Myelofibrosis is a rare bone marrow cancer characterized by excessive scarring that disrupts the production of red blood cells. Current treatment options for these patients with anemia are limited. Luspatercept, which was recently approved in the U.S. and Europe, enhances erythropoiesis and has shown promise for treating these patients by increasing hemoglobin and reducing the need for transfusions. A phase 2 clinical trial published in Blood Advances has confirmed the safety and efficacy of luspatercept for anemic patients with myelofibrosis. Briefly, 95 adults with anemia caused by myelofibrosis (median age, 71 years; 61% male) were grouped into four cohorts. Cohorts 1 (n=22) and 3A (n=14) did not require transfusions to treat anemia; cohorts 2 (n=21) and 3B (n=38) were transfusion dependent (requiring 4-12 RBC units in the 12 weeks preceding the start of the study). All patients received luspatercept subcutaneously every 21 days for 24 weeks, and patients in cohorts 3A and 3B also received ruxolitinib, a Janus kinase inhibitor, before and during the study. Luspatercept improved anemia in patients with and without transfusion dependence. Median hemoglobin increased 0.8 g/dL and 1.2 g/dL in non-transfusion dependent patients without and with concomitant ruxolitinib treatment, respectively; and approximately half of all patients in both transfusion dependent cohorts had at least a 50% reduction in transfusion burden. After three years of follow-up, the safety of luspatercept was consistent with previous studies. Additional phase 3 studies of luspatercept for anemic patients with myelofibrosis receiving concomitant ruxolitinib are currently underway.
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