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Luspatercept in patients with lower-risk myelodysplastic syndromes

New England Journal of Medicine Jan 16, 2020

Fenaux P, Platzbecker U, Mufti GJ, et al. - Experts performed a double-blind, placebo-controlled study to evaluate the utility and safety of luspatercept in patients with lower-risk myelodysplastic syndromes. Of 229 enrolled patients, 153 were randomized to receive luspatercept and 76 to receive placebo. The investigators noted balanced baseline patient characteristics. Interestingly, they observed transfusion independence for 8 weeks or longer in 38% of the patients in the luspatercept group vs 13% of those in the placebo group. Fatigue, diarrhea, asthenia, nausea, and dizziness were the most common luspatercept-associated adverse events. The severity of anemia was decreased by luspatercept in patients with lower-risk myelodysplastic syndromes with ring sideroblasts who had been receiving regular red-cell transfusions and who had disease that was refractory to or unlikely to respond to erythropoiesis-stimulating agents or who had discontinued such agents owing to an adverse event.

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