. If you have more questions ladies and gentleman, a reminder that the AML conversation continues next month on October 15th with another webinar. ASH is coming up at the end of December where AML will loom large in presentations and posters and Patient Power will bring it all to you at patientpower.info so please stay tuned and stay online. I’m Carol Preston and in the matter of your health, never stop asking questions. 

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

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Page last updated on October 1, 2019