Long-term outcomes of ropeginterferon alfa-2b-njft in polycythemia vera: a review of safety, efficacy, and potential disease modification

Polycythemia vera (PV) is a chronic myeloproliferative neoplasm characterized by erythrocytosis, thrombotic risk, and possible progression to aggressive myeloid neoplasms. Optimal management should achieve durable hematologic response, tolerability, and potential for long-term disease modification. Interferon alpha (IFN-α) is unique among current PV treatments for its disease-modifying potential; however, long-term tolerability concerns remain. Ropeginterferon alfa-2b-njft (ropeg), a novel monopegylated IFN-α, is the only IFN-α approved in the US for PV treatment. Phase III data from PROUD-PV (NCT01949805; EudraCT, 2012-005259-18) and its phase IIIb extension, CONTINUATION-PV (NCT02218047; EudraCT, 2014-001357-17), demonstrate that ropeg, compared with hydroxyurea, achieves hematologic response more slowly but provides greater, more durable responses after ∼18 months, underscoring the importance of long-term adherence. Early adverse events can challenge adherence, emphasizing the need for proactive symptom management. This review offers evidence- and experience-based perspectives on long-term ropeg treatment and adverse event management to support adherence and maximize therapeutic benefit in PV.