Efficacy and safety of ropeginterferon alfa-2b in Japanese patients with polycythemia vera: an open-label, single-arm, phase 2 study

• Published in: International journal of hematology 2022-08, Vol.116 (2), p.215-227
• Main Authors: Edahiro, Yoko, Ohishi, Kohshi, Gotoh, Akihiko, Takenaka, Katsuto, Shibayama, Hirohiko, Shimizu, Takayuki, Usuki, Kensuke, Shimoda, Kazuya, Ito, Masafumi, VanWart, Scott A., Zagrijtschuk, Oleh, Qin, Albert, Kawase, Hiroaki, Miyachi, Narihisa, Sato, Toshiaki, Komatsu, Norio, Kirito, Keita
• Format: Article
• Language: English
• Subjects: Adverse events; Blood; Blood cancer; Effectiveness; Hematology; Interferon; Janus kinase 2; Kinases; Labels; Medicine; Medicine & Public Health; Oncology; Original Article; Patient safety; Patients; Phlebotomy; Polycythemia; Polycythemia vera; Safety; Thromboembolism; Thrombosis; α-Interferon
• ISSN: 0925-5710; 1865-3774
• DOI: 10.1007/s12185-022-03341-9

Ropeginterferon alfa-2b is a novel, site-selective, monopegylated recombinant human interferon alfa-2b. Safety and efficacy of ropeginterferon alfa-2b for the treatment of polycythemia vera were demonstrated in clinical studies conducted in European countries, but clinical studies in Japanese patients are lacking. This phase 2, open-label, multicenter, single-arm study investigated the safety and efficacy of ropeginterferon alfa-2b in 29 Japanese patients with polycythemia vera including young patients and patients with low thrombosis risk who are difficult to receive guideline-based standard treatments. The primary outcome of durable complete hematologic response without phlebotomy at months 9 and 12 was achieved by 8/29 (27.6%) patients. The fastest complete hematologic response was observed at week 12. A corresponding reduction in the JAK2 V617F allele burden from baseline to 52 weeks was also observed (mean ± standard deviation =  − 19.2% ± 22.6%). No new safety concerns were identified in Japanese patients when compared with previous studies of ropeginterferon alfa-2b in European populations; the most common treatment-related adverse events were alopecia (55.2%), fatigue (27.6%) and influenza-like illness (27.6%). Most treatment-related adverse events were mild or moderate, with none of grade ≥ 3. Ropeginterferon alfa-2b is a safe and efficacious treatment option in Japanese patients with polycythemia vera.