Department of Hematology and Oncology, University of Yamanashi, 1110 Shimokato, Chuo, Yamanashi, 409-3898, Japan.
Department of Hematology and Oncology, Mie University Graduate School of Medicine, 2-174 Edobashi, Tsu, Mie, 514-8507, Japan.
Int J Hematol. 2024 Dec;120(6):675-683. doi: 10.1007/s12185-024-03846-5. Epub 2024 Oct 3.
Ropeginterferon alfa-2b (ropegIFN), a new-generation interferon-based agent, has been approved in Japan for patients with polycythemia vera (PV) who are ineligible for or respond inadequately to conventional treatment. However, long-term outcomes with ropegIFN in Japanese patients have not been reported. This extension of a phase 2 study of ropegIFN in Japanese patients with PV aimed to determine its long-term safety/efficacy, and changes over time in JAK2 V617F allele burden. Here, we report data from the phase 2 study and subsequent extension over a period of 36 months. The primary endpoint was the complete hematologic response (CHR) maintenance rate without phlebotomy (hematocrit value < 45% without phlebotomy during the previous 12 weeks, platelet count ≤ 400 × 10/L, and white blood cell count ≤ 10 × 10/L). The CHR maintenance rates were 8/27 (29.6%), 18/27 (66.7%), and 22/27 (81.5%) at 12, 24, and 36 months, respectively. No thrombotic or hemorrhagic events occurred. The median allele burden change from baseline was - 74.8% at 36 months. All patients experienced adverse events; 25/27 (92.6%) experienced adverse drug reactions (ADRs), but no serious ADRs or deaths occurred. This interim analysis demonstrated the safety and efficacy of ropegIFN over 36 months in Japanese patients with PV.
罗匹尼罗干扰素 alfa-2b（ropegIFN）是一种新一代基于干扰素的药物，已在日本获批用于不适合或对常规治疗反应不足的真性红细胞增多症（PV）患者。然而，尚未报道日本患者使用罗匹尼罗干扰素的长期结果。本研究旨在评估罗匹尼罗干扰素治疗日本 PV 患者的长期安全性/疗效，以及 JAK2 V617F 等位基因负担随时间的变化。在此，我们报告了一项罗匹尼罗干扰素治疗日本 PV 患者的 2 期研究的扩展数据，随访时间为 36 个月。主要终点是不进行放血的完全血液学缓解（CHR）维持率（在过去 12 周内未进行放血时的血细胞比容值＜45%，血小板计数≤400×10/L，白细胞计数≤10×10/L）。在 12、24 和 36 个月时，CHR 维持率分别为 8/27（29.6%）、18/27（66.7%）和 22/27（81.5%）。没有发生血栓或出血事件。从基线到 36 个月时，等位基因负担中位数变化为-74.8%。所有患者均发生不良事件；27 例患者中有 25 例（92.6%）发生药物不良反应（ADR），但没有发生严重 ADR 或死亡。这项中期分析表明，罗匹尼罗干扰素在日本 PV 患者中使用 36 个月是安全有效的。
