The following is a summary of “Rusfertide, a Hepcidin Mimetic, for Control of Erythrocytosis in Polycythemia Vera,” published in the February 2024 issue of Hematology by Kremyanskaya et al.

Researchers performed a retrospective study investigating the safety and effectiveness of rusfertide, an iron-regulating drug, in managing polycythemia vera patients who rely on phlebotomy.

They conducted part 1 of the international phase 2 REVIVE trial, enrolling patients for a 28-week assessment of rusfertide dosages. In part 2, patients were randomly assigned in a 1:1 ratio to receive rusfertide or placebo for 12 weeks in a double-blind manner. The primary efficacy endpoint was achieving a response, defined by hematocrit control, no need for phlebotomy, and completing the trial regimen during part 2. Patient-reported outcomes were evaluated using the modified Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) patient diary, with scores ranging from 0 to 10 indicating symptom severity.

The results showed 70 participants in part 1 of the trial; 59 were assigned to receive rusfertide (30 participants) or placebo (29 participants) in part 2. Before rusfertide, the estimated mean (±SD) number of phlebotomies per year was 8.7±2.9, dropping to 0.6±1.0 during part 1 (estimated difference, 8.1 phlebotomies per year). Mean maximum hematocrit declined from 44.5±2.2% to 50.0±5.8% pre-rusfertide. A response was noted in 60% of rusfertide recipients versus 17% of placebo recipients during part 2 (P=0.002). Rusfertide treatment decreased symptom scores on the MPN-SAF for patients with moderate or severe symptoms at baseline. Grade 3 adverse events occurred in 13% of participants during parts 1 and 2, with no instances of grade 4 or 5 events. Injection-site reactions of grade 1 or 2 were prevalent.

They concluded that rusfertide effectively controlled hematocrit levels in polycythemia vera patients, potentially offering a novel treatment option and reducing reliance on phlebotomy.

Source: nejm.org/doi/full/10.1056/NEJMoa2308809