Yoon Seug Yun, Yoon Sung-Soo, Yang Deok-Hwan, Lee Gyeong-Won, Sohn Sang Kyun, Shin Ho-Jin, Bae Sung Hwa, Choi Chul Won, Choi Eun-Ji, Cheong June-Won, Bang Soo-Mee, Park Joon Seong, Oh Suk Joong, Park Yong, Park Young Hoon, Lee Sung-Eun
Division of Hematology & Medical Oncology, Department of Internal Medicine, Soonchunhyang University Seoul Hospital, Seoul, Korea.
Department of Internal Medicine, Seoul National University College of Medicine, Seoul, Korea.
Int J Cancer. 2025 Aug 1;157(3):526-533. doi: 10.1002/ijc.35411. Epub 2025 Mar 15.
To prevent thrombosis in patients with polycythemia vera (PV), achieving a complete hematologic response (CHR) is highly recommended in practice. In addition, a reduced JAK2 V617F mutation burden is expected to have a disease-modifying effect, and its molecular response (MR) is currently of significant interest. This study aimed to assess the association between CHR and MR in patients with PV following treatment with ropeginterferon alfa-2b. This phase 2, single-arm, open-label, investigator-initiated trial was conducted at 16 sites in South Korea. Ninety-nine patients were treated with ropeginterferon alfa-2b subcutaneously every 2 weeks, at doses of 250 μg (week 1), 350 μg (week 3), and 500 μg (week 5), until week 48. CHRs were 27% (25/94), 46% (40/87), 56% (47/84), and 63% (51/81) at 12, 24, 36, and 48 weeks, respectively. The MR rates were 32% (28/88), 36% (29/81), 49% (38/77), and 57% (42/74) at 12, 24, 36, and 48 weeks, respectively. The Phi Coefficient for the association between CHR and MR was 0.6146 (p < .0001) at 48 weeks. In the subgroup analysis, patients with hydroxyurea resistance or intolerance, and those who were hydroxyurea-naïve, had similar results in terms of the CHR. In conclusion, CHR and MR were observed to be associated in patients with PV treated with ropeginterferon.
为预防真性红细胞增多症（PV）患者发生血栓形成，在实际应用中强烈建议实现完全血液学缓解（CHR）。此外，预计JAK2 V617F突变负担降低会产生疾病改善作用，其分子缓解（MR）目前备受关注。本研究旨在评估接受罗培戈干扰素α-2b治疗的PV患者中CHR与MR之间的关联。这项2期、单臂、开放标签、研究者发起的试验在韩国的16个地点进行。99例患者每2周皮下注射罗培戈干扰素α-2b，剂量分别为250μg（第1周）、350μg（第3周）和500μg（第5周），直至第48周。在第12、24、36和48周时，CHR分别为27%（25/94）、46%（40/87）、56%（47/84）和63%（51/81）。MR率在第12、24、36和48周时分别为32%（28/88）、36%（29/81）、49%（38/77）和57%（42/74）。在第48周时，CHR与MR之间关联的Phi系数为0.6146（p <.0001）。在亚组分析中，对羟基脲耐药或不耐受的患者以及未使用过羟基脲的患者，在CHR方面有相似结果。总之，在接受罗培戈干扰素治疗的PV患者中观察到CHR与MR相关。
