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

Yoko Edahiro, Kohshi Ohishi, Akihiko Gotoh, Katsuto Takenaka, Hirohiko Shibayama, Takayuki Shimizu, Kensuke Usuki, Kazuya Shimoda, Masafumi Ito, Scott A. VanWart, Oleh Zagrijtschuk, Albert Qin, Hiroaki Kawase, Narihisa Miyachi, Toshiaki Sato, Norio Komatsu, Keita Kirito

Research output: Contribution to journalArticlepeer-review

Abstract

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.

Original languageEnglish
Pages (from-to)215-227
Number of pages13
JournalInternational journal of hematology
Volume116
Issue number2
DOIs
Publication statusPublished - 2022 Aug

Keywords

  • Hematologic response
  • JAK2 V617F allele burden
  • Molecular response
  • Polycythemia vera
  • Ropeginterferon alfa-2b

ASJC Scopus subject areas

  • Hematology

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