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Follicular Lymphoma Patient Expert Q&A: Dr. Brad Kahl

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Dr. Brad Kahl from Washington University School of Medicine explores the transformative potential of emerging therapies for follicular lymphoma and their significance for patients and families. He also addresses the unique challenges of living with follicular lymphoma and its impact on patients’ lives today.

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Related Resources:

What’s the News on Follicular Lymphoma and Bispecific Antibodies

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 What Can Follicular Lymphoma Patients Expect With Remission

Transcript:

Lisa Hatfield:

Welcome to this START HERE Patient Empowerment Network program. This program bridges the expert and patient voice, enabling patients and care partners to feel comfortable asking questions of their health care team. Joining me today is hematologist-oncologist Dr. Brad Kahl, Professor of Medicine in the Division of Oncology at the Washington University School of Medicine and Director of the lymphoma program at the Alvin J. Siteman Cancer Center in St. Louis, Missouri. Thank you so much for joining us, Dr. Kahl.

Dr. Brad Kahl:

It’s a pleasure. Thanks for having me, Lisa.

Lisa Hatfield:  

The world is complicated, but understanding your follicular lymphoma diagnosis and treatment options along your journey doesn’t have to be. The goal of Start Here is to create actionable pathways for getting the most out of your follicular lymphoma treatment and survivorship. No matter where you are on your journey, this program is designed to provide easy to understand, reliable, and digestible information to help you make informed decisions. And most of all, we’re asking questions from you. I’m thrilled you’ve joined us.

Please remember to download the program resource guide via the QR code. There is great information there that will be useful during this program and after. Let’s start here. Dr. Kahl, there is a great deal going on in the follicular lymphoma landscape, and I want to dig into that. But before we do, as is custom for this program, I’d like to start with a brief overview of this disease. What is follicular lymphoma? And can you break it down a little bit, the key differences between Hodgkin and non-Hodgkin lymphoma and how follicular lymphoma fits into that?

Dr. Brad Kahl:

Sure. The terminology can be kind of confusing to patients, so I’ll try to explain it. Hodgkin lymphoma is a specific kind of lymphoma. Non-Hodgkin’s lymphoma just means it’s not Hodgkin’s. So non-Hodgkin’s lymphoma is just a big, broad, descriptive term. It’s like saying automobile. But there are lots of different kinds of cars, obviously. So follicular lymphoma is a specific type of non-Hodgkin’s lymphoma. So it’d be like saying Chevy Malibu or something specific within that automobile term. So there’s like 100 different kinds of non-Hodgkin lymphoma. Follicular lymphoma is one of those. A

nd it’s kind of a unique answer biologically and clinically. Follicular lymphoma is characterized by this particular mutation inside the cells that sends a signal to the cells that says don’t die. So instead of being a disease of rapid cellular proliferation and growth, it’s more of a disease of slow cellular accumulation. If people can picture that, the cells are just accumulating slowly. So it’s kind of a slow-moving cancer. And probably when patients are diagnosed, they’ve probably had it for a long time already.

They just didn’t know it, because follicular lymphoma often doesn’t cause symptoms. And usually when we get a patient with newly diagnosed follicular lymphoma, the disease is very widespread. And that obviously makes people fearful. And so we spend a lot of time trying to reassure them that’s not a problem that’s typical for follicular lymphoma. Everybody wants to know their stage, of course. And I try to tell them, the stage doesn’t really matter that much in follicular lymphoma. In some cancers, the stage is a big deal. But those are cancers that you can kind of remove surgically.

But there’s really no role for surgery as a treatment in follicular lymphoma. The disease is typically very widespread in diagnosis, meaning it’s all over the body. And so when we do treat it, we pick treatments that will work everywhere. And our treatments tend to work just as well when the disease is at a more advanced stage. That’s why as the doctors, we don’t spend too much time worrying about the stage. It’s just not, it’s not as important in follicular lymphoma.

Lisa Hatfield:

Okay. Thank you. And just to clarify, when you mentioned that there is a mutation or often mutations in follicular lymphoma, is that in the cancer cells themselves, or is that in a mutation, like a BRCA mutation that a patient can be tested for? I presume it is.

Dr. Brad Kahl:

Right. That’s a great question. The mutation is specific to the cancer cells. So people are not born with this mutation. It’s not a mutation that you pass along in your family to children. It’s a mutation that is acquired in these cells at some point in the patient’s lifetime. Another confusing term is this whole idea of B-cell lymphoma or T-cell lymphoma.

And just to try to clarify that. So we have different kinds of lymphocytes in our body, and these lymphocytes, they have jobs to do as part of our immune system. And one kind of lymphocyte is a T cell, and that has specific roles in our immune system. And another kind is a B cell, and that has specific jobs to do in our immune system. Follicular lymphoma is derived from a B cell, a B-cell lymphocyte. So the…a B cell gets this mutation, and that turns it from a normal healthy B cell into a follicular lymphoma cell.

Lisa Hatfield:  

Okay. Thank you for explaining that and for that overview. That’s really helpful. I appreciate that. So, Dr. Kahl, you also mentioned treatments and how oftentimes it’s not a cancer where you can just remove the cancer. Can you talk about some of the exciting developments with treatments and new innovative therapies, and what are the most important highlights for patients and families?

Dr. Brad Kahl:

Yeah. There’s a lot to talk about here. So I’ll start with how we approach a newly diagnosed patient, and then we’ll go into how we approach patients who have relapsed disease. So the most often, or the most common way a follicular lymphoma patient comes to medical attention is they just either notice a lump from an enlarging lymph node, or some enlarged lymph nodes are just found incidentally because they’re having some testing for some other condition.

And so, like I said, very often patients don’t have symptoms. That’s very typical. Occasionally, the patients will have symptoms, and those symptoms might be pain from a large lymph node mass that’s pushing on something. Occasionally, they might have fevers or night sweats. They wake up in the middle of the night just drenching wet, or unexplained weight loss. Those would be symptoms that can occur in follicular lymphoma. But most patients who come to see us for the first time don’t have symptoms.

When we have a newly diagnosed patient and it takes a biopsy to make the diagnosis, we then need to do the staging evaluation. So that involves some sort of imaging. And nowadays that’s usually in the form of what’s called a PET scan, which gives us a good snapshot of the whole body. And it’ll show us enlarged lymph nodes. And then the PET portion of the scan will show us if the lymph nodes are metabolically more active.

So they show up as these bright spots on the PET scan. And that’s what allows us to stage the patient. It tells us where the disease is located and how much of the disease we see. And so I’m often telling patients, I don’t worry so much about the stage. I worry more about the disease burden. So the way I explain that to patient is, suppose I could take all the follicular lymphoma cells out of your body, and I made a pile. How big is the pile? And that’s actually, I think, more important than the stage in determining our initial strategy.

Because believe it or not, if we have a patient who comes to us with a new diagnosis of follicular lymphoma and they have no symptoms, and it turns out that their tumor burden is very low, we often will recommend an initial approach of no treatment, which is a strange thing for patients to hear. And we spend a lot of time trying to explain the rationale for that. So I’ll try to explain that to you now. Follicular lymphoma is hard to cure.

So it’s this weird cancer in that it’s slow-moving. It often doesn’t make people sick, and we have good treatments for it, but curing it, like making it go away once and for all, proves to be kind of difficult. And studies in the past have shown if you have a patient who has no symptoms and is low tumor burden, that their prognosis is just as good if you leave them alone at the beginning. And many patients will not need any treatment at all for two years, three years, five years. I even have follicular lymphoma patients who I’ve been observing for more than 10 years that have never needed any treatment.

About two out of every 10 patients that are newly diagnosed can go 10 years without needing any treatment. So that’s why we’ll start that strategy for some patients. And that’s psychologically can be difficult for patients. You’re telling me I’ve got a new cancer diagnosis. You’re saying you have good treatments for it. And yet you’re saying you don’t want to use any of those treatments. And so it takes a lot of talking and explaining to try to get people comfortable with that.

Some people never get comfortable with that, I admit it. But some people get very comfortable with it. But it is a very appropriate initial strategy for a low tumor burden asymptomatic person just to observe and get a handle on the pace of the disease. If the disease starts to grow, or if the patient starts to get symptoms, we can start our treatment at that time. And the treatment is going to work just as well as it would have had if we started it last year, or two years ago.

So we feel like we’re putting the patient in no harm, no risk of harm by starting on this strategy of a watch and wait. On the other hand, some patients have high tumor burden, they have a lot of disease, or they have symptoms. And for those patients we need to start them on treatment because the treatment can put them in remission and get them feeling better. Right now, the most common frontline treatment in follicular lymphoma will be a combination of some chemotherapy and some immunotherapy.

The most commonly used regimen in the United States right now is a two drug regimen, a chemotherapy drug called bendamustine (Treanda), and an immunotherapy drug called rituximab (Rituxan). And you give that treatment every 28 days for six months. And it’ll put 90 percent of people into remission. And on average, those remissions last five plus years. And it’s a very, very tolerable treatment.  It’s not too bad as far as chemotherapy goes. There’s no, most people don’t lose their hair. They don’t get peripheral neuropathy, that sometimes chemotherapy drugs give.

It’s not too bad for nausea and things like that. I’m not saying it’s easy or it’s fun. It’s none of that. But as far as chemo goes, it’s not too bad. And it’s effective, it is very effective. And I’ve given that treatment and I have people who are still in their first remission 10 years later, so you can get, for some people can get these really long remissions. But the reality is most patients, their disease does come back, they do relapse at some point. And then we have to start talking about what to do for second line treatment or third-line treatments.

And that’s where things have really taken off in follicular lymphoma in the last few years, there are a number of brand new treatment options in play for relapsed follicular lymphoma that are very exciting, and proves that we’re moving away from chemotherapy. We have drugs that are oral, that are, we call them targeted agents, they hit like a molecular pathway inside the cell a lot, and they kill the cells a lot differently than chemotherapy does. And we have a number of new drugs that work through the immune system, and try to attack the lymphoma that way.

So when we have patients who relapse, probably the most commonly used second-line treatment right now is a combination of a drug called lenalidomide (Revlimid), which is a pill that’s used in a few different cancers. It works very well for certain cancers, and it works well in follicular lymphoma. And that’s given with the immunotherapy drug called rituximab. And that was proven in a study to be very effective. About 80 percent of people will respond to the regimen, and that remission on average lasts in the two to three-year range.

So that’s probably the most commonly used second line regimen right now in the U.S. for follicular lymphoma. And then there are a number of treatments that are now available in third-line and beyond that are new within the past, say three, four years. And these newer treatments that I’m about to describe are now being tested as second line treatments and even as first-line treatments.

So it’s possible that some of these treatments I’m about to describe will become in the future, our go to regimens for first line treatment or second line treatment. And we hope they do move up, because that means they’re, it means they’re even better than what we’ve been using. So probably the treatments that we’re most excited about right now in follicular lymphoma are the drugs called bispecific monoclonal antibodies. There are two that are now FDA-approved. One’s called mosunetuzumab-axgb (Lunsumio), and that was approved about a year-and-a-half ago.

And the other one’s called epcoritamab-bysp (Epkinly), and that was approved just a month ago. And basically these drugs are infused or injected under the skin, infused intravenously injected under the skin and their proteins that will literally stick to the lymphoma cells. And when it does that, it kind of coats the cancer cells. And then after these bispecific antibodies coat the tumor cells, they literally will trick the patient’s T cells or healthy T cells to come in and attack the cancer.

So it’s a way of trying to trick the patient’s own immune system to come in and start fighting the cancer. And these two drugs are very promising in the relapse setting. They work about 80 percent of the time to get some kind of response. About 60 percent of the time patients will go into complete remission, which means we can’t find any evidence for the lymphoma on scans. And they’re both so new that I don’t think we have a full understanding of how durable these remissions are going to be right now.

It looks that like about, if you do get a complete remission, that about half of those patients are holding that complete remission at two and three years. But we’re, we don’t know about four years and five years yet because the drugs are too new. And we expect that if, as these drugs move up and are tested in the second-line setting and in the first-line setting, they’ll work even better because the cancer cells tend to be easier to kill in earlier lines of therapy. Other agents that have moved into the relapse follicular lymphoma space would include CAR T-cell therapy.

This is a fairly sophisticated complicated approach where you actually will run the patient’s blood through apheresis machine and you will extract the patient’s T cells and those T cells get genetically modified in a lab and then expanded and then are shipped back to the center and then re-infused back into the patient. So now again, we’re tricking the patient’s T cells into fighting their B-cell lymphoma.

And there are three CAR T products that are now FDA approved for use in follicular lymphoma, and they have very high response rates. With seemingly good durability we’re now getting three and four-year follow-up for these CAR T products with about half of people still in remission. The CAR T products probably have a little more toxicity and a little more risk than the bispecifics. So I think most of us are thinking we would try the bispecifics before CAR T, but there might be certain patients where a CAR T strategy is more appropriate to use before a bispecific.

So we’re very excited to have these tools in our toolbox. It’s always good to have more options. And then I should just mention the small molecule inhibitors. So here’s an example. Just this past year there was approval for a small molecule called zanubrutinib (Brukinsa). It targets an enzyme called BTK or Bruton’s tyrosine kinase. This is a pill really well tolerated. It’s given in a combination with an immunotherapy drug called obinutuzumab (Gazyva). This zanubrutinib-obinutuzumab combination got FDA-approved just this year for recurrent follicular lymphoma.

The results look very good for that. It’s very well-tolerated. There’s another oral agent called tazemetostat (Tazverik), which was approved a couple of years ago. It targets a mutated protein in follicular lymphoma. This is, again, is a pill super well-tolerated, very few side effects. So, there’s just a few examples for you of all the different treatment options we have for follicular lymphoma that has recurred after initial treatment.

And believe it or not, the decision-making can be difficult when you have so many choices and so many good choices, that’s a good problem to have. And I find myself a lot of times spending a lot of time with the patient and their family as we talk through these different options, and we try to think what’s best for them at this point in time, talking through the pros and the cons, how active it is, what side effects do we need to be concerned about. And it’s a lot for patients to digest when you have so many choices. But like I mentioned that’s actually a good problem to have.

Lisa Hatfield:

I think you’re right. There’s a lot of hope in those options. I do have two follow-up questions. One of them is when you talk about lenalidomide or brand name Revlimid, CAR T bispecific antibodies, this new small molecule, are these all quality of life is so important for cancer patients. Are these all limited duration treatments for recurrent disease when there’s a recurrence of the disease or are they long-term treatments for the disease?

Dr. Brad Kahl:

Yeah, really good question. And the answer is different for every agent. So I’ll try to just kind of run through the list. For the CAR T products, the three different CAR T products, it’s like a one-time treatment and then you’re done because the cells that get infused will persist in the patient’s body for months and months and months. So they’re infused and then the cells will hang around a long time acting on the cancer. So for the CAR T it’s a one-time treatment. For the bispecifics, the mosunetuzumab-axgb product is a time-limited treatment that is done in less than a year. The epcoritamab-bysp is designed to be given indefinitely.

So those are, there are some pros and cons of those two agents, the two small molecules that I mentioned, the zanubrutinib is meant to be given indefinitely and the tazemetostat is meant to be given indefinitely. And then the first one I mentioned was the lenalidomide. That is in follicular lymphoma that it was developed to be given for 12 months in this setting. So the duration of therapy is unique for each of the different agents that I mentioned.

Lisa Hatfield:

Okay. Thank you for that overview of all those emerging therapies. That’s great to know for patients, Dr. Kahl. All right. It’s that time where we answer questions we’ve received from you. Remember, as patients, we should always feel empowered to ask our healthcare providers any and all questions we might have about our treatment, our disease, and our prognosis. Please remember, however, this program is not a substitute for medical care. Always consult with your own medical team.

So, Dr. Kahl, we have several patients who have submitted some questions. The first question is regarding emerging technologies. And I think that you probably have answered that very well actually in a discussion here. So the second question this patient had is how might future innovations build on the latest treatments to offer even better outcomes for patients? You, I think maybe have touched on that, but maybe speak to that a little bit more as far as longer remissions. Yeah.

Dr. Brad Kahl:

Right, right. So I think right now the main emphasis in research is to take some of these really promising drugs that were developed for relapsed follicular lymphoma and do two things with them, test them in combinations in the relapse setting to see if you can make them even more active. So an example of that would be take the drug lenalidomide, which is really active in the relapse setting and pair it with the drug mosunetuzumab-axgb, which is very active in the relapse setting, and pair them together and see if you can get better results than either drug alone.

So there are studies trying to answer questions like that at this time. And then the other area of major interest is to take these promising new treatments approved in the relapse setting and test them upfront. So there are studies being literally designed right now as we speak that will test bispecific monoclonal antibodies in the frontline setting.

So patients can envision being offered a chance to have a chemo-free strategy where they’re just getting a bispecific monoclonal antibody as their initial treatment. And there are studies that will test these drugs as single agents, and there are studies that will test these drugs in combinations with other agents in the frontline setting, like lenalidomide, for example. So we have no results from any of these trials yet, but these trials are just starting to enroll patients and this could fundamentally change the way we’re managing follicular lymphoma in the future if any of these new strategies turn out to be more promising than what we have done historically.

Lisa Hatfield:

Thank you. Okay. Another question, Dr. Kahl. How do outcomes differ for patients with relapsed/refractory disease compared to those who respond well to initial treatment?

Dr. Brad Kahl:

So that’s a really good question. And when we have a patient going through frontline treatment, we’re all really crossing our fingers that that first remission is incredibly durable. Because when the disease relapses, the remissions do tend to get shorter and shorter and shorter, which is frustrating for everybody.And so we love it when we get a nice long first remission. And in the older days when all we had to offer was chemotherapy and some different immunochemotherapy regimens, the remissions in second line and third line might be two years or one year.  It can get frustrating as you go through treatment after treatment after treatment. It’s hard on patients. The side effects start to accumulate. And that’s one of the reasons we’re so excited about all these new agents that we have for relapsed disease with the bispecifics and the CAR T products and the small molecule inhibitors like tazemetostat and zanubrutinib. Because it appears as though these remissions for relapsed disease might be getting longer than what we have seen historically. So there’s no question that dealing with relapsed follicular lymphoma is more difficult than dealing with frontline follicular lymphoma. But we’re optimistic that these newer treatments we have are improving outcomes for patients with relapsed disease.

Lisa Hatfield:

Okay. Thank you. And another question, which patients are considered the most vulnerable when it comes to follicular lymphoma and why, and what measures can be taken to better support these populations in terms of treatment and care? And I’m not sure if they’re talking about different age groups or ethnic groups or geographic groups like rural versus more urban areas, but if you can speak maybe to general terms to answer that question, that would be great.

Dr. Brad Kahl:

Yeah, right. Well, the first thing that comes to mind are older patients. Older patients are always more challenging to take through cancer therapies. The older patients are more fragile. They don’t tolerate the treatments quite as well. They don’t have the physiologic reserve. They’re more susceptible to complications and infections. So I always think when we have older patients that need treatment in follicular lymphoma, the doctor has to be extra, extra careful, sort of the Goldilocks principle. You don’t want the treatment too hot and you don’t want it too cold, too hot, it might work great, but you might get unacceptable side effects too cold, maybe no side effects, but not enough activity against the disease. So we’re always trying to get that patient the best remission we can get them, but doing the least amount of harm along the way.

So I think that takes a little bit of art, a little bit of experience to figure out how to get your older more fragile patients through follicular lymphoma therapy. And then I think the whole idea of patients who live in rural areas, that can often be challenging too, because they may be hours and hours away from medical care. So if they do have a complication of treatment, an infection, for example, it can be challenging to get them the care they need in a quick amount of time. So when I have patients who I know live way out in the country, far away from our center, I just, we always give them a card, it’s got our phone number and I’m like, you feel like something’s going wrong, call us. I don’t care if it’s 2 in the morning, you call us.

It’s not your job to figure out what’s going wrong. That’s our job. It’s just your job to describe to us what you’re experiencing and then we’ll figure out over the phone whether we want you to drive the three hours to come see us or whether we think you just need to go to the closest place, which might be 30 minutes away. So at least you’re in the hands of some medical professionals. And then they can call us with an update on what they’re noticing, what the tests are saying. So taking care of patients who live far away from the medical center poses some additional challenges.

Lisa Hatfield:

Okay. Thank you. And that’s a great takeaway for patients. If you have a question, call your provider. They can help take the stress away from making that decision yourself. 

Well, here’s a loaded question for you, Dr. Kahl. Why does relapse happen in the first place, and what are the changes in the body that signal when and if treatment is likely going to fail?

Dr. Brad Kahl:

Boy, we wish we understood why relapse happens in the first place. Last I mentioned, most of these treatments can get people into remission, which means that they can kill the vast majority of the cancer cells, maybe 99.9 percent of them, but for some patients, there’s just a few stubborn cells that remain behind. Maybe those cells are just sitting there, not growing at all, which follicular lymphoma cells can do.

And when the cells are not trying to divide, not trying to grow, they’re kind of protected from killing. They’re just sitting there doing nothing. And so we think it’s this property that how the cells kind of protect themselves. And so these rare cells that are just kind of sitting there, quiescently not growing, not dividing, these might be the cells then that just hang around for years and then contribute to that relapse five years down the road.

But I admit we don’t fully understand why one patient will relapse two years after a treatment and the next patient is still in remission 10 years later. These are things that we don’t fully understand. Every patient’s lymphoma is a little different, I’m afraid. So two people with follicular lymphoma, they don’t really have the same cancer, cancer, they are sort of like snowflakes. No two are alike. And so they can have different mutations inside the cells that’ll make the cancer behave a little differently from one patient to another. It might make it respond to treatment a little differently from one patient to another. And so what is true for one follicular lymphoma patient may not be true for another.

So if a patient’s symptoms are not being relieved, that might be a clue that the treatment isn’t working as well as we want it to. And then in some cases the only way to figure out if a treatment is working is by scanning. So we’ll have a before picture from a PET scan or a CT scan, and then we’ll take them through a few cycles of treatment, and then we’ll get another scan to prove that the treatment is working like we want it to work. And if it’s not working like we want it to work, then we’ll say, okay, this one isn’t working for you. Let’s go to the what we think is the next best option for you.

Lisa Hatfield:

Okay. Thank you. And just listening to you and hearing about all these nuances with follicular lymphoma, I would probably recommend as a patient myself with a different kind of cancer, seeking out at least a consult from somebody who specializes mostly in follicular lymphoma, at least a hematologist who can tease through some of these nuances to help you as a patient find the best treatments and therapies and quality of life. So just a little tidbit there. So, Dr. Kahl, thank you so much for being part of this Patient Empowerment Network START HERE program.It’s these conversations that help patients truly empower themselves along their treatment journey. And on behalf of patients like myself and those watching, thank you for joining us, Dr. Kahl.

Dr. Brad Kahl:

Thank you for having me.

Lisa Hatfield:  

I’m Lisa Hatfield, thank you for joining this Patient Empowerment Network program.

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Elevate | Expert Advice for Accessing Quality AML Care and Treatment Resource Guide

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A Patient’s Proactive Path to an Acute Myeloid Leukemia Diagnosis

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A Patient’s Proactive Path to an Acute Myeloid Leukemia Diagnosis from Patient Empowerment Network on Vimeo.

Meet Paloma, a 58-year-old acute myeloid leukemia (AML) survivor. After experiencing breathlessness, sore gums, and other symptoms that were initially misdiagnosed, she trusted her instincts and sought further medical support, leading to her AML diagnosis. Discover Paloma’s journey and the vital importance of being proactive and staying [ACT]IVATED in your cancer care.

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Transcript:

Being ACTIVATED in your cancer care is critical and also a continuous journey.  My name is Paloma, and I’m eager to share my journey as an acute myeloid leukemia patient in the hopes that it will help other patients and families. AML doesn’t discriminate; it can affect anyone, regardless of lifestyle or healthy eating habits.

I was 58 when I was diagnosed with AML, and my diagnosis was pretty shocking to me. I learned that you really need to trust your instincts when it comes to your health. I felt like something was off with my body, but my initial symptoms were only some breathlessness upon exertion and sore gums. But then additional symptoms started including a dry cough, some flu-like symptoms, and lumps under my armpits. I saw my primary care provider, and she prescribed antibiotics and sent me for a chest x-ray that came back without issues. 

When my breathlessness worsened along with profound fatigue, my doctor then sent me to get an ECG and additional blood tests to help determine what might be wrong. While I was waiting for my test results, my co-workers noticed that I looked thinner with my skin also being paler than usual. This was just the beginning of my AML journey. My blood tests came back with abnormal hemoglobin and blast levels, and my doctor arranged for me to be admitted to a well-regarded cancer center. I was fortunate that it was only 20 miles away but realize that not all cancer patients are this fortunate.

After seeing my AML specialist at the cancer center, he wanted to start my chemotherapy right away to fight the cancer. I was fortunate that I didn’t have issues with my intravenous line for receiving my chemotherapy. But I learned that this can be a common issue for AML patients after I joined an online AML support group. I had my first round of chemotherapy, and my daughter was able to visit me during this time. However, my care team kept monitoring my neutrophils and decided that I needed to start a second round of chemotherapy. It was now during the COVID-19 pandemic, and hospital visitors weren’t allowed.

I counted myself as fortunate that I could still continue with receiving chemotherapy. What would have happened if I’d gotten seriously ill during the early pandemic? I shudder to think that things likely would not have been easy. The hospital staff helped to keep my spirits up and also with setting up a tablet for me to do video calls with my family and friends while I was in the hospital.

Though that round of chemotherapy put me into remission for a period of time, I later came up as MRD-positive and received a targeted chemotherapy as a third round of therapy followed by a stem cell transplant. I had some graft-versus-host disease issues but got through them. I feel fortunate that there are some different treatment options for AML and would like to participate in a clinical trial to help advance treatments if I need another option on my journey. I’ve kept in touch with other patients in my AML support group during my journey from diagnosis, treatments, and recovery. I know that I couldn’t have gotten through my physical and mental challenges without them.

Though AML sounded scary at first, the future of treatment looks bright to me with emerging research and treatment options. I hope that sharing my story will make a difference for other AML patients and especially those who may come up against barriers. 

No matter who you are, being proactive is a critical step in your AML journey. Stay [ACT]IVATED by being informed, empowered, and engaged in your care.

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Advice for Setting Myeloma Treatment Goals and Collaborating on Care Decisions

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Advice for Setting Myeloma Treatment Goals and Collaborating on Care Decisions from Patient Empowerment Network on Vimeo.

This animated video defines common myeloma treatment goals, provides an overview of available myeloma therapies, and includes advice for making care decisions and the importance of being your own advocate.

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Transcript:

Bianca: 

Hi! I’m Bianca, a nurse specializing in myeloma. In this video, we’ll discuss myeloma therapy and explain how to work with your healthcare team so that you can choose a care plan best suited for YOUR myeloma. 

I’d also like to introduce you to Suzanne. Suzanne is a patient advocate living with myeloma.  

Suzanne: 

Thanks, Bianca. I’m happy to share my own experience and to talk about how I worked with my healthcare team to decide on a care plan. 

When my doctor and I were first considering my options, we started by setting treatment goals. Bianca, can you define treatment goals?  

Bianca: 

Sure! Each patient is unique, so it may vary by person. You should collaborate WITH your healthcare team to determine YOUR treatment goals. Common goals of myeloma treatment may include: 

  • Reducing and managing your symptoms. 
  • Slowing the progression of the disease. 
  • Inducing remission. 
  • And, helping you live longer while maintaining quality of life.

Suzanne: 

Thanks for explaining this. When I discussed treatment goals with my doctor, I pointed out that I still wanted to be able to play pickleball with my friends, and to care for my young grandchildren. We reviewed options that could allow me to stay as active as possible and manage my myeloma at the same time. 

Bianca: 

That’s a great point, Suzanne. It’s also important to remember that your treatment goals can change throughout the course of your myeloma. Discuss them regularly, not just with your care team but also your care partner – that can be a family member or even a close friend. 

Suzanne: 

That’s right. I discussed my care with my husband AND my adult children – it’s important to keep your support team in the loop.  

And it’s also essential to understand the treatment options available to you. Bianca, what are the types of therapy available to treat myeloma? 

Bianca: 

There are a number of different classes of therapy, which include: 

  • Proteasome inhibitors; 
  • Immunomodulatory therapies or IMiDs; 
  • Monoclonal antibodies; 
  • Stem cell transplants; 
  • There are also the new and recently approved therapies such as bispecific antibodies and CAR (Chimeric Antigen Receptor) T-cell therapy; 
  • And, of course, clinical trials.   

Clinical trials can be a good option for patients at any stage of disease, often giving patients early access to a viable and cutting-edge therapy. When considering treatment, you should ask your doctor if there is a clinical trial that may be right for you.  

Suzanne: 

Good point! When I was deciding on a therapy, my doctor and I, along with my husband, discussed the risks and benefits of each approach as well as the potential outcome of each option. My doctor also walked through the clinical trials that were available to me. 

Bianca, given all of the options, how do you decide which therapy is appropriate for a particular patient? 

Bianca: 

That’s a great question. As mentioned in our previous video, results of in-depth testing, which determine if a patient has low-risk or high-risk myeloma, can affect your choices. Other factors that impact treatment decisions may include: 

  • Your age, overall health, and any pre-existing conditions. 
  • Potential side effects of the treatment. 
  • Previous therapies that may have been used to treat your myeloma. 
  • The financial impact of a treatment plan. 
  • And the patient’s lifestyle and preference.  

Suzanne: 

That’s right. And, you shouldn’t hesitate to weigh in on what YOUR preference is. Do your own research so you understand each treatment approach, then work WITH your healthcare team to determine what might be best for you.  

Bianca: 

That’s excellent advice, Suzanne. So, when making treatment decisions, you should: 

  • Work with your healthcare team to understand your treatment goals. 
  • Talk with your doctor about all available treatment options. 
  • And, inquire about any clinical trials that may be right for you. 
  • Then, discuss the pros and cons of each option. 
  • Bring a care partner with you to appointments and take time post-visit to discuss the proposed plan and treatment options. 
  • And, always remember that you have a voice in your care. Speak up and ask questions. You are your own best advocate. 

Suzanne: 

Great advice, Bianca. Don’t forget to visit powerfulpatients.org/myeloma to learn more. Thank you for joining us!  

Expert Overview | AML Treatment Options and Phases of Therapy

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Expert Overview | AML Treatment Options and Phases of Therapy from Patient Empowerment Network on Vimeo.

What are the treatment options and phases of therapy for AML? Dr. Gail Roboz discusses the various therapies available to treat AML and to maintain remission, the timing of these therapies, and novel treatment approaches offered. 

Dr. Gail Roboz is director of the Clinical and Translational Leukemia Programs and professor of medicine at Weill Cornell Medicine and the New York Presbyterian Hospital. Learn more about Dr. Roboz.
 

Related Resources:

Choosing Therapy | How Are AML Treatment Goals Determined?

Choosing Therapy | How Are AML Treatment Goals Determined?

 What Key Testing Occurs Following an AML Diagnosis?

What Key Testing Occurs Following an AML Diagnosis?

 What Are AML Inhibitor Therapies and How Do They Work? 

What Are AML Inhibitor Therapies and How Do They Work?

Transcript: 

Katherine Banwell:

Dr. Roboz, would you provide a brief explanation of the phases of therapy for AML?  

Dr. Gail Roboz:

Yeah. So, here, too, I have to say that it’s more confusing than it used to be for the following reasons. So, historically and currently, we typically talk about induction as the first therapy that you’re going to get to get into remission. 

Then, the treatment paradigm is you do something to get into remission; do some treatment to get into remission. After that, in the realm of post-remission therapy, there are different things that can happen. There can be something called consolidation, which might be another round of chemotherapy. Some patients get consolidation, some patients don’t. After consolidation, there can be a transplant.  

So, you get into remission, you may or may not get a little bit of what’s called consolidation chemotherapy, and then go on to a transplant. 

However, sometimes either after the transplant or after chemotherapy before ever getting or instead of ever getting a transplant, there might be ongoing treatment in a lower intensity ongoing basis that is called maintenance.  

So, you’ve got to think about it as induction as what happens first, consolidation is something that happens when you’re in remission, and then maintenance usually refers to ongoing therapy that is different from consolidation. 

It’s usually lower intensity, easier to take, oral types of treatment that may go on and on. And just to be incredibly confusing, it’s different from something like breast cancer, where often the patients are given, “You get six cycles of this, and then you’re done.” From AML, there’s actually often not that type of an obvious plan right out of the gate for the patient. 

The answer will be, “It depends.” It depends. It depends how your treatment looks at this point in time. It depends how you look at this point in time. 

So then, the patients say, “Well, aren’t you going to cure me of this? What are you doing? Aren’t you going to get rid of it?” So, historically, there are some patients who get cured with chemotherapy. They get chemotherapy to get into remission, they get some chemotherapy afterwards, and there’s a cure rate for some patients with that. The majority of patients who are cured with AML get an allotransplant, or a transplant from somebody else. 

Then there’s a whole group of patients where we’re asking the question now, is it possible to get those patients beyond five years – so in oncology, five years is typically defined as cure. Can we get some patients with ongoing therapy to that past-five-year mark without a transplant? That’s in the zone of the ‘coming soon.’ Don’t have a ton of patients in that group right now, but hopefully we will. 

Katherine Banwell:

You’ve mentioned some various treatment types that are used to treat AML. Can you share a brief overview of available treatments? 

Dr. Gail Roboz:

So, the terminology that we use is a little bit annoying, because it is a little bit general. We say intensive and not intensive. 

But historically, intensive chemotherapy referred to a combination of generally two types of agents, cytarabine (Cytosar-U) and an anthracycline, which is a class of chemotherapy, that either just those two together or in combination with sometimes a third or a fourth drug usually keeps people in the hospital for around a month. Not that the chemotherapy takes that long, but the treatment gets rid of basically a lot of cells in the bone marrow, good guys and bad guys, and it takes about three weeks for those normal cells to recover. 

So, a standard intensive induction for AML is often around three to four weeks in the hospital, somewhere between three and five or so days of chemotherapy up front, depending on exactly what the protocol is. The classic regimen is actually still called 3+7, three days of one drug, seven of the other. But there are many variations of that that work. 

The chemo is then stopped, the patient hangs out in the hospital, very frequently getting transfusions and antibiotics, and we wait for the bone marrow to recover.  

Another current path that many patients are getting – almost all older patients, with ‘older’ being defined not by a specific age cutoff, but often 75 and older, almost everybody agrees no longer gets the classic chemotherapy that I just described. At some institutions, that 75 is going down, and even 70 and 65 and above are getting a new type of therapy, mostly because the new type of therapy is working pretty well. That is a combination of something called a hypomethylating agent.  

Drugs like azacitidine (Vidaza, Onureg) or decitabine (Dacogen) in combination with a pill that has changed the landscape of AML more than any other called venetoclax (Venclexta). Venetoclax is a drug that is not exclusively used for AML. 

It actually was originally approved for another type of leukemia. But I think that not many people would argue with the statement that what has changed absolutely the face of AML treatment has been this drug, because it’s a BCL2 inhibitor. What it does is it actually – cancer cells and leukemia cells in particular are very, very good at staying alive.  

They don’t undergo cell death, they don’t want to die, and venetoclax brings down their forcefield so that those cells can actually undergo apoptosis and die. 

Venetoclax in combination with azacitidine or decitabine has transformed the care of the disease, because many patients older than 65 – and the median age of diagnosis of AML is around 68 to 70. So, many patients never were well enough to have the intensive therapy. They weren’t going into remission, and they weren’t having prolonged survival often beyond a few months. 

But now, those patients do actually much better with the combination of aza [azacitidine] and venetoclax. So typically, the induction path is going to be deciding who gets an intensive therapy backbone, usually associated with long hospitalization. Who gets a less intensive backbone – by the way, that is often associated with just the same hospitalization. So, that’s why I don’t love the term ‘low intensity,’ because that implies that it doesn’t work.  

It does, and it also implies that you’re not going to be in the hospital. You probably will, because in the same way as for the more so-called intensive therapies, getting into remission involves getting rid of bone marrow cells and waiting for the normal ones to recover. Even if you are a patient who is getting the venetoclax combined with the azacitidine or decitabine, which is typically called low intensity, you may very well be in the hospital for a month. 

Because depending on where you live and who your family is and how sick you might be, you will probably want us to watch you carefully during that first month, but it’s worth it. Because if you have a good chance of getting into remission, remission is what makes life better and life longer. So, we want to get patients into remission, even if it means upfront time in the hospital. 

What Key Testing Occurs Following an AML Diagnosis?

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What Key Testing Occurs Following an AML Diagnosis? from Patient Empowerment Network on Vimeo.

What key tests occur following an AML diagnosis? Dr. Gail Roboz explains the procedures and tests to confirm the diagnosis, assess disease risk, examine AML genetic markers, and develop a treatment plan.

Dr. Gail Roboz is director of the Clinical and Translational Leukemia Programs and professor of medicine at Weill Cornell Medicine and the New York Presbyterian Hospital. Learn more about Dr. Roboz.
 

Related Resources:

Choosing Therapy | How Are AML Treatment Goals Determined?

Choosing Therapy | How Are AML Treatment Goals Determined?

 Expert Overview | AML Treatment Options and Phases of Therapy

Expert Overview | AML Treatment Options and Phases of Therapy

 What Are AML Inhibitor Therapies and How Do They Work? 

What Are AML Inhibitor Therapies and How Do They Work?

Transcript: 

Dr. Gail Roboz:

We often generally recommend a bone marrow biopsy, even if we know we can make the diagnosis from a blood test, because even though the bone marrow biopsy is not the most fun test in the world, it does offer better information for follow-up care than what you can get initially from the blood. 

So, every once in a while, we do have a patient for whom a bone marrow biopsy itself for whatever reason can’t be done. But almost always, we need a bone marrow biopsy, and on that biopsy, you’re going to look under the microscope and see what the cells look like. You’re going to get back standard testing, which is called flow cytometry, which is going to tell the difference between what are the different cells that you’re seeing under the microscope. 

But then you’re actually going to get progressively much more fancy testing, including things called chromosomes or cytogenetics, and then ultimately, the majority of patients, if at all possible, will be having mutational testing to identify certain subgroups of AML that benefit from very particular treatments. Next-generation sequencing, PCR, fusion proteins, FISH, cytogenetics, I can go on and on with all kinds of terminology that is very confusing, even to hematology fellows, let alone to patients.  

Usually, we use a combination of tests to decide, “Is this patient likely to be able to be cured with chemotherapy alone, or might this patient benefit from a stem cell transplant from somebody else after they go into remission?” 

That’s basically what the prognostic scoring systems used to be asking, but now it’s a lot more complicated than that. Because even in the favorable categories, even in the adverse categories, where there used to be very little subtlety, now there is a lot of subtlety. 

It’s all about defining getting into remission, and what do I give you once you’re in remission to keep you there? It’s no longer this windshield wiper thing of good, bad, transplant, no transplant. There’s a lot more to AML than there used to be.   

Choosing Therapy | How Are AML Treatment Goals Determined?

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Choosing Therapy | How Are AML Treatment Goals Determined? from Patient Empowerment Network on Vimeo.

How are AML treatment goals determined? Dr. Gail Roboz explains the collaborative decision-making process between patients and clinicians when exploring treatment options, important questions to ask about AML treatment goals, and the objectives of the first phase of treatment.

Dr. Gail Roboz is director of the Clinical and Translational Leukemia Programs and professor of medicine at Weill Cornell Medicine and the New York Presbyterian Hospital. Learn more about Dr. Roboz.
 

 

Related Resources:

What Key Testing Occurs Following an AML Diagnosis?

What Key Testing Occurs Following an AML Diagnosis?

 Expert Overview | AML Treatment Options and Phases of Therapy

Expert Overview | AML Treatment Options and Phases of Therapy

 What Are AML Inhibitor Therapies and How Do They Work? 

What Are AML Inhibitor Therapies and How Do They Work?

Transcript: 

Katherine Banwell:

When it comes to choosing therapy for AML, it’s important to work with your healthcare team to identify what will work best for you, the patient. So, I’d like to know how you define shared decision-making.  

Dr. Gail Roboz:

The problem with AML sometimes is that it can be such an acute, emergency-type of presentation and urgent decision-making that I think your question is almost right out of the gate for some patients that will, “Wait, I don’t even have a minute, here. How do I build a team, do the research, look online if people are telling me that I’m in the middle of an emergency?”  

That isn’t always the case for acute leukemia, but it sometimes is. I think that what happens in AML in particular for patients is a building of knowledge and a building of the team, and figuring out, first of all, where am I when I am being told this diagnosis, and is it really an emergency? Do I have to make decisions really right now, because is it life-threatening today, I don’t have time to look around? Or do I have a minute to pause and get more information? 

I definitely feel that with the Internet era and with so much connection between doctors and teams, there is much more ability to reach out instantaneously for doctors, too, to get advice on a patient who might be in a smaller hospital that doesn’t have AML experience. But I think that the first thing is to try to figure out very, very quickly, what needs to happen to me as a patient immediately, and what can wait a minute, so that I can figure out what am I being told, and what are my options? 

Katherine Banwell:

Right, right. It can be confusing for patients, just finding out this new information. Part of making care decisions is setting goals. What are AML treatment goals, and how are they determined?   

Dr. Gail Roboz:

I would say that leaving cure on the table from the beginning is always a good place to start, because you want to figure out, first of all, what am I dealing with? What are the actual options?  

But when AML strikes, and a patient who has multiple medical conditions or comorbidities that are truly compromising function independently of the diagnosis of AML, that’s going to be a special path of what is actually reasonable for someone who is terribly medically ill or otherwise frail right from the beginning? That can be defining goals, but I think from the beginning, the best thing is to leave everything on the table. What can actually be done to make me better, first of all, to get me out of my immediate trouble? What can be done to make me better, and if I’m getting better, well, I like that, how do I stay there?  

What can be done to hang on to the state of ‘better,’ which is sometimes defined as remission? In AML, the goal is to get the bone marrow working again, functioning again, get rid of the acute emergency problem, if there is one, which there may or may not be in acute leukemia. 

Sometimes it’s truly an emergency, and sometimes it isn’t. But once I get better, can I stay there? What is required to keep me with a working bone marrow for as long as possible?  

But once you are starting to sort through the diagnosis, you realize that saying that somebody has acute myeloid leukemia is not telling me nearly enough information. This is a disease that is what we call biologically heterogeneous, which means there are lots of different forms. It’s like saying you’re sick. What exactly does that mean? There are lots of things that can make you sick. There are lots of different subtypes of AML, and fairly quickly in most institutions, we start getting back some information specifically on the subtype and biological characteristics of the disease.  

This can be very, very important in the initial treatment planning, and depending on where you are, the information that you get back can sometimes take 24 hours, 48 hours, 72 hours, a week. So, you start learning very quickly though that, “If I’m not in a complete emergency that requires instantaneous treatment, can I get back more information about the biological subtype of the disease so that I can start treatment planning of what is my best option right out of the gate?” That’s usually called induction, or the first therapy that you’re going to get with the goal, ‘getting rid of leukemia cells and getting into remission.’ That’s part one, and then everything that comes after that is about keeping you in remission.   

But for the initial goal, what is the therapy that the patient needs to get to get into remission? In order to figure that out, the good news is there are a lot of different ways to slice and dice getting into remission, and actually, it used to be such a weighty decision. 

Now, I would actually encourage people to – not relax, you can never use the word ‘relax’ with acute leukemia. But there are several different induction strategies for most patients that would be okay.  

So, even if you get started with one strategy and you hear five days later that another doctor might do something different, there are a lot of ways to safely get into remission. I think everybody should be pleased about the fact that we’re doing much better than we used to for patients across the board, all the way from children to much older adults, to safely getting people into remission. 

Katherine Banwell:

So, what sort of factors then do you take into consideration when you’re choosing a therapy? 

Dr. Gail Roboz:

So, out of the gate, there are the patients that I think I referred to earlier who truly, truly are in situations based on their other diseases that there are certain treatments we would just cross out right out of the gate. 

If there are patients with very, very severely compromised cardiac or renal or lung function or are terribly ill from other conditions, AML doctors will right out of the gate for those patients eliminate certain treatments. But absent that scenario, what we try to look for is the biology of the disease. Not look at the age, not look at the comorbidities unless they are so severe that they make obvious certain choices. 

But rather, what I like to do is say, “What kind of AML is this, and what is the best treatment that I have to get this patient into remission?” And then ask the question, “can this particular patient handle this therapy?” Sometimes, these days, there actually may be more than one route to get to remission depending on the biology of the disease, and then, if that’s the case, then I can start getting picky and look at the individual patient. Where does the patient live? Who’s the patient’s family? What other diseases has the patient been treated for?  

Is there something that I can use? If I have a choice, if there are a couple of different things that might work, how do I fit the treatment to best take care of the needs of this particular patient? If I don’t have choices, then my question is, “Okay, how do I get this patient through my one therapy that I think is the truly, truly best option?” 

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Elevate | What You Should Know About Your Role in AML Treatment and Care Decisions

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Elevate | What You Should Know About Your Role in AML Treatment and Care Decisions from Patient Empowerment Network on Vimeo.

How can you elevate your AML care and treatment? AML expert Dr. Gail Roboz discusses the importance of participating in AML treatment decisions, reviews key factors that may impact therapy options, and shares advice for advocating for yourself.
 
Dr. Gail Roboz is director of the Clinical and Translational Leukemia Programs and professor of medicine at Weill Cornell Medicine and the New York Presbyterian Hospital. Learn more about Dr. Roboz.
 

Related Resources:

FLT3 inhibitors for AML Update

 Thriving With AML | Advice for Setting Goals and Making Treatment Decisions

Thriving With AML | Advice for Setting Goals and Making Treatment Decisions

 Expert Advice | Managing AML Symptoms and Treatment Side Effects

Expert Advice | Managing AML Symptoms and Treatment Side Effects

Transcript: 

Katherine Banwell:

Hello, and welcome. I’m your host, Katherine Banwell. It’s no secret that the quality-of-care patients receive can vary, and patients who are educated about their condition and involved in their care may have improved outcomes. That’s why the Patient Empowerment Network created the Elevate series, to help AML patients and their care partners feel well-informed when making treatment decisions with their healthcare team. 

In today’s program, an AML expert will join us to share advice for accessing better overall care. Before we get into the discussion, please remember that this program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you. Well, let’s meet our guest today. Joining us is Dr. Gail Roboz. Dr. Roboz, would you please introduce yourself? 

Dr. Gail Roboz:

Absolutely. Thank you so much for having me. My name is Gail Roboz. I’m a professor of medicine and director of the clinical and translational leukemia programs at Weill Cornell Medicine and the NewYork-Presbyterian Hospital in New York City. Thank you again for having me. 

Katherine Banwell:

Well, thank you so much for joining us today. We really appreciate it. I’d like to start by discussing your role as a researcher. You’re on the frontlines for advancements in the AML field. What led you here, and why is it important to you? 

Dr. Gail Roboz:

So, I’m actually asked that question quite frequently, because AML is a challenging, difficult, scary disease, and people don’t necessarily assume that somebody in medical school would gravitate toward it. 

But I have to say that what is incredibly fascinating back then and now about leukemia is the continuous access to the disease. Patients will maybe giggle or groan as I’m saying that, because you can get a blood sample really anytime. You can even get a bone marrow sample anytime, although patients don’t enjoy that so much. 

But from a research perspective, it is absolutely extraordinary to be dealing with a disease where you can, in real time, truly run back and forth to a laboratory and see what’s happening, what is the new drug or the old drug doing, what’s happening with the patient, and I would say that from a fascination of a medical student perspective that grabbed me then and still does today.  

Katherine Banwell:

When it comes to choosing therapy for AML, it’s important to work with your healthcare team to identify what will work best for you, the patient. So, I’d like to know how you define shared decision-making.  

Dr. Gail Roboz:

The problem with AML sometimes is that it can be such an acute, emergency-type of presentation and urgent decision-making that I think your question is almost right out of the gate for some patients that will, “Wait, I don’t even have a minute, here. How do I build a team, do the research, look online if people are telling me that I’m in the middle of an emergency?”  

That isn’t always the case for acute leukemia, but it sometimes is. I think that what happens in AML in particular for patients is a building of knowledge and a building of the team, and figuring out, first of all, where am I when I am being told this diagnosis, and is it really an emergency? Do I have to make decisions really right now, because is it life-threatening today, I don’t have time to look around? Or do I have a minute to pause and get more information? 

I definitely feel that with the Internet era and with so much connection between doctors and teams, there is much more ability to reach out instantaneously for doctors, too, to get advice on a patient who might be in a smaller hospital that doesn’t have AML experience. But I think that the first thing is to try to figure out very, very quickly, what needs to happen to me as a patient immediately, and what can wait a minute, so that I can figure out what am I being told, and what are my options?  

Katherine Banwell:

Right, right. It can be confusing for patients, just finding out this new information. Part of making care decisions is setting goals. What are AML treatment goals, and how are they determined?   

Dr. Gail Roboz:

I would say that leaving cure on the table from the beginning is always a good place to start, because you want to figure out, first of all, what am I dealing with? What are the actual options?   

But when AML strikes, and a patient who has multiple medical conditions or comorbidities that are truly compromising function independently of the diagnosis of AML, that’s going to be a special path of what is actually reasonable for someone who is terribly medically ill or otherwise frail right from the beginning? That can be defining goals, but I think from the beginning, the best thing is to leave everything on the table. What can actually be done to make me better, first of all, to get me out of my immediate trouble? What can be done to make me better, and if I’m getting better, well, I like that, how do I stay there?  

What can be done to hang on to the state of ‘better,’ which is sometimes defined as remission? In AML, the goal is to get the bone marrow working again, functioning again, get rid of the acute emergency problem, if there is one, which there may or may not be in acute leukemia. 

Sometimes it’s truly an emergency, and sometimes it isn’t. But once I get better, can I stay there? What is required to keep me with a working bone marrow for as long as possible? 

But once you are starting to sort through the diagnosis, you realize that saying that somebody has acute myeloid leukemia is not telling me nearly enough information. This is a disease that is what we call biologically heterogeneous, which means there are lots of different forms. It’s like saying you’re sick. What exactly does that mean? There are lots of things that can make you sick. There are lots of different subtypes of AML, and fairly quickly in most institutions, we start getting back some information specifically on the subtype and biological characteristics of the disease.  

This can be very, very important in the initial treatment planning, and depending on where you are, the information that you get back can sometimes take 24 hours, 48 hours, 72 hours, a week. So, you start learning very quickly though that, “If I’m not in a complete emergency that requires instantaneous treatment, can I get back more information about the biological subtype of the disease so that I can start treatment planning of what is my best option right out of the gate?” That’s usually called induction, or the first therapy that you’re going to get with the goal, ‘getting rid of leukemia cells and getting into remission.’ That’s part one, and then everything that comes after that is about keeping you in remission.  

But for the initial goal, what is the therapy that the patient needs to get to get into remission? In order to figure that out, the good news is there are a lot of different ways to slice and dice getting into remission, and actually, it used to be such a weighty decision. 

Now, I would actually encourage people to – not relax, you can never use the word ‘relax’ with acute leukemia. But there are several different induction strategies for most patients that would be okay.  

So, even if you get started with one strategy and you hear five days later that another doctor might do something different, there are a lot of ways to safely get into remission. I think everybody should be pleased about the fact that we’re doing much better than we used to for patients across the board, all the way from children to much older adults, to safely getting people into remission. 

Katherine Banwell:

Right. So, what sort of factors then do you take into consideration when you’re choosing a therapy? 

Dr. Gail Roboz:

So, out of the gate, there are the patients that I think I referred to earlier who truly, truly are in situations based on their other diseases that there are certain treatments we would just cross out right out of the gate.  

If there are patients with very, very severely compromised cardiac or renal or lung function or are terribly ill from other conditions, AML doctors will right out of the gate for those patients eliminate certain treatments. But absent that scenario, what we try to look for is the biology of the disease. Not look at the age, not look at the comorbidities unless they are so severe that they make obvious certain choices. 

But rather, what I like to do is say, “What kind of AML is this, and what is the best treatment that I have to get this patient into remission?” And then ask the question, “can this particular patient handle this therapy?” Sometimes, these days, there actually may be more than one route to get to remission depending on the biology of the disease, and then, if that’s the case, then I can start getting picky and look at the individual patient. Where does the patient live? Who’s the patient’s family? What other diseases has the patient been treated for?  

Is there something that I can use? If I have a choice, if there are a couple of different things that might work, how do I fit the treatment to best take care of the needs of this particular patient? If I don’t have choices, then my question is, “Okay, how do I get this patient through my one therapy that I think is the truly, truly best option?” 

Katherine Banwell:

Okay. I’d like to turn to test results for a moment. What sort of tests should be done following an AML diagnosis?  

Dr. Gail Roboz:

We often generally recommend a bone marrow biopsy, even if we know we can make the diagnosis from a blood test, because even though the bone marrow biopsy is not the most fun test in the world, it does offer better information for follow-up care than what you can get initially from the blood. 

So, every once in a while, we do have a patient for whom a bone marrow biopsy itself for whatever reason can’t be done. But almost always, we need a bone marrow biopsy, and on that biopsy, you’re going to look under the microscope and see what the cells look like. You’re going to get back standard testing, which is called flow cytometry, which is going to tell the difference between what are the different cells that you’re seeing under the microscope. 

But then you’re actually going to get progressively much more fancy testing, including things called chromosomes or cytogenetics, and then ultimately, the majority of patients, if at all possible, will be having mutational testing to identify certain subgroups of AML that benefit from very particular treatments. Next-generation sequencing, PCR, fusion proteins, FISH, cytogenetics, I can go on and on with all kinds of terminology that is very confusing, even to hematology fellows, let alone to patients.  

Usually, we use a combination of tests to decide, “Is this patient likely to be able to be cured with chemotherapy alone, or might this patient benefit from a stem cell transplant from somebody else after they go into remission?” 

That’s basically what the prognostic scoring systems used to be asking, but now it’s a lot more complicated than that. Because even in the favorable categories, even in the adverse categories, where there used to be very little subtlety, now there is a lot of subtlety. 

It’s all about defining getting into remission, and what do I give you once you’re in remission to keep you there? It’s no longer this windshield wiper thing of good, bad, transplant, no transplant. There’s a lot more to AML than there used to be. 

Katherine Banwell:

I’d like to add that if you, the viewer, are interested in learning more about AML testing and treatment, PEN has a number of resources available for you. You can find these at powerfulpatients.org/AML, or by scanning the QR code on your screen.  

Before we get into specific treatment types, Dr. Roboz, would you provide a brief explanation of the phases of therapy for AML? You mentioned induction therapy earlier. Would you tell us what that is? 

Dr. Gail Roboz:

Yeah. So, here, too, I have to say that it’s more confusing than it used to be for the following reasons. So, historically and currently, we typically talk about induction as the first therapy that you’re going to get to get into remission.  

Then, the treatment paradigm is you do something to get into remission; do some treatment to get into remission. After that, in the realm of post-remission therapy, there are different things that can happen. There can be something called consolidation, which might be another round of chemotherapy. Some patients get consolidation, some patients don’t. After consolidation, there can be a transplant.  

So, you get into remission, you may or may not get a little bit of what’s called consolidation chemotherapy, and then go on to a transplant. 

However, sometimes either after the transplant or after chemotherapy before ever getting or instead of ever getting a transplant, there might be ongoing treatment in a lower intensity ongoing basis that is called maintenance.  

So, you’ve got to think about it as induction as what happens first, consolidation is something that happens when you’re in remission, and then maintenance usually refers to ongoing therapy that is different from consolidation. 

It’s usually lower intensity, easier to take, oral types of treatment that may go on and on. And just to be incredibly confusing, it’s different from something like breast cancer, where often the patients are given, “You get six cycles of this, and then you’re done.” From AML, there’s actually often not that type of an obvious plan right out of the gate for the patient. 

The answer will be, “It depends.” It depends. It depends how your treatment looks at this point in time. It depends how you look at this point in time. 

So then, the patients say, “Well, aren’t you going to cure me of this? What are you doing? Aren’t you going to get rid of it?” So, historically, there are some patients who get cured with chemotherapy. They get chemotherapy to get into remission, they get some chemotherapy afterwards, and there’s a cure rate for some patients with that. The majority of patients who are cured with AML get an allotransplant, or a transplant from somebody else. 

Then there’s a whole group of patients where we’re asking the question now, is it possible to get those patients beyond five years – so in oncology, five years is typically defined as cure. Can we get some patients with ongoing therapy to that past-five-year mark without a transplant? That’s in the zone of the ‘coming soon.’ Don’t have a ton of patients in that group right now, but hopefully we will. 

Katherine Banwell:

You’ve mentioned some various treatment types that are used to treat AML. Can you share a brief overview of available treatments? 

Dr. Gail Roboz:

So, the terminology that we use is a little bit annoying, because it is a little bit general. We say intensive and not intensive. 

But historically, intensive chemotherapy referred to a combination of generally two types of agents, cytarabine (Cytosar-U) and an anthracycline, which is a class of chemotherapy, that either just those two together or in combination with sometimes a third or a fourth drug usually keeps people in the hospital for around a month. Not that the chemotherapy takes that long, but the treatment gets rid of basically a lot of cells in the bone marrow, good guys and bad guys, and it takes about three weeks for those normal cells to recover. 

So, a standard intensive induction for AML is often around three to four weeks in the hospital, somewhere between three and five or so days of chemotherapy up front, depending on exactly what the protocol is. The classic regimen is actually still called 3+7, three days of one drug, seven of the other. But there are many variations of that that work. 

The chemo is then stopped, the patient hangs out in the hospital, very frequently getting transfusions and antibiotics, and we wait for the bone marrow to recover.  

Another current path that many patients are getting – almost all older patients, with ‘older’ being defined not by a specific age cutoff, but often 75 and older, almost everybody agrees no longer gets the classic chemotherapy that I just described. At some institutions, that 75 is going down, and even 70 and 65 and above are getting a new type of therapy, mostly because the new type of therapy is working pretty well. That is a combination of something called a hypomethylating agent.  

Drugs like azacitidine (Vidaza, Onureg) or decitabine (Dacogen) in combination with a pill that has changed the landscape of AML more than any other called venetoclax (Venclexta). Venetoclax is a drug that is not exclusively used for AML. 

It actually was originally approved for another type of leukemia. But I think that not many people would argue with the statement that what has changed absolutely the face of AML treatment has been this drug, because it’s a BCL2 inhibitor. What it does is it actually – cancer cells and leukemia cells in particular are very, very good at staying alive.  

They don’t undergo cell death, they don’t want to die, and venetoclax brings down their forcefield so that those cells can actually undergo apoptosis and die. 

Venetoclax in combination with azacitidine or decitabine has transformed the care of the disease, because many patients older than 65 – and the median age of diagnosis of AML is around 68 to 70. So, many patients never were well enough to have the intensive therapy. They weren’t going into remission, and they weren’t having prolonged survival often beyond a few months. 

But now, those patients do actually much better with the combination of aza [azacitidine] and venetoclax. So typically, the induction path is going to be deciding who gets an intensive therapy backbone, usually associated with long hospitalization. Who gets a less intensive backbone – by the way, that is often associated with just the same hospitalization. So, that’s why I don’t love the term ‘low intensity,’ because that implies that it doesn’t work.  

It does, and it also implies that you’re not going to be in the hospital. You probably will, because in the same way as for the more so-called intensive therapies, getting into remission involves getting rid of bone marrow cells and waiting for the normal ones to recover. Even if you are a patient who is getting the venetoclax combined with the azacitidine or decitabine, which is typically called low intensity, you may very well be in the hospital for a month. 

Because depending on where you live and who your family is and how sick you might be, you will probably want us to watch you carefully during that first month, but it’s worth it. Because if you have a good chance of getting into remission, remission is what makes life better and life longer. So, we want to get patients into remission, even if it means upfront time in the hospital. 

Katherine Banwell:

You mentioned one inhibitor as targeted therapy, but there are a couple of others. Would you briefly tell us about those? 

Dr. Gail Roboz:

So, over the years recently, we have identified certain specific targets in AML which are resulting in the addition of medications on these standard backbones. So, the target for venetoclax is something called BCL2, and actually, venetoclax probably makes all chemotherapy better. It’s kind of a controversial statement, but I’m going to stand by it. But in AML, it has been shown that the addition of venetoclax to lots of different backbones makes them work better. There are other things to hit, though.  

For example, there are patients with AML who have something called a FLT3, F-L-T-3 mutation. This mutation also has specific inhibitors that are FDA-approved drugs that target specifically the FLT3 mutation, and if you have one of those, your doctor may add on a FLT3 inhibitor to either a lower intensity or an intensive backbone. Similarly, there are agents called IDH inhibitors. There are IDH1 and IDH2 inhibitors. 

If I start getting into isocitrate dehydrogenase pathways on this webinar, I think everybody will click off, because it’s certainly bored all of the medical students in med school, and it’s pretty tough to understand. But the bottom line is it’s very cool stuff because that boring pathway in medical school that nobody really thought about too much is actually part of very, very, central cellular functions that are a vulnerability now that have been identified in leukemic cells that, if you hit them with these specific inhibitors, patients do better.  

Now, couple of things for patients. It doesn’t mean that it’s better to have a FLT3 or an IDH mutation because the targeted therapies are available. So, a lot of patients are disappointed when they don’t have mutations. I don’t want you to think in that way. It’s not that it’s better, it’s different.  

It identifies a different biology. If you have certain mutations, there are certain medications that may help you more.  

That’s why I think the patients are learning quickly, too, to ask the doc – they may not remember the letters of the alphabet soup, but “Do I have something about my AML that can get one of these targeted therapies added on?” I think is a good question to think about. “Do I have something about my disease that has a specific drug that we’ve already learned makes outcomes better?”  

Katherine Banwell:

There’s a new emerging therapy as well. Is it the menin inhibitor? 

Dr. Gail Roboz:

I think that, in understanding different targets and different pathways, it leads me to a general statement that if you can get yourself potentially onto a clinical trial at an academic center, that is something to consider right out of the gate. Because there is a lot, a lot, a lot going on in this field right now. 

What we are hoping, and the reason that I am talking to you about venetoclax and FLT3 inhibitors and IDH inhibitors, is because of all the patients who jumped onto those clinical trials and proved that those drugs are better. Some of them are my patients! I was fortunate on some of those early trials to have some real winners in patients who got onto the trials. They’re the ones who drove the success. 

So, for example, menin inhibitors, which are very, very exciting, targeted agents for NPM1 and KMT2A mutations and rearrangements – these are complicated to remember as a patient, but there’s a cool drug out there that might be for you. I think that patients who really think about asking the question wherever they are, the “Hey, I just got a diagnosis of AML. Is there a clinical trial that might look good for me?” I think is a great question to ask pretty much out of the gate. 

Katherine Banwell:

The symptoms of AML as well as the side effects of certain medications can vary greatly among patients. So, how do you approach symptom management with your patients? 

Dr. Gail Roboz:

Patients will giggle because I repeat this line. You have to be afraid of the disease, not the treatment. I think that if you read the package insert on a Tylenol, you’re certainly not going  to think you’re going to live for more than 20 minutes if you take one of those. You can certainly appreciate that, with chemotherapy drugs and including some of the novel agents that I’m talking about, if you read package inserts and look at some of the signs and symptoms and things that can happen, it’s extraordinarily overwhelming. 

I think that a lot of what I do for patients is I keep them close. Because if the patient is in the hospital or coming in very frequently in clinic, I think that that everyday assessment of, “What are you experiencing?” and “What can I tell you is the disease’s fault, and what can I tell you is the medication’s fault?” is so, so important. 

Especially in the newly diagnosed patients, where the disease is active. Of course, we want to try to minimize anything that we can do to make the process better for patients, more comfortable for patients, but there are certain things that we do tell people, “You’ve got to slug through this particular problem, because this is the disease’s fault.” This is different from a patient in remission, where they might be getting ongoing therapy with something, or we say, “Hey, wait a minute. You’d be feeling fine, except now you’re taking this medication. How do we minimize messing up quality of life in remission?” 

Because we want you to feel great when you’re in remission. I think the real answer of that is to have a really close collaboration with the healthcare team, and for the patients to really understand – I repeat this because it’s so important. What is the disease’s fault, and what is the treatment’s fault? If there’s something that is therapy-related, do I have a substitute or do I not have a substitute?  

Because if the drug is essential to get us where we need to go, well, what can we do to manage comfort and to manage symptoms until you get to the place where your marrow is working again? 

Katherine Banwell:

That’s great advice, Dr. Roboz. I would like to get to an audience question that we received prior to the program. This one comes from Johanna. “How can I better understand my lab test results? What questions should I be asking my provider about those results?”  

Dr. Gail Roboz:

One of the things that I would say to patients is to be careful when interpreting your own results, because I really am not exaggerating to say that patients have had absolute trauma looking at things that I look at it and say, “Oh, this looks great.” So, the first thing is, be careful being your own doctor. 

The second thing though is that the author of the question has to understand that there’s going to be a tsunami of data coming in with respect to AML treatment. Sometimes in the hospital on a daily basis when you’re in the middle of an induction, there is a true – tsunami is the right word – a deluge of data, and you have to work with your team to say, “What am I following here? What’s important at this phase in my treatment? What’s the number I’m looking at?” Patients sometimes tell me, “I don’t want to know any of this,” and I’m fine with that.  

I think it’s actually okay. Sometimes patients will say, “Give me guidance,” and I will be specific. Because you can actually have a leukemia induction patient where every single laboratory value is abnormal. They might be getting pushed to a device, in the morning, sitting in the hospital on your iPad, 50 abnormal results. You’re trying to battle back the disease and be positive and advocate for yourself, but there are 50 abnormal results in front of you. 

I think you have to really work with the team to say, “What am I looking at today? What are the numbers that are the really important ones? There are 50 abnormal ones here; everything is getting a yellow or a red light in this. How do I go through this?”  

And to appreciate, also, that at different points in the treatment, the beginning of treatment induction post-remission therapy, you’re looking for different things. So, work with your team so that you’re not assessing every single result with equivalent weight, because I think you’re going to stress yourself out.  

Katherine Banwell:

That’s great advice, Dr. Roboz. Thank you. As we close out the program, I’d like to find out what you would like to leave the audience with. Why are you hopeful? 

Dr. Gail Roboz:

AML is changing incredibly rapidly. And  I can tell you it is a lot more fun to be an AML doctor now than it used to be, with respect to what I am offering for patients. We have always fought really, really hard to have our wins, but we’re winning more. I do think that it is a complicated space to navigate for patients, but there is room for a lot of optimism. 

I think we are getting patients transplanted  –  patients that we never thought would ever go through a transplant or getting transplanted. Patients who never had a chance of even living more than six or eight months or living much longer than that. Is it perfect? No. Do we have as many cures as we want?  

No, but there’s a lot going on. I think if patients feel that excitement, they will also feel the need to ask about those clinical trials. Because I think that for a lot of patients, clinical trials is an area where they would be worried. They’re not sure that they want to. “I don’t want to be a guinea pig,” and yet here I can say in the AML space, one after another after another drug approvals in the last several years, with the patients on those trials being awfully happy that they participated. 

So, I think that it’s a very, very terrifying diagnosis. There’s nothing that I can do to take the sting out of that. But try to find yourself in an optimistic place with options that are being offered to the very, very, very best that we can do. There are patients who are listening, I’m sure, who have relapsed or refractory disease who are not feeling that optimism. 

I want to address you specifically, because we don’t have enough yet. We’re trying. When you have AML that has come back or come back multiply, that’s dangerous and difficult. But for those patients in particular, try really hard to get onto clinical trials. If the drugs that we have out there – if you’ve already taken them and they haven’t worked for you or if they’re not serving you well, if you’re in good shape and the drugs that we have aren’t good enough, well, let’s see if we can get you on something that’s investigational. 

Katherine Banwell:

Dr. Roboz, thank you so much for taking the time to join us today. 

Dr. Gail Roboz:

Thank you for having me. 

Katherine Banwell:

I also want to thank all of our collaborators. 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 today.  

Expert Perspective | New and Emerging Progress in Lung Cancer Treatment

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Expert Perspective | New and Emerging Progress in Lung Cancer Treatment from Patient Empowerment Network on Vimeo.

What’s the latest in lung cancer research? Dr. Thomas Marron from the Tisch Cancer Institute at Mount Sinai Hospital discusses the advances in targeted therapy and immunotherapy and what this progress means for patients with lung cancer. 

Dr. Thomas Marron is Director of the Early Phase Trials Unit at the Tisch Cancer Institute at Mount Sinai Hospital. Dr. Marron is also Professor of Medicine and Professor of Immunology and Immunotherapy at the Icahn School of Medicine at Mount Sinai. Learn more about Dr. Marron.

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Transcript:

Katherine Banwell:

Dr. Marron, you’re a leading researcher in the field. What new and emerging progress in lung cancer care are you excited about? 

Dr. Thomas Marron:

So, there’s many extremely exciting, targeted therapies that’re in development. And so, as I mentioned, we do genetic sequencing, and we get three to 500 genes’ worth of data. But we only have drugs to target around 10 of those.  

So, hopefully in the coming years, in the next three to five years, we’ll have many more options based on somebody’s genetic profile of their tumor. I think that also, within the field of immunotherapy, which typically are given to patients who don’t have those targetable mutations. 

Immunotherapy is really, has revolutionized the treatment of lung cancer and with immunotherapy, we’re actually able to cure a subset of patients while in the past, we always said patients with metastatic disease had incurable disease, but it was treatable disease, just not curable.  

Now, we are curing a subset of patients. Unfortunately, we’re not curing the majority of patients. But the field of immunotherapy is evolving very quickly with new therapies targeting new parts of the immune system.  

So, similar with targeted therapies, it’s really an umbrella term. So, targeted therapy is an umbrella term for dozens of different drugs. Immunotherapy, similarly, is an umbrella term for dozens of different approaches to the immune system. So, dozens of different ways to turn on the immune system so that the immune system does its job and recognizes and kills cancer. Because your immune system is in your body to tell the difference between foreign things like COVID and normal things like your lung.  

And cancer is somewhere in between and there’s probably hundreds of different ways in which cancer finds an ability to hijack our immune system and then turn our immune system off. And so, I think with these emerging therapies that we’re developing now and will be further developed in the next five to 10 years, I think we’re going to see another revolution happen in the setting of immunotherapy.  

Katherine Banwell:

So, what do these advances mean for non-small cell lung cancer patients?  

Dr. Thomas Marron:

So, in non-small cell lung cancer, immunotherapy has really changed the way that we’re treating patients from 10 years ago when we were giving chemotherapy alone, or maybe 15 years ago. Ten or 15 years ago, when I saw a patient with metastatic disease, I would have to have a very frank conversation with them and tell them that the median survival was 10 months and that this was an incurable illness that would eventually take their life. Now, with the introduction of immunotherapy, patients are living more than twice as long on average. 

And there are a subset of patients, somewhere between 10 to 20  percent of people that go into remission and stay in remission. And so, that really has revolutionized the treatment. Obviously, we’re not done, because we still have to help the remainder of those patients and our goal is 100 percent cure. But the fact that we’re even using the C-word, cure in our cancer clinics is really amazing. 

Personalized Medicine for Myeloma Treatment | What Patients Should Know

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Personalized Medicine for Myeloma Treatment | What Patients Should Know from Patient Empowerment Network on Vimeo.

What is personalized medicine, and how can myeloma patients access this type of care? Myeloma expert Dr. Omar Nadeem defines personalized medicine and shares how test results can impact myeloma care and treatment options.

Dr. Omar Nadeem is the Clinical Director of the Myeloma Immune Effector Cell Therapy Program and Associate Director of the Multiple Myeloma Clinical Research Program at the Dana-Farber Cancer Institute. Learn more about Dr. Nadeem.

Download Resource Guide

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Transcript:

Katherine:

Well, Dr. Nadeem, we’ve been hearing the term personalized medicine more frequently in recent years. How would you define personalized medicine for myeloma, and how can patients access this type of care?  

Dr. Nadeem:

Yeah, personalized medicine or precision medicine is a term that we’ve really sort of used for many oncologic conditions over the last decade or so. I would say, for multiple myeloma, in terms of identifying a target within the myeloma cell that’s unique to the patient. 

And then deploying a certain therapy to that patient because of that target is still lacking. We do have one example where patients have, for example, an 11;14 translocation, which we see in about 15 percent of myeloma patients.  

There’s an agent called venetoclax (Venclexta) that is very active against that particular cohort of patients, although that is still not approved to be used, but that’s one example where that agent specifically benefits that type of myeloma. Other than that, most of the therapies that we have benefit essentially everybody with myeloma, which is great, but it’s not so personalized.  

Where I would say there’s the most personalization happening now, at least in my practice, is looking at which types of therapies an individual patient may receive. What I mean by that is if somebody’s in an excellent response, with quadruplet-based induction therapy, I have a very real discussion with them about the pros and cons of stem cell transplant.  

We make those decisions in real time depending on how the patient doing, depending on how their response is.  

And then kind of deciding a whole kind of what are the kind of risks and benefits and what makes sense for that individual patient. Similarly, when you go on to maintenance therapy, maintenance therapy means that after you’ve gone through the initial phase of your myeloma therapy and the disease is under control, what type of therapy can we keep you on to keep it under control for as long as possible? Historically, that has been lenalidomide or Revlimid. Now we’re adding drugs such as daratumamab (Darzalex) and other agents to Revlimid to see if that can further prolong the response to that initial therapy.  

So, all those decisions are so individualized that you have to discuss with your provider what makes sense for you and what are the pros and cons of doing one approach versus the other.   

Katherine:

Well, if we’re talking about in-depth testing, how do the results of that testing affect treatment options? 

Dr. Nadeem:

So, right now we use conventional blood tests to get a sense of response in the vast majority of patients. That includes the serum protein electrophoresis and the serum free light chain assay.  

Most patients have detectable levels of these proteins, abnormal proteins in the blood at diagnosis and then you can follow them using a blood test. There’s a subset of patients that have disease only that shows up on scans. So, we then kind of incorporate some of those scans and then, also, utilize the bone marrow results both in the beginning and in subsequent analyses to kind of give a big-picture composite response assessment for that particular patient. Nowadays, there are also other tools that we’re using, such as MRD, or minimal residual disease.  

That is a test that is done on a bone marrow biopsy to determine, if you don’t have detectable protein in the blood, do you have myeloma cells present at the deepest level possible? And if you do versus if you don’t, trials have shown that there is a difference in terms of prognosis. Now, while that hasn’t fully been utilized yet to make treatment decisions in patients that are not on clinical trials, we do get prognostic information out of it, and nowadays, more and more of those trials are using these MRD tests to determine what to do with treatment.  

And I think that’s how it’s going to be in the future. So, having those extra tests available but, again, important to discuss with your provider what is the utility of this test. How are we going to use this information for your individual case to make some decisions? 

Combination AML Therapy for Newly Diagnosed Patients | What Are the Long-Term Effects?

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Combination AML Therapy for Newly Diagnosed Patients | What Are the Long-Term Effects? from Patient Empowerment Network on Vimeo.

A Patient Empowerment Network community member wants to know the length of time that patients can stay on the combined treatment of azacitidine (Vidaza) and venetoclax (Venclexta). AML specialist Dr. Jacqueline Garcia responds, sharing an update on the long-term follow-up data for this combination treatment.

Dr. Jacqueline Garcia is an oncologist and AML researcher at the Dana-Farber Cancer Institute. Learn more about Dr. Garcia.

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Transcript:

Katherine Banwell:

Jerry had this question. “How long can patients stay on azacitidine (Vidaza) and venetoclax (Venclexta) before relapse or toxicities force them to abandon treatment?”  

Dr. Jacqueline Garcia:

So, this is a good question. I would say azacitidine and venetoclax just got FDA-approved just shy of five years now, and it’s totally changed our treatment paradigm in many great ways. It was initially approved for patients that could not get intensive chemotherapies or were above 75. We call these our older patients, our more vulnerable.   

And we demonstrated and compared to azacitidine alone. It was given with placebo. We saw that the combination of azacitidine and venetoclax not only was safe, well-tolerated, it led to two-and-a-half times higher complete remission rates and impressively longer survival. That’s all we care about, patients are living longer. So, one of the things that we are appreciating in 2023 now, now that we have more patients on azacitidine and venetoclax, is that we have many patients that are long-term responders.  

So, in the original clinical trial we’ve been reported – and we just submitted the update for the long-term follow-up that we presented at the American Society of Hematology meeting in 2022, in December.   

We presented the long-term follow-up data that shows that responses can be durable and even as long as two years or three years in some patients. The average amount of time the patients are on therapy is somewhere between one-and-a-half to two years. But not every patient performs like an average patient.  

We have some that respond for less time. We have some that respond for a longer time. So, I definitely have a few patients that have been on combination therapy, and we’ve gone to year three, then four, and two that got to year five. And that was using the original indication of older the 75, no intensive chemotherapy. Most of those patients in the original trial and led to the approval were not transplant candidates. But once those drugs got approved, more patients that were older started getting this therapy.  

And so, the durability of this treatment might be longer for people that don’t have competing health problems and for specific mutation subtypes. There are a couple of mutation subtypes that include IDH2 and NPM1, where we’ve seen some extreme long-term responders.  

And then, there are others that are much shorter. So, I would say it’s very individual. In terms of toxicities in general, the regimens very well-tolerated. And if it’s not, often it’s because there should be supportive care, prophylaxis, and adjustments to the dosing strategy, which has been well-published. Sometimes, if you have a treating oncologist that is less familiar, they won’t know the nuances of how to adjust the doses, so I would ask your local oncologist to reach out to anybody that was part of the original trials. Often, a lot of us are very responsive to helping out our colleagues to help patients to stay on treatment.   

But at the end of the day, if a patient loses response or has a bad toxicity that makes it very difficult, we have to move on to another therapy.   

Stem Cell Transplant for AML | What Patients Should Know

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Stem Cell Transplant for AML | What Patients Should Know from Patient Empowerment Network on Vimeo.

When is stem cell transplant an option for AML care? AML specialist Dr. Alice Mims discusses who this procedure is most appropriate for and how patients are monitored after transplant. Dr. Mims also addresses common issues following stem cell transplant, including joint pain. 

Dr. Alice Mims is a hematologist specializing in acute and chronic myeloid conditions. Dr. Mims serves as the Acute Leukemia Clinical Research Director at The Ohio State University Comprehensive Cancer Center – James. Learn more about Dr. Mims. 

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Transcript:

Katherine Banwell:

Janet wants to know what factors enable a patient to achieve and continue in remission if they are not able to achieve stem cell transplant due to age restrictions.  

Dr. Alice Mims:

So, I think first and foremost, I think it’s very important that there — that patients are aware that there shouldn’t be just strict, stringent cutoffs of age as a requirement for stem cell transplant.  

 And really, there’s a lot of research going on that we should take into account. Physiological age, and there are ways to measure that just to be sure that stem cell transplant really is not an option. And for patients who stem cell transplant is not an option, I think as we talked about earlier, so there can still be really great treatments that can get patients into remission and ongoing therapies with dosing adjustments again to decrease toxicity and improve quality of life and thinking about things like maintenance therapy as appropriate. 

Katherine Banwell:

What are the age restrictions, and why are they there? 

Dr. Alice Mims:

So, sometimes you will hear age 75. Really, no one above age 75 should move forward with transplant. And that’s based off of past data where they’ve explored transplant and seen increased toxicity. And from transplant in itself, increased side effects, increased risk of early mortality. And so, I do think it’s important to take the patient as a whole into consideration because again, you could have someone who’s 77 who may be running marathons, and in great shape, and not a lot of other healthcare issues, who may still do really well with treatment. And so, I think that’s – really needs to be taken in account, really the overall picture of health for the patient before making…  

Katherine Banwell:

So, the… 

Dr. Alice Mims:

…just a firm cutoff. 

Katherine Banwell:

Right. Okay. So, it’s not cut and dry. 

Dr. Alice Mims:

Exactly.  

Katherine Banwell:

If you’re 75 or older, then you definitely can’t have stem cell transplant. 

Dr. Alice Mims:

That’s correct. 

Katherine Banwell:

Then you’re looking at everyone individually. 

Dr. Alice Mims:

Yeah. So, it really should be looked at. And I still have some patients who will come to me and say, “Oh, I was told I’m 68 years old, I’m not a candidate.” And that always makes me take a step back. And then we kind of have to have that discussion again. And they may still not be a good candidate based off of other comorbidities or healthcare issues, but it shouldn’t just be a number rules you out for having that as an option. 

Katherine Banwell:

Good to know. We received this question from Carl, “What does treatment look like following transplant? And what are doctors looking for when monitoring through blood tests?”  

Dr. Alice Mims:

Sure. So, after transplant, the first three months is pretty intensive of being seen very frequently at your transplant center twice to once a week. You’re also on immunosuppressive medications to try to help prevent issues like graft-versus-host disease, which can be a complication from transplant. 

And then over time if you’re doing well, we try to start tapering off those immunosuppressive regimens to see if you can tolerate that. And what I say to most of my patients for – who are undergoing transplant, it can take some time to really feel back to being yourself. It can take six months, it can take a year or longer. And sometimes your normal is a new normal based off of how you do and the side effects of the transplant in itself. So, you may not go back to if you’re here before transplant and before your diagnosis, it may be that this is your new normal. Just so people can be prepared and know what they’re signing up for.  

Katherine Banwell:

And with the blood testing, what are you looking for when you’re monitoring a patient? 

Dr. Alice Mims:

Sure. There are a few different things that we’re looking for when monitoring patients. So, one, making sure that the stem cells or the graft from the donor are recovering. 

You want to see that blood counts, levels of white blood cells, red blood cells, platelets are getting to normal levels. You’re also assessing and making sure you’re not seeing signs of relapse. You’re checking levels of donor cells versus the patient cells within the stem cell — sorry, within the stem cell compartments. And so, we’re taking all of those into account as well as checking organ function and making sure there’s no signs of potential graft-versus-host disease as well.  

Katherine Banwell:

Ryan wants to know, “I’m a year-and-a-half post-transplant, how can you tell if the aches and pains in your joints are normal aging, host versusgraft disease, the AML returning, or even something else?” 

Dr. Alice Mims:

So, I think that’s also a difficult question to answer, because it really is patient-dependent. And so, I think if you’re having new joint aches or pains, it’s always important to reach out to your transplant team to make sure that – it could be any of the above. 

And so you’re doing the appropriate workup with lab work, imaging, things that would be appropriate, or seeing certain specialists. Maybe orthopedist if needed because it could be I’d say less likely leukemic relapse, but still want to be sure. But it could be definitely complications from GVHD, or there are some joint issues that can evolve post-transplant, especially for people who are on long-term immunosuppressant medications. Or it could be the normal effects of aging. So, it’s always good to have that reassurance.  

Expert Advice | Managing AML Symptoms and Treatment Side Effects

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Expert Advice | Managing AML Symptoms and Treatment Side Effects from Patient Empowerment Network on Vimeo.

The symptoms of acute myeloid leukemia (AML), or side effects of treatment, can have an impact on daily life. Dr. Alice Mims, an AML specialist, discusses how common issues are treated and talks about why it’s important to share what you’re going through with your healthcare team. 

Dr. Alice Mims is a hematologist specializing in acute and chronic myeloid conditions. Dr. Mims serves as the Acute Leukemia Clinical Research Director at The Ohio State University Comprehensive Cancer Center – James. Learn more about Dr. Mims. 

See More from Thrive AML

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 Thriving With AML | Advice for Setting Goals and Making Treatment Decisions

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Understanding AML Treatment Categories 

Transcript:

Katherine Banwell:

When it comes to living and thriving with AML, Dr. Mims, managing disease symptoms and treatment side effects is a big part of that. 

Would you talk about how symptoms and side effects can impact life with AML? 

Dr. Alice Mims:

Sure. So, I think from my perspective, what we are always trying to do when we’re moving forward with a treatment plan is of course, get patients into remission, but the purpose of getting into remission is not just to achieve that, but for patients to have quality of life. And so, there needs to be continued dialogue between the patient and the treatment team about how you’re feeling during treatment. Because they’re definitely based off of therapy, different side effects, things that could be not necessarily due to active leukemia anymore. And so there may need to be dose adjustments and other things that we do to the regimens in order to make you feel as good as possible while continuing on treatment. 

Katherine Banwell:

Why is it so important for patients to speak up about any issues they may be having? 

Dr. Alice Mims:

I think it’s important because you’re your own best advocate. Being the patient, being the person who’s living with having this diagnosis and going through the treatment, myself, or other colleagues as physicians, we can have a sense of what may be going on based off of numbers. But we’re not truly going to know how you’re feeling unless you speak up and let us know. And there may be things we could do with supportive medications, dosing adjustments as mentioned, that could help in making you hopefully feel better and less side effects and toxicities from treatment. 

Katherine Banwell:

What are some common symptoms and side effects that you hear about?  

Dr. Alice Mims:

Okay. Sure. So, different side effects that I would say that people can have, people can feel fatigued just from treatment in general. Some of our therapies can cause neuropathy, skin rashes, nausea, vomiting, diarrhea. And so, all of those are important along as mentioned with symptoms you may have from decreased blood counts that we do have interventions that we could implement to help the – make the therapy more tolerable. 

Katherine Banwell:

So, for the side effects like fatigue, for example, what do you do about that? 

Dr. Alice Mims:

So, I think it depends on the level of fatigue. Of course, we don’t have – I wish we had a pill that could just make fatigue improve. But if it’s really that the treatment is deriving it, and it’s impacting your quality of life, there are dose reductions or things we can do that may help with the level of fatigue you’re experiencing.  

Katherine Banwell:

And what about some of the other side effects. You mentioned diarrhea. 

Dr. Alice Mims:

Sure. 

Katherine Banwell:

How is that handled? 

Dr. Alice Mims:

Yeah. So, for issues from GI complications such as nausea, vomiting, diarrhea, we have really lots of choices for anti-nausea medicines and different combinations we can use or newer antiemetics that can help with that. And from a diarrhea perspective it depends on the treatment. But, of course, we want to make sure first and foremost there’s no infection. And if not, then there are good antidiarrheals we could add on to the regimen to help with that as well.  

Coping With AML | Financial and Mental Health Resources

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Coping With AML | Financial and Mental Health Resources from Patient Empowerment Network on Vimeo.

What emotional and financial support is available for patients with acute myeloid leukemia (AML)? Dr. Alice Mims shares advice about how to access mental health support and financial assistance for AML care.

Dr. Alice Mims is a hematologist specializing in acute and chronic myeloid conditions. Dr. Mims serves as the Acute Leukemia Clinical Research Director at The Ohio State University Comprehensive Cancer Center – James. Learn more about Dr. Mims.

See More from Thrive AML

Related Resources:

Thriving With AML | Advice for Setting Goals and Making Treatment Decisions

 Acute Myeloid Leukemia Care | Who Are the Essential Team Members

Transcript:

Katherine Banwell:

Let’s talk a little bit about mental health resources. Managing the worry associated with a diagnosis or concerns about relapse, or even various side effects can lead to emotional symptoms like anxiety and fear.  

Why is it important for people with AML to share how they’re feeling with their healthcare team? 

Dr. Alice Mims:

So, I think it’s very important because, one, all of those feelings are normal feelings. I think they’re sometimes that from going through such a rapid diagnosis and then having to start treatment pretty quickly and going through all the ups and downs with these types of diagnosis can really lead to for some patients PTSD-type symptoms. And then there are also things that can evolve over time where their anxiety or even survivorship guilt as you go if you move forward and are doing well, where you may have some friends or people you met along the way who may not have had as good outcomes. And so, there are resources available based off of where you are.  

But for survivorship, oncology-specific counseling to deal with some of these feelings that are understandable and normal for what patients have been through. 

Katherine Banwell:

Can a social worker help? And are there other people on the healthcare team who can support a patient’s emotional needs? 

Dr. Alice Mims:

Oh, absolutely. So, I think it’s really place-dependent on where you are but yes, absolutely. Social workers are a great resource for patients. There may be other collaborative teams based off of where you’re receiving your treatment that may be available that are maybe patient support groups where you can go and be with other patients or Facebook, social media support groups. And I think all those can be very helpful. And I know at least at our center, we also have patient mentors who have been through and gotten through to the other side of transplant or whatnot who are great resources, because they’ve lived and experienced it. 

And I think that’s just as a physician, I can talk about things that I don’t have that personal experience having lived through it. And I think that’s very important… 

Katherine Banwell:

Yeah. It’s a… 

Dr. Alice Mims:

…to be able to have somebody to talk to. Yeah. 

Katherine Banwell:

Yeah. What about the financial aspect of treatments? There are many people who would find it difficult to find and maybe they don’t have insurance, or their insurance doesn’t cover a lot. How do you help patients who are dealing with financial restrictions?  

Dr. Alice Mims:

Sure. So, I think that we’re fortunate here because we have a lot of support staff to help patients with our financial counseling team. We also have people within the medication assistance programs who can help find foundation grants to help with medication support, travel support. 

I think for patients who may not have those things available at their individual center, The Leukemia & Lymphoma Society is a great place to reach out for. And there are other foundations as well who at least may have navigators to help patients figure out other resources or funding available.