Ropeginterferon versus Standard Therapy for Low-Risk Patients with Polycythemia Vera.

Area of Science:

• Hematology
• Oncology
• Clinical Trials

Background:

• Polycythemia vera (PV) management often involves phlebotomy to control hematocrit.
• The efficacy of phlebotomy alone in maintaining target hematocrit in low-risk PV is not well-established.

Purpose of the Study:

• To compare ropeginterferon alfa-2b (ropeg) with phlebotomy alone for maintaining hematocrit levels in PV patients.
• To assess the long-term efficacy and safety of ropeg versus phlebotomy in PV management.

Main Methods:

• A phase 2, open-label, randomized trial comparing ropeg (100 μg every 2 weeks) with phlebotomy.
• Primary endpoint: maintenance of median hematocrit ≤45% over 12 months.
• Crossover allowed if primary endpoint not met; follow-up to 24 months.

Main Results:

• Ropeg achieved the primary endpoint in 81% of patients, versus 51% in the phlebotomy group.
• Ropeg responders showed significantly fewer moderate/severe symptoms and palpable splenomegaly.
• Neutropenia occurred in 9.2% of ropeg patients; no dose-limiting toxicities were noted.

Conclusions:

• Ropeginterferon alfa-2b is superior to phlebotomy alone for maintaining target hematocrit in polycythemia vera.
• Ropeg treatment is associated with improved symptom burden and reduced spleen size.
• Ropeg demonstrates a favorable safety profile for long-term PV management.