Efficacy and safety outcomes in Japanese patients with low-risk polycythemia vera treated with ropeginterferon alfa-2b

Int J Hematol. 2024 Aug;120(2):151-156. doi: 10.1007/s12185-024-03804-1. Epub 2024 Jul 1.

Abstract

Polycythemia vera (PV) is a Philadelphia chromosome-negative myeloproliferative neoplasm characterized by clonal erythrocytosis. A phase 2 study reported that ropeginterferon alfa-2b is a well-tolerated and effective treatment for PV in Japanese patients. This post hoc analysis of the phase 2 data further evaluated outcomes in patients at low risk of thrombosis (low-risk PV). Among 20 patients with low-risk PV, 60.0% (12/20) and 85.0% (17/20) achieved < 45% hematocrit by weeks 24 and 52, respectively. The proportion of responders with complete hematologic response (CHR) was 60.0% (12/20) at week 52, and the median time to response was 11.9 months. The mean JAK2 V617F allele burden decreased from 75.8% at baseline to 53.7% at week 52. No patient experienced thrombosis or bleeding episodes. All patients experienced treatment-emergent adverse events (TEAEs) related to ropeginterferon alfa-2b, but no grade ≥ 3 TEAEs or deaths related to ropeginterferon alfa-2b occurred, and no new safety concerns arose. This analysis indicated that ropeginterferon alfa-2b may be an effective treatment option for Japanese patients with low-risk PV.

Keywords: JAK2 V617F allele burden; Hematologic response; Low-risk; Polycythemia vera; Ropeginterferon alfa-2b.

Publication types

  • Clinical Trial, Phase II

MeSH terms

  • Adult
  • Aged
  • Asian People
  • East Asian People
  • Female
  • Humans
  • Interferon alpha-2* / administration & dosage
  • Interferon alpha-2* / adverse effects
  • Interferon alpha-2* / therapeutic use
  • Interferon-alpha* / administration & dosage
  • Interferon-alpha* / adverse effects
  • Interferon-alpha* / therapeutic use
  • Janus Kinase 2 / genetics
  • Japan
  • Male
  • Middle Aged
  • Polycythemia Vera* / drug therapy
  • Polyethylene Glycols* / administration & dosage
  • Polyethylene Glycols* / adverse effects
  • Polyethylene Glycols* / therapeutic use
  • Recombinant Proteins* / administration & dosage
  • Recombinant Proteins* / adverse effects
  • Recombinant Proteins* / therapeutic use
  • Treatment Outcome

Substances

  • Recombinant Proteins
  • Interferon-alpha
  • Polyethylene Glycols
  • Interferon alpha-2
  • peginterferon alfa-2b
  • Janus Kinase 2
  • JAK2 protein, human