Efficacy and Safety of Ropeginterferon Alfa-2b in Low-Risk Polycythemia Vera.
Key claim: Ropeginterferon alfa-2b substantially cuts phlebotomy need and achieves high complete hematologic response rates with manageable safety in low-risk polycythemia vera patients requiring cytoreduction.
Abstract
PURPOSE: Conventional management of low-risk polycythemia vera (PV), consisting of phlebotomy and aspirin, often fails to adequately control symptoms and hematologic parameters. This study evaluated the efficacy and safety of ropeginterferon alfa-2b (Ropeg) in low-risk PV patients requiring cytoreductive therapy. MATERIALS AND METHODS: In this sub-analysis of an open-label, multicenter trial, 42 patients received Ropeg for 48 weeks. The primary endpoint was a reduction in phlebotomy frequency, while secondary endpoints included complete hematologic response (CHR), changes in JAK2V617F allele burden, and safety. RESULTS: Among 42 patients, Ropeg significantly reduced mean phlebotomy frequency per year from 3.0 to 0.5 (p < 0.05) and improved CHR rates (69.1% at 48 weeks). The JAK2V617F allele burden decreased, and hydroxyurea-naïve patients showed better responses. The most frequently reported treatment-related adverse events included elevated liver enzymes and alopecia. Most adverse events were mild or moderate, with no grade 4 or 5 events reported. CONCLUSION: These findings suggest that Ropeg is a promising treatment option for low-risk PV by effectively reducing the need for phlebotomy and demonstrating efficacy and safety.
Why this matters
Low-risk PV has traditionally been managed with phlebotomy plus aspirin, with cytoreduction reserved for high-risk or phlebotomy-intolerant patients. Prospective evidence that ropeginterferon alfa-2b delivers high complete hematologic response and phlebotomy independence with acceptable safety in the low-risk subset strengthens the case for earlier disease-modifying intervention in PV natural history, aligning with the broader ropeg-first paradigm shift emerging from PROUD/CONTINUATION-PV, Low-PV, and cross-study analyses.