THURSDAY, March 26, 2020 (HealthDay News) — For patients with transfusion-dependent β-thalassemia, significantly more have a reduction in transfusion burden with receipt of luspatercept versus placebo, according to a study published in the March 26 issue of the New England Journal of Medicine.

M. Domenica Cappellini, M.D., from the University of Milan, and colleagues randomly assigned adults with transfusion-dependent β-thalassemia to receive either best supportive care plus luspatercept (224 patients) or placebo (112 patients) for at least 48 weeks in a phase 3 trial. In both groups, luspatercept or placebo was administered for a median of about 64 weeks.

The researchers found that compared with the placebo group, in the luspatercept group, a significantly higher percentage of patients had a reduction in transfusion burden of at least 33 percent from baseline during weeks 13 through 24 plus a reduction of at least two red-cell units during this 12-week interval (21.4 versus 4.5 percent). The percentage of patients who had a reduction in transfusion burden of at least 33 percent was greater in the luspatercept versus placebo group during any 12-week interval (70.5 versus 29.5 percent), as was the percentage with a reduction of at least 50 percent (40.2 versus 6.3 percent).

“A five-year open-label extension phase is under way to provide long-term data on the safety of luspatercept and its effects on the transfusion burden and iron outcomes,” the authors write.

Several authors disclosed financial ties to pharmaceutical companies, including Celgene, the manufacturer of luspatercept, which funded the study in collaboration with Acceleron Pharma.

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