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SOHO 2026 | Insights into the biggest shifts and current unmet needs in the treatment landscape for LR-MDS

Valeria Santini, MD, University of Florence, Florence, Italy, shares insights into the evolving treatment landscape for low-risk myelodysplastic syndromes (LR-MDS), highlighting a shift toward therapies that may prolong survival, including luspatercept and erythropoiesis-stimulating agents (ESAs). Prof. Santini also notes the importance of characterizing and stratifying patients beyond traditional classification systems, as well as the potential of NEK7 inhibitors to address inflammation in LR-MDS. This interview took place at the 14th Annual Meeting of the Society of Hematologic Oncology (SOHO 2026) in Houston, TX.

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Transcript

It has changed, in fact. Thank you for your question because the, let’s say, the broad spectrum of options for low-risk MDS therapy has opened some doors that seemed to be closed and we actually gave more attention to patients with low-risk MDS. Let me explain. We were thinking that this patient could be decently treated just by supportive care or increasing their hemoglobin or platelets, but we never really took much attention, paid much attention, on the prolongation of survival and survival...

It has changed, in fact. Thank you for your question because the, let’s say, the broad spectrum of options for low-risk MDS therapy has opened some doors that seemed to be closed and we actually gave more attention to patients with low-risk MDS. Let me explain. We were thinking that this patient could be decently treated just by supportive care or increasing their hemoglobin or platelets, but we never really took much attention, paid much attention, on the prolongation of survival and survival. Although we cared for their quality of life, we never coupled it to a prolongation of survival. Now, the big shift has been that together with very active drugs, like luspatercept, but also with the optimal use of ESAs, we may actually obtain prolonged survival. And this is really a shift in the approach to therapy in low-risk MDS patients. And beside this shift, what was clear in the session, it was that we have more care in characterizing and stratifying different patients. So not only with a classification, not only by IPSS-M, but also by identifying specific mutations that can be targeted like IDH2 and IDH1 that have specific mutation inhibitors. So there are trials that are targeting IDH2 with very good results in terms of transfusion independence and the reticulocyte response. Of course, we are especially having good results in treating anemia, less for other cytopenias for low-risk MDS, but still the road seems to be going well ahead. And the attention is also posed on the inflammation, that is a well-known overarching phenomenon, especially on low-risk MDS. But we never had really active drugs in this sense. Many trials were attempted, blocking cytokines like interleukin-1 and others. But it seems that now the NEK7 inhibitor ofirnoflast that was presented during the session seems to be very active. It really seems that we have a novel tool to block the inflammasome and increase the hemoglobin level in these patients.

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