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ASH 2025 | Unmet needs in LR-MDS: the need for strategies to treat anemia
Moshe Mittelman, MD, Tel Aviv Sourasky Medical Center, Tel Aviv, Israel, discusses the unmet needs in the treatment of patients with lower-risk myelodysplastic syndromes (MDS). He highlights that the majority of these patients are anemic and require transfusions, with current treatments having limited response rates and durations. This interview took place at the 67th ASH Annual Meeting and Exposition, held in Orlando, FL.
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Transcript
Well, as you know, patients with lower-risk MDS, all of them are anemic. 90-95%, I should say, of 100% are anemic, and many of them require transfusions. About 30-50% require transfusions at presentation, and others along the course of the disease. So yes, we need treatments. Now we have, for about two or three decades, we have erythroid-stimulating agents for these patients. They are effective, but they are effective in no more than 40-50% of the patients...
Well, as you know, patients with lower-risk MDS, all of them are anemic. 90-95%, I should say, of 100% are anemic, and many of them require transfusions. About 30-50% require transfusions at presentation, and others along the course of the disease. So yes, we need treatments. Now we have, for about two or three decades, we have erythroid-stimulating agents for these patients. They are effective, but they are effective in no more than 40-50% of the patients. And the duration of the effect is no more than two years at best. Since we are talking about patients with lower risk, and they all live several good years, it means that many of them remain without treatment after that. Now, we have already the luspatercept which has been approved, but again, the response rate is no more than 40%. The response rate is probably one year, maybe a little bit more. So again, we are left with patients for whom we do not have good options. Recently, imetelstat was approved by the FDA, but still, it is not approved in many countries in Europe, as well as in my country, Israel. And these patients indeed have a problem. The only one possibility after they fail or after they stop responding to these agents is transfusions. Sometimes we are trying to treat them with hypomethylating agents, and if they have a higher risk, this is not a good option. So the bottom line, there is still an unmet need for effective, easy-to-use agents that might address the anemia of patients with lower-risk. A small percentage of patients have thrombocytopenia. Fortunately, a relatively small percentage. Unfortunately, we do not have good agents for these patients. We used to have two agents which could treat the thrombocytopenia, but the development of both of them has been stopped. And currently, unfortunately, we don’t have a good solution for the thrombocytopenia. So I would say the major unmet need is for the anemia after the patients do not respond anymore to these agents which I have mentioned and in a smaller percentage thrombocytopenia.
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