Individualized Treatment for MDS

EP: 1.Myelodysplastic Syndrome Diagnosis and Risk Assessment

EP: 2.Clinical Considerations in the Diagnosis of Patients with MDS

EP: 3.The Impact of Mutational Status on Treatment Considerations for MDS

EP: 4.Staging Models in Diagnosis and Risk Assessment of MDS

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EP: 5.Individualized Treatment for MDS

EP: 6.Guidelines for the Treatment of MDS

EP: 7.ESA Use in the Treatment of MDS

EP: 8.Luspatercept: An Erythroid Maturation Agent in MDS

EP: 9.Treatment of Transfusion Dependent MDS

EP: 10.Use of HMAs for the Treatment of MDS

EP: 11.HMAs in High-Risk MDS

EP: 12.Role of Transplant in MDS

EP: 13.Treatment in Older Patients with High-Risk MDS

EP: 14.Emerging Role of Venetoclax in the Treatment of MDS

EP: 15.Promising Data for Pevonedistat in High-Risk MDS

EP: 16.Unmet Needs in High-Risk MDS

Gail Roboz, MD: James, how are you - what are you guys doing? So, you have a patient where you've made the decision that this is an MDS patient that you're gonna support with transfusions. How do we get through this 20-minute argument? And the patient lands in a different hospital and then has to have a fight in the emergency room over the number? What's the number?

James Foran, MD: Yeah, it's a real issue, and it's a real problem. And I'm going to say something that's probably heretical. But I don't think that these Choosing Wisely Guidelines that say, "Don't give blood until seven grams and protect the blood supply." I do not think that's doing the right thing for patients. This is an individualized decision. It must be what keeps them functional, what keeps them getting dressed in the morning, what keeps them - if your performance status is NECCOG zero or one, a KPS 90 let's say, I'm OK with whatever your hemoglobin is. But anything less than that, add heart disease to that too. If there's heart disease, I'm automatically using eight or eight and a half grams as a bare minimum. Our blood bank's been very collaborative, but it's exceptional. Where nine - nine and a half grams, there's the rare patient who just needs to stay up there to maintain functional life in the community. Others do find it 7.8. So that's a case-by-case basis. In the hospital, different story. And in some of the small hospitals in Central Florida and South Georgia, try getting a transfusion for somebody if they're not actually dying in the moment, it's very hard to do. You're absolutely right. And that is a patient - patient discussion and decision and collaboration.

Gail Roboz, MD: And Amy at Hopkins, I have to have this. My New York patients are very, very different. Azra and I know this. The New York patients are just a different group. My patients will come in and start fighting with the, "Well, how old is this unit of red cells? Because I've read 12 papers that if you give me a new fresh unit versus an old unit, I will feel better for longer." So is there any - what is that? And do you guys have anything about age of the unit that comes into any conversation with people?

Amy DeZern, M.D., M.H.S.: Lots of our patients do bring up the age of the unit on occasion. But I think a lot of what James said is so important. It's just how individualized it is. I do try and deemphasize. Too much focus on the age of the unit. Because the reality is blood is a limited resource. And we sometimes especially in heavily transfuse MDS patients, do have to think about the antibody issue and what units are appropriately matched. But I sort of give a spiel about the life of a red blood cell is 42 days, and sometimes you get the seven day old and sometimes you get the 39 day old, but some transfusion episodes will augment your energy a little bit better. And that's part of the normal ebb and flow of MDS.

Gail Roboz, MD: I think we're all saying that patients have different functional status, they have different comorbidities, there are - there is a patient who is going to have a hemoglobin of 8.2, who's running around feeling great, no problems doing everything. And there's a patient who's going to have a hemoglobin of 8.2, who can't get up, who can't get out of bed, who can't reach up to take something off the shelf, because they're going to feel dizzy. We have to emphasize that. That it's the quality and the functional status and the comorbidities, not the number on the page, and that they shouldn't check their hemoglobin every hour and a half because that number does change a little bit and that can drive you crazy. The other thing that drives you crazy, though, and that patients are really worried about it as as they get it. They're getting transfused, this is not a curative therapy. They're trying to maintain their functional status, but they're worrying because they're tallying on their app or on their phone or on an index card, how many units there are and then they're wanting to check a ferritin every five minutes. I'm taking a nosedive into the hotbed controversy of collation. And 100 years ago, Azra and I were at a meeting. It was 100 years ago, and we were both offered in an incredibly small plastic cup, a swig of an oral chelator, as part of a meeting that we attended to see how non icky it tasted. And I sat there with my cup of chelation therapy thinking this is very weird. And I don't think I drank it and I'm not sure if you did, but are you chelating MDS patients and if so, how do you make that decision?

Azra Raza, MD: I mean, I think that chelation is one of the most dramatic medical revolutions for patients for example, with sickle cell disease and palicemia[ph]. It has really made a huge difference with the quality of their lives and to their total survival time. It's amazing. I think that in MDS, we have hugely underutilized the rule of chelation and that is because it is good for low risk MDS and it is almost impossible to prove the benefit of chelation, whether it's quality of life improvement or survival improvement, impossible in low risk MDS patients because the true lower risks can go on for years and years and there is no way to produce prospective trials to produce one or the other onset. That is why I think the field has suffered from under-treatment in terms of chelation. I'm very particular about it, I keep counting the kind of number of transfusions patient is receiving and in addition to that, of course, the ferritin level which can be misleading at times because it is an acute phase reactant and it can go up and down with many different things. The other thing is, patients are so well educated, especially in New York, some of them are completely obsessive about following the ferritin number. If it goes up by five points or dips by three, they have these mood swings also along with their ferritin levels. So, too much of concentration on a, on any one of these parameters is simply not practical or wise. I do think for chelation, we have to assess what is the risk that the patient has, are they going to, are we expecting them to survive at least six months to a year? Whether chelation is going to be important or not and then once we decide that, that yes, chelation is a good idea for this person giving their good risk for survival. Then the question is, how high is the ferritin? Some people use you know, the 20 units of blood and 1,000 mg of ferritin as the guiding light. Others, we use 2,500 or more ferritin consistently over several values that have been repeatedly obtained. I think these criteria need to be more tightened I feel, and people need to be more aware of the need for chelation. I'm very particular when patients have had 20 units of blood, then I start thinking and having a conversation with them and initiated as soon as they are ready. Of course, given that their liver and renal function and everything else is going to be good enough to tolerate the treatment.

Transcript edited for clarity.