Anemia is a well-recognized poor prognostic clinical variable in myelofibrosis (MF) and is present in approximately a quarter of MF patients at time of diagnosis and 75% during the course of disease. Red blood cell (RBC) transfusion dependence is captured as an independent risk factor within the Dynamic Prognostic Scoring System plus (DIPSS+) and contributes to significant reduction in QoL. Therefore, the alleviation of anemia is an important therapeutic goal for many patients with MF. Currently approved JAK2 inhibitors address spleen and symptom burden but do not correct anemia and in many cases can exacerbate this due to on-target myelosuppression. The use of recombinant erythropoietin, danazol and IMiDs are employed to improve hemoglobin levels and attain RBC transfusion independence with response rates of 20-40%, but are frequently limited in duration of response. Emerging data with the activin ligand trap, luspatercept, the JAK1/2 inhibitor, momelotinib, and the pan BET inhibitor, CPI-0610, suggest the possibility of novel approaches to addressing this unmet need in MF.
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