Management of Myelofibrosis-Associated Anemia: Focus on Standard Agents and Novel Therapeutics in Phase 3 Clinical Trials

AbstractPurpose of ReviewThe management of myelofibrosis is risk-adapted when considering transplant eligibility and symptom-directed, prioritizing the most burdensome symptoms for the patient. Unfortunately, myelofibrosis-anemia is common, multifactorial in its origin, and impactful regarding prognosis. While clinical trials are advised, not all patients have convenient access, and therefore, hematologists should be aware of the data supporting the use of conventional agents such as erythropoietin-stimulating agents, steroid treatments (danazol and prednisone), and immunomodulatory drugs (thalidomide and lenalidomide). This review summarizes the conventional approach to treating myelofibrosis-anemia and highlights recent data from 3 novel agents that are under phase 3 evaluation.Recent FindingsMomelotonib is a JAK1/2 and ACVR1 inhibitor that has demonstrated not only improvements in splenomegaly and symptoms, but also amelioration of anemia on the SIMPLIFY 1 and 2 clinical trial program. This may occur through suppression of hepcidin production. Luspatercept promotes late-stage hematopoiesis, and the phase 2 study has shown promise in ameliorating anemia as a monotherapy, and especially in combination with ruxolitinib. Finally, CP-0160, a BET inhibitor, has shown efficacy as an anemia-directed agent, when used as monotherapy and in combination. This agent reduces cytokine production and promotes erythroid differentiation. Results have been presented for patients previously t...
Source: Current Hematologic Malignancy Reports - Category: Hematology Source Type: research