THURSDAY, Feb. 22, 2024 (HealthDay News) — For patients with polycythemia vera, rusfertide treatment reduces the use of phlebotomy and maintains hematocrit of less than 45 percent, according to a study published in the Feb. 22 issue of the New England Journal of Medicine.

Marina Kremyanskaya, M.D., Ph.D., from the Icahn School of Medicine at Mount Sinai in New York City, and colleagues examined the safety and efficacy of rusfertide in patients with phlebotomy-dependent polycythemia vera. Seventy patients were enrolled in a 28-week dose-finding assessment (part 1) and 59 were randomly assigned to receive rusfertide or placebo for 12 weeks in part 2 (30 and 29 patients, respectively).

The researchers found that the estimated mean number of phlebotomies per year was 8.7 ± 2.9 and 0.6 ± 1.0 during the 28 weeks before the first dose of rusfertide and during part 1, respectively. The corresponding mean maximum hematocrit was 50.0 ± 5.8 and 44.5 ± 2.2 percent. During part 2, 60 and 17 percent of the patients who received rusfertide and placebo, respectively, had a response. For patients with moderate or severe symptoms at baseline, rusfertide treatment was associated with a reduction in the individual symptom scores on the Myeloproliferative Neoplasm Symptom Assessment Form between baseline and the end of part 1. Grade 3 adverse events occurred in 13 percent of patients during parts 1 and 2; none had a grade 4 or 5 event.

“Rusfertide is a potentially effective treatment option for achieving and sustaining hematocrit control in patients with polycythemia vera, reducing the use of phlebotomy and the occurrence of debilitating disease-related symptoms,” the authors write.

The study was funded by Protagonist Therapeutics, which developed rusfertide.

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