Pomalidomide Efficacious in Hereditary Hemorrhagic Telangiectasia

MONDAY, Sept. 23, 2024 (HealthDay News) -- For patients with hereditary hemorrhagic telangiectasia (HHT), pomalidomide yields a significant reduction in epistaxis severity, according to a study published in the Sept. 19 issue of the New England Journal of Medicine.

Hanny Al-Samkari, M.D., from Massachusetts General Hospital in Boston, and colleagues conducted a randomized, placebo-controlled trial to assess the safety and efficacy of pomalidomide for HHT. Patients were randomly assigned to receive 24 weeks of pomalidomide at a dose of 4 mg daily or matching placebo (95 and 49 patients, respectively).

After a planned interim analysis met a prespecified threshold for efficacy, the trial was closed to enrollment in June 2023. The researchers found that the baseline mean Epistaxis Severity Score was 5.0 ± 1.5, consistent with moderate-to-severe epistaxis. The mean difference between the pomalidomide and placebo groups in the change from baseline in the Epistaxis Severity Score was −0.94 points at 24 weeks. The mean between-group difference in the change in the HHT-specific quality-of-life score was −1.4 points. The pomalidomide group more often had adverse events, including neutropenia, constipation, and rash.

"The benefits of pomalidomide were most apparent over the course of the second 12 weeks of the trial, persisted during the four weeks after the end of the treatment period, and were not dependent on HHT genotype or baseline epistaxis severity," the authors write.

Bristol Meyers Squibb donated the pomalidomide and matching placebo.

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