Luspatercept shows promise in alleviating myelofibrosis-associated anemia and has a safety profile consistent with previous research, according to a study published in Blood Advances.
The most common therapeutics in myelofibrosis include erythropoiesis-stimulating agents, androgens, corticosteroids, and lenalidomide. However, many of these are associated with significant adverse events (AEs). Researchers are investigating therapeutic agents that are highly effective against anemia while having an acceptable safety profile.
Luspatercept is an erythropoietin maturation agent that has been approved in the United States for treating anemia in some individuals with myelodysplastic syndromes or beta-thalassemia who need red blood cell (RBC) transfusion. This therapeutic has been shown to induce transfusion independence in approximately 38% of patients. Researchers sought to explore if the success of luspatercept can be replicated in myelofibrosis and conducted a study to assess its use in patients with myelofibrosis-associated anemia, with or without transfusion dependence.
Researchers reported results from a phase 2, multicenter, open-label trial that assessed the use of luspatercept in myelofibrosis. They recruited adult patients with myeloproliferative neoplasm (MPN)-associated myelofibrosis who possessed an Eastern Cooperative Oncology Group performance status score of 2 or less and had evidence of anemia. Patients were divided according to their transfusion dependence status and whether they were on ruxolitinib therapy.
Participants received subcutaneous luspatercept at a dose of 1.0 mg/kg (with titration up to 1.75 mg/kg every 21 days for a total of 24 weeks). They were then assessed for their disease response at day 169; if they demonstrated clinical benefits, they could continue receiving luspatercept treatment for approximately 2 years longer. The primary endpoint of this study was anemia response at the end of the 24-week period.