The New England Journal of MedicineRusfertide, a Hepcidin Mimetic, for Control of Erythrocytosis in Polycythemia Vera

Rusfertide’s Role in Erythrocytosis Management

The REVIVE trial offers a comprehensive examination of rusfertide, a hepcidin mimetic, in managing erythrocytosis in patients with polycythemia vera. This study highlights the potential of rusfertide to alter the treatment landscape for this myeloproliferative neoplasm by focusing on its efficacy and safety profile.

Study Design:

• International, phase 2 REVIVE trial, divided into a 28-week dose-finding phase and a 12-week double-blind randomized withdrawal phase.
• Enrolled 70 patients in the dose-finding phase; 59 proceeded to the randomized phase, receiving either rusfertide or placebo.
• Primary efficacy endpoint: hematocrit control, absence of phlebotomy, and trial regimen completion during the randomized phase.
• Patient-reported outcomes measured using the modified Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF).

Key Findings:

• Significant reduction in the number of phlebotomies per year from 8.7 before rusfertide to 0.6 during the trial.
• Mean maximum hematocrit decreased from 50.0% pre-treatment to 44.5% during the study.
• 60% response rate in the rusfertide group compared to 17% in the placebo group during the randomized phase (P=0.002).
• Improvement in symptom scores on the MPN-SAF for patients with moderate or severe symptoms at baseline.
• Grade 3 adverse events occurred in 13% of patients; no grade 4 or 5 events reported. Injection-site reactions were the most common side effects.

HCN Medical Memo
Rusfertide treatment in polycythemia vera patients resulted in maintained hematocrit levels below 45% over 28 weeks and showed higher response rates compared to placebo during a 12-week study period.

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