Real-World Use and Outcome of Ropeginterferon ALFA-2B in Polycythemia VERA Patients: A Multicenteric Study

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Ropeginterferon alfa-2b (Ropeg) emerges as a novel, long-lasting mono-pegylated IFN approved in Italy for treating polycythemia vera (PV) in adult patients without symptomatic splenomegaly and reimbursed for PV patient intolerant to hydroxyurea (HU), women of childbearing potential, and individuals with a history of skin cancer (SC). Ropeg proved effective and safe in phase 3 trials, but more data is needed to understand its real-world use. The study collected clinical data to assess the safety and efficacy of Ropeg in PV patients across five hematological centers in Campania. Ropeg was administered as per the data sheet, complete hematological response (CHR) was evaluated according to ELN criteria. Additionally, HCT, PLT, WBC and molecular responses were analyzed separately. A total of 47 PV patients were enrolled, with a median age at Ropeg start of 60 years (range 28-90), and 70% (33/47) were male. Thirty patients switched from prior HU after a median time of 11.8 months (range 1-299), due to intolerance. Three patients also transitioned from ruxolitinib, and one from peg-IFN. Ropeg was used as first-line therapy for 3 women of childbearing age, 2 patients with prior SK, and 8 male patients who were intolerant to phlebotomies and declined HU. The median time from PV diagnosis and Ropeg initiation was 45 months (range 1-345). In the year prior to starting Ropeg, the median number of phlebotomies was 2 (range 1-5). The median time on Ropeg was 12 months (range 1-21), with the median starting dose being 125 mcg every two weeks (range 50-150). At 12 months CHR rate was 64.7% for the 17 evaluable patients, HCT response was 82.4%. Ten patients (21.2%) required additional phlebotomy sessions during the treatment. At the same time point PLT and WBC responses were 82.4% and 100%, respectively and all but one had JAK2V617F allele burden reduction. CHR rate, HCT, PLT and WBC response increased over treatment as well as the median Ropeg dose that at 12 months was 200 mcg (range 100-250). Three patients had grade 1 liver function test increases, which resolved after dose reduction, and one patient developed autoimmune thyroiditis without discontinuing treatment. After three months, four patients discontinued Ropeg for the following reasons: suspected Ropeg-related depression, gastrointestinal bleeding, worsening splenomegaly, autoimmune disease; one patient died from an unrelated cause. Our findings indicate that Ropeg is generally well-tolerated and shows improvements in hematologic parameters. Although some response rates may vary, the overall trend is positive, with several measures reaching complete or near-complete response over time.

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