Polycythemia vera (PV) is a rare blood disorder characterized by excessive production of red blood cells, leading to various complications. The REVIVE study (PTG-300-04; NCT04057040) investigated the potential of rusfertide, a novel medication mimicking hepcidin, to control erythrocytosis in PV patients.
The REVIVE trial had three phases: 1, 2 (randomized withdrawal phase), and 3 (open-label extension). Eligible participants experienced at least 3 therapeutic phlebotomies (TPs) in the 28 weeks before the study, with or without cytoreductive therapy. The results of phase 2 were presented at the 11th Annual Meeting of the Society of Hematologic Oncology, Houston, Texas.
Patients received rusfertide via subcutaneous injections weekly. The rusfertide dosage was adjusted individually to maintain hematocrit levels below 45%.
In phase 2, patients were considered responders if they met specific criteria, including sustained HCT control without the need for TP and no TP required during 12 weeks of treatment.
A total of 53 participants completed Part 2 (27 received a placebo, and 26 received rusfertide). Rusfertide showed a significantly higher response rate (69.2%) compared to placebo (18.5%, P =.0003).
The drug’s efficacy was evident in patients receiving TP alone or TP with cytoreductive therapy, as it improved response maintenance, reduced TP necessity, and sustained HCT control.
Rusfertide was generally well-tolerated, with most treatment-related adverse events being mild to moderate.
The REVIVE study demonstrated that rusfertide effectively controls erythrocytosis in PV patients, particularly those requiring therapeutic phlebotomy. It offers potential symptom relief, reduces the need for invasive procedures, and is generally well-tolerated.
Pemmaraju N, Kremyanskaya M, Kuykendall A, et al. Targeted Therapy of Uncontrolled Erythrocytosis in Polycythemia Vera With the Hepcidin Mimetic, Rusfertide: Blinded Randomized Withdrawal Results of the Phase 2 Revive Study. Abstract MPN-541. Presented at the 11th Annual Meeting of the Society of Hematologic Oncology; September 6-9, 2023; Houston, Texas.