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Results from the 36-month follow-up of a single-arm, multicenter, phase II trial (NCT05485948) evaluating ropeginterferon alfa‑2b under a higher initial dose and accelerated titration (HIDAT) regimen in 49 patients with hydroxyurea (HU)-intolerant polycythemia vera (PV) were published in Blood Advances by Suo et al. Endpoints included reduction in JAK2V617F variant allele frequency (VAF) from baseline, complete molecular response (CMR) rate, partial molecular response (PMR) rate, complete hematologic remission (CHR) rate, event-free survival (EFS), and safety.
Key data: At 36 months, 97.7% of patients exhibited absolute reductions in JAK2V617F VAF from baseline. A molecular response was observed in 86% of patients, with 34.9% demonstrating CMR and 51.2% demonstrating PMR. CHR rates remained high from 27 months to 36 months (84.1% to 75.0%). Kaplan–Meier analysis showed 100% EFS in patients achieving CMR vs ~70% in those without molecular response (p = 0.035). Treatment-emergent adverse events (TEAEs) occurred in 81.8% of patients, none of which exceeded Grade 3 (9.1%, Grade 3 TEAEs).
Key learning: The HIDAT regimen of ropeginterferon alfa‑2b demonstrated sustained CHR rates, high CMR rates, and favorable tolerability in patients with HU-intolerant PV at 3 years
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