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The Myelodysplastic-Syndrome (MDS) condition center is a comprehensive resource for cancer news & oncologist insights on MDS. Read more about cancer at CURE.

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CURE’s myelodysplastic syndrome (MDS) page is an extensive resource of cancer information featuring the latest myelodysplastic syndrome news, expert oncologist insights on the latest cancer treatments, oncology research, advocacy efforts, and medical data on MDS.

Treatment with bexmarilimab and Vidaza elicited high overall response rates in both frontline and relapsed/refractory higher-risk myelodysplastic syndrome.

Bexmarilimab Plus Vidaza Elicit Responses in Myelodysplastic Syndromes

In an upside-down, topsy-turvy world, cancer can be a distraction for us.

Sometimes Cancer is a (Welcome) Distraction

The European Commission has approved Rytelo treatment in transfusion-dependent anemia due to very low–, low– or intermediate-risk myelodysplastic syndrome.

EU Approves Rytelo for Transfusion-Dependent Anemia in Low-Risk MDS

As a cancer survivor, I talk about where to look for the light when in utter darkness.

Looking For The Light After MDS

As a cancer survivor, I describe battling both cancer and infections and the toll it takes on my body.

Cancer is Hard Enough Without Infections

For patients with MDS, close monitoring by doctors may help determine the appropriate timing to undergo stem cell transplants before the disease worsens.

‘Tempo’ of MDS Helps With Timing for Stem Cell Transplants 

During the treatment process for patients with myelodysplastic syndromes (MDS), the time in which patients receive stem cell transplants matters, an expert told CURE® at the Society of Hematologic Oncology Annual Meeting.

However, many patients will have already met with an MDS specialist who has consistently monitored their disease over time, said Dr. Sergio A. Giralt.

Giralt is a professor of medicine at Weill Cornell Medical College and attending physician in the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center, both in New York.

Throughout the treatment process, Giralt noted that patients and their doctors begin to understand the “tempo of the disease” after months or years with MDS. By doing so, the team is able to determine whether there are progressive cytopenias (consistently low blood cell counts, including red blood cells, white blood cells and platelets).

He emphasized that certain potential progressive abnormalities in the body may lead to higher-risk MDS.

“Before that happens, one should intervene because this is the time when the patient’s going to be youngest and their disease is going to the be the weakest,” Giralt explained.

Regarding transplant eligibility in patients with MDS, he emphasized that patients’ fitness may weigh more than their age. Before deciding on whether transplants are an option, patients take some assessments — including a physical examination — to help doctors determine eligibility.

Well, for many patients, they've already established care with an MDS specialist and a transplant specialist who've monitored these patients for many months or years, and the tempo of the disease, one can see whether there is progressive cytopenias or progressive acquisition of other molecular abnormalities, or increase of high-risk molecular abnormalities, or development of cytogenetic abnormalities that suggest that this disease is going to evolve rapidly into a more higher risk [disease], and that before that happens, one should intervene because this is the time when the patient's going to be youngest and their disease is going to be the weakest.

More important than age is a patient's fitness, and this is determined by a comprehensive geriatric assessment and a full physical exam and assessment by the transplant center physician.

For more news on cancer updates, research and education, don’t forget to 

subscribe to CURE®’s newsletters here.

About two-thirds of patients with myelodysplastic syndrome (MDS) are not being given early treatment, according to Christopher Cogle, M.D., a professor of Medicine at the University of Florida Health.

Cogle was involved with a study that examined which patients were more likely to be treated with Vidaza (azacitidine), Dacogen (decitabine) or Revlimid (lenalidomide). While he initially thought that age and frailness would be the factors that were associated with not being treated, he actually found that it was patients with higher-risk disease, private insurance and genetic testing who were more likely to be prescribed one of the three agents.  

Many patients with myelodysplastic syndrome are not receiving early treatment.

Early Treatment for Myelodysplastic Syndrome: Many Patients Are Missing Out

It’s an exciting time in the field of myelodysplastic syndrome (MDS), explained Amy DeZern, M.D., associate professor of Oncology and Medicine at the Johns Hopkins University School of Medicine.

Researchers and practitioners alike are learning more about the biology of the disease, as well as the molecular mutations that come along with it. This is helping to differentiate different subtypes of MDS — it is not a heterogenous disease – which will lead to better diagnostic and prognostic accuracy, as well as paving the way for the use of targeted therapy in this space.

Learning more about MDS will lead to better diagnostics and more personalized treatments.

It's an Exciting Time for Myelodysplastic Syndrome

Rami S. Komrokji, M.D., clinical director of Hematologic Malignancies at Moffitt Cancer Center, discusses an unmet need in the treatment of myelodysplastic syndrome (MDS).

While some agents have made progress in the treatment of MDS, stem cell transplant is still the only curative method, and patients need more options. But now that researchers have spent the last few years better understanding the biology of the disease, there are some promising trials and treatments in the pipeline, Komrokji says. 

Filling an Unmet Need in MDS Treatment

Jason R. Gotlib, associate professor of Medicine (Hematology) at Stanford University Medical Center, discusses JAK inhibitors for the treatment of patients with myelofibrosis.

Gotlib discusses whether next-generation JAK inhibitors should be used after resistance to Jakafi (ruxolitinib) or in the frontline setting. Gotlib also provided an overview of drugs that are being studied in combination with these agents.

 in the category of Commitment to the Individual and is also an international leader in MPN research.

Jason R. Gotlib discusses JAK inhibitors for the treatment of patients with myelofibrosis.

Jason R. Gotlib on JAK Inhibitors for the Treatment of Myelofibrosis

Rebecca N. Claassen, Unit Educator at the University of Kansas Cancer Center, discusses the importance of educating nurses about myeloproliferative neoplasms (MPNs).

 in the category of Commitment to the Individual. Claassen has demonstrated outstanding support for patients with MPNs. As a dedicated Unit Educator at the University of Kansas Cancer Center, Claassen has brought a greater understanding of MPNs to the nursing staff. Her fervent advocacy and teaching about MPNs has gone beyond mere communication. Her efforts have helped colleagues make a positive impact on the quality of care for patients with MPNs.

In caring for patients, Claassen provides meaningful guidance about financial assistance, clinical trials, and treatment options, and support avenues to help them overcome daily challenges. In short, her tireless advocacy brings MPNs to the forefront for her peers and provides a sense of hope for patients.

Rebecca N. Claassen discusses the importance of educating nurses about myeloproliferative neoplasms (MPNs).

Rebecca N. Claassen on the Importance of Educating Nurses About MPNs

Susan Melvin Hill, patient advocate, discusses how she meets with and helps patients with myeloproliferative neoplasms (MPNs) in Idaho. Hill was named one of our 

 in the category of Commitment to the Broader MPN Community. Hill's journey began when she transitioned from myelofibrosis patient to dedicated patient advocate. In her dedication to help others with MPNs, Hill established Idaho’s first and only MPN support group. She currently mentors patients with MPNs in several states.

Hill continues to be involved in grassroots awareness activities. These include organizing the annual Rare Disease Day at the Idaho State Capitol, working to include MPN educational materials in the Leukemia & Lymphoma Society’s annual Light the Night Walk, supporting MPN Awareness Day with Governor’s Proclamations, and providing feedback on educational and patient advocacy materials for MPN publications. When she is not supporting MPN activities, Hill somehow finds the time to knit blankets for babies in the neonatal intensive care unit of her local hospital.

Susan Melvin Hill, patient advocate, discusses how she meets with and helps patients with myeloproliferative neoplasms (MPNs) in Idaho.

Susan Melvin Hill on Supporting MPN Patients in Idaho

I’d like to ask you a question about the multidisciplinary management of MDS [myelodysplastic syndrome]. What other members of the patient care team are involved, and what are their roles? For us at Cleveland Clinic, our core team includes some of the transfusion nurses and our pharmacists, who are part and parcel of the other issues in the drug-drug interactions. There are a number of people who are on our team who help care for patients. What is it like for you at The University of Texas MD Anderson Cancer Center in the management of your MDS patients?

 This is a fundamental question that we actually started on at the beginning of our conversation. Because our patients are older, they tend to have comorbidities. What we have learned through the work of many groups is that there might be some type of connection between these comorbidities and myelodysplastic syndrome. There might be a potential connection between heart disease and MDS and so forth, so this is being explored. This really emphasizes that we need to be able to provide multidisciplinary care.

I’m sure this is actually ordinary in a place like Cleveland Clinic, where you have not only top-notch leukemia doctors but also amazing cardiac care. We are a big cancer center, but this is a very important topic because this will probably translate into better outcomes. If we can integrate geriatric care for our very old patients or try to maximize the internal medicine needs of our patients, this is something we’ve been paying more attention to over the last couple of years. We used to disregard this, thinking that a diagnosis of MDS meant that we don’t have to worry much about your diabetes or high blood pressure. It turns out that it’s probably crucial. Having that kind of multidisciplinary care should be very important.

In our group, we are improving our integration of stem cell transplants. We’re starting to transplant older patients. Before, it was thought that if you were older than 50 years old, it was impossible. Then the threshold became 60. Now we’re starting to consider transplants in some patients over 70. Integrating the transplant team with the MDS leukemia team is really important. Earlier, you mentionedgenetic screening. Many of the centers have created leukemia programs where, for some patients with high-risk mutations, screening families is something that needs to be discussed.

It’s becoming a really complex multidisciplinary approach. From the typical group like you and me, you have nurses, physician assistants—the people who really provide the care. You also involve the transplant team, and in some cases, maybe hereditary clinics and geriatric internal medicine. This is really important. This kind of care is what will eventually translate into improved survival of our patients.

If it was in my family, this is what I would look for: clarity in terms of the diagnosis, a clinical trial that will help because we’re really far from curing a lot of our patients, and multidisciplinary care. Finally—I emphasized this at the beginning—the family support is critical. These are complex diseases. In older individuals, the conversation is difficult and having good family or friend support is really critical. Try to come to the clinic with another set of eyes and ears to help you and support you. I admire the families of our patients because without them, we would probably not be able to deliver a third of what we can. It’s critical to think about them as part of our team because they really are.

 What I hear you saying is recapitulated at our practice as well. The team is extensive. It’s first and foremost the patients and the caregivers around that patient at the home front who really help ensure that all the information needed to be communicated is being communicated directly from the physician team. That’s inclusive of the nursing support that’s helping give chemotherapy and transfusions but also the cardiac management, drug-drug interactions from pharmacists, and just making sure there aren’t issues with the medications. We spoke about the future for some patients, including therapies such as transplant or even clinical trials. It is really important to ask questions so that you have the answers. Pursue that. Don’t be shy about asking questions and advocating for yourself or your loved one. More than ever, we want to encourage the MDS community. We are receptive to answering those questions, and we want you to be engaged and involved in as deep a way as possible in the management of MDS.

I think that we’ve been fortunate at our home institution to have the incorporation of genetic counselors and also the incorporation of the bone marrow transplant teams. That helps us really support patients who are able to pursue those types of therapies and answers with regard to the underlying pathogenesis of MDS.

I’m grateful for the time we’ve had today to talk about the management of patients with MDS. We’ve covered a lot of ground in the last hour and a half, and I can’t underscore how fortunate we are as doctors. I want to echo the comments of Dr. Garcia-Manero. We learn a lot and are very privileged to help take care of patients with this diagnosis. We’re humbled by the grace that you all show us in sharing a difficult time. We learn from you all how to remain open. We’re just delighted to help at a time in your life where you can lean on physicians and caregivers to help you at a time where there is some uncertainty. But we do want to leave the message that we are incredibly hopeful and excited about the state of affairs with the treatment options for patients with MDS, as well as the future.

 I would like to echo that to close, Hetty. It’s been an honor to spend this time with you. You’ve been a great colleague for so many years, so it’s nice to see you. I would also like to say that we are living through a transformative period of time. For many years, we had 2 or 3 drugs, so the best we could do was better diagnose this disease and try to do the best in our research. But now it’s starting to pay off, so I am optimistic.

This is a serious condition, but we’re going through a transformation, and things are moving fast. It’s thanks to the academic researchers, the families, and the patients who were part of all this. Luspatercept was one of the first things that happened in many years, but it shows how perseverance is going to help us achieve better therapeutics for our patients. Thank you, Hetty.

A Multidisciplinary Team in Treating Myelodysplastic Syndromes

 I just want to acknowledge a lot of work that you’ve done in your career for patients with myelodysplastic syndrome [MDS] and ask you some questions about the lower doses that you’ve been investigating for patients with myelodysplastic syndrome. What has your experience been like? What might you share with the audience today about what your institution has done with regard to newer therapies for patients, whether it’s the lower-risk hypomethylating agent approach or novel therapies that we need to look for in the upcoming year or years?

 Thank you, Hetty. You’re too kind, because you’re part of this effort as much as I am. First, the message for the audience is that patients and their families need to be engaged. We talked earlier about how you need to be active with this disease. These are very complex disorders. Not to be critical, but many times, even hematologists may not be 100% on top of what’s happening or the latest. Medicine is getting very complex. Hetty and I treat two or three disorders. Beyond that, we probably don’t know much. So it’s really critical for the families and the patients to be engaged.

In my opinion, it’s really critical for families to ask these questions that we talked about earlier: “What is my risk? What are my chromosomes? What are my mutations?” You really need to ask the doctors and be aggressive in a positive way in terms of the therapy working, what is next, and if there’s a clinical trial close by that could help you. This is a big country, but we have big research centers all over that likely will have some degree of expertise or a clinical trial.

With regard to your specific question, we’re going through a transformation in our ability to treat patients with this disease. Maybe we can talk about high-risk disease someday. There are a lot of new doublets that are very effective. In low-risk disease, we’re seeing something that is eventually going to be very positive. You and I, through the MDS Clinical Research Consortium in the United States, work in these lower-risk trials and try to use the hypomethylating agents at a lower dose. That was an intuitive concept because we thought that lower doses could be effective and possibly less toxic. It turned out that, for some subset of patients with lower-risk disease, they can actually be beneficial.

We are also developing oral hypomethylating agents. Hypomethylating agents are given subcutaneously or through IV [intravenous] for three, five, or seven days a month. This gets tiring. The injections are short, but they take some time, and that may be a week of your month that goes away. So for many years, we’ve been working on oral hypomethylating agents. We now have two of these compounds that I think are going to help our patients very much, because they are oral and because they’re going to allow us to actually take a lower-intensity approach without coming in so many days during the month for the therapy. There is a revolution on treatments, and these treatments may be easier to receive and more patient-friendly. This is going to be a major breakthrough for our patients.

We believe that a lot of these are some type of combination where it’s not just one drug or the other. You may see combinations of all these compounds helping our patients and hopefully, at some point, transforming and improving their survival. Thank you for that, Hetty.

 Exciting things are to come in the upcoming years with regard to what therapies we might use in the up-front setting in ways that are different from the current state of affairs. Combination therapies are likely to emerge for patients with MDS, inclusive of some of the targets for the mutations on the blasts or the cancer cells themselves. They then allow for combinations with the backbones of therapy that we use. We have specific agents that are going to be targeting 

. Many of us in this space of MDS and AML [acute myeloid leukemia] are very excited about some of these 

 modulators that may be used in combination with hypomethylating-based therapy. Those studies are soon to emerge as well.

There are exciting things that are ongoing in the clinical trial space. There’s a lot of hope for the future for our patients with MDS, even with higher-risk MDS. It’s important, exciting, and at the forefront of research.

Possible Treatment Options for Myelodysplastic Syndromes

 I really appreciate your comments and you encouraging patients to enroll in clinical trials, because the clinical trials really lead to new drugs being FDA approved, like with luspatercept, and it’s very exciting. Can you share with me what your experience with luspatercept has been? When might you consider increasing the dose?As you’ve managed patients with this new drug, has there been a cut-off time due to a lack of response? What toxicities should patients be aware of with luspatercept?

 This drug is an injectable compound that you give every 3 weeks. The audience needs to realize that these ESAs [erythropoietin-stimulating agents] have been with us for 25 years or so—almost since I was a student. They’re basically imprinted in the practice of doctors. It’s not that they are good drugs or bad drugs or that we don’t know to use them. They are actually very important compounds that help a lot of our patients. But eventually, they either stop working or they do not work, and we know when that’s going to happen. Right now, luspatercept is used when these ESAs don’t work or with some patients for whom we can predict.

We’ve learned a number of things. Because it’s a new drug, we’re phasing in to what I’m about to say. We know that luspatercept works better in patients that have fewer transfusions. That’s why it’s so important for the physicians to realize that, at some point, you need to transition from a traditional ESA to luspatercept. I am not at all advocating to stop the ESA if it’s working to go for this other new drug. But we know that if the patient becomes transfusion dependent and is not responding to the ESA, we should probably consider transitioning earlier because that’s when you have a greater chance of response to this new compound.

Similar to the ESAs, based on algorithm, you could increase the dose that is based on the weight of the patient if you don’t see a response. This is described very well in the drug insert. If you don’t see an improvement in transfusions after a few weeks, you may ask the doctor if the dose should be increased.

These are not chemotherapies, so they don’t have the kind of toxicity that everybody associates with that kind of treatment, but luspatercept can have some side effects. In particular, I have seen in my practice that sometimes the patients may experience fatigue. This is important because the whole goal of the treatment is to improve the hemoglobin, improve quality of life, and have more energy. Unfortunately, some of our patients sometimes complain of feeling a little fatigued with this compound. It’s critical with this disease, and any other disease, that you describe the expectations up front every time you introduce a new drug, so you won’t have poor adherence or misunderstanding in terms of what the compound will do. I don’t think any fatalities have been reported from this drug. But fatigue is something I have seen in my patients. I don’t know about your experience, Hetty.

 So far, it’s been pretty well tolerated. You’re also struggling to come up with some heavy toxicity, and I think that’s teaching us that people tend to be tolerating it well. I do think it matters to escalate the therapy as you go through, and if the therapy isn’t helping patients, to be mindful of stopping it at those same time points that we talked about with ESA-based therapy. The every-three-week administration is nice for patients, although many of them, if they’re transfusion requiring, need to come and have their counts checked anyway. But from that standpoint, the administration is pretty straightforward for patients and the toxicities are pretty well managed.

 It’s a simple injection. You are right, the three weeks doesn’t mean that they may not require some checking in between, unfortunately.

Luspatercept in the Treatment of Myelodysplastic Syndromes

As we’ve talked about, the things we’re focusing on have been supportive measures to help decrease transfusion requirements, but ESA [erythropoiesis-stimulating agents]–based therapies are really not addressing the underlying disease. You smartly started with hypomethylating therapies and other agents in the high-risk category, where we try to modulate the disease rather than just treat a cytopenia. If I’m not mistaken, that’s what you were probably alluding to.

 You brought up a very important point: These bone marrows change in time. The blasts, chromosomes, and mutations can all change, so it’s crucial for the patients and audience to realize that, through the course of this disease, you may need to repeat a bone marrow test in addition to all the tests we were talking about earlier to know exactly where you are.

Hetty, you were talking about a new compound and new therapies for second-line treatment of anemia in patients with MDS [myelodysplastic syndromes]. You were saying that this was the first drug approved after 14 or 15 years. The study that led to that approval was called the MEDALIST trial. Perhaps you can teach us about the study and what we found.

 Earlier, you talked a little about this new agent, luspatercept, that was FDA approved. This agent is what we call a ligand trap. It works to inhibit the TGF-beta superfamily, which allows for the maturation of the red blood cell or the erythrocyte. This is a very special drug. In the first study, the PACE-MDS study, there were low or intermediate-1 MDS patients who were no longer responsive to this ESA-based therapy. We found that those patients were more likely to respond to luspatercept if they had the presence of ring sideroblasts or an 

 mutation. In the PACE study, they found that about 70% of patients with ring sideroblasts had an erythroid hematologic improvement, and about 40% of patients had transfusion independence with regard to red blood cell transfusions. The same numbers, in general, held true whether those patients had the presence of ring sideroblasts or an 

MDS

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