我们报告了 2 期 COMBI II 临床试验 (#EudraCT2018-004150-13) 的两年研究结束结果，该试验研究了鲁索替尼和低剂量聚乙二醇化干扰素-α2a 联合治疗新诊断的真性红细胞增多症患者。主要结局是安全性，关键次要终点是疗效，基于血液学参数、生活质量测量和 JAK2V617F 变异等位基因频率 (VAF)。我们使用 2013 年 ELN 和 IWG-MRT 缓解标准。缓解标准包括症状、脾肿大、外周血计数和骨髓的缓解。我们纳入了 25 名 PV 患者，中位年龄为 70 岁；其中 5 人既往有血栓栓塞事件，3 人有 CT 证实的脾肿大。两名患者停止了两种研究药物，其中一名患者因进展为真性红斑狼疮后骨髓纤维化；只有该患者为 3 级感染。没有观察到带状疱疹感染事件。没有患者因精神症状而停止治疗。 24个月时外周血细胞计数缓解率为92%。使用 2013 年 ELN 和 IWG-MRT 缓解标准，14 例 (56%) 在 24 个月时实现缓解； 3 名（12%）获得完全缓解，11 名（44%）获得部分缓解。骨髓增生性肿瘤症状总症状评分中的以下项目显着降低：腹部不适、盗汗、瘙痒和骨痛。 JAK2V617F VAF 中位值从 47% (95% CI, 35-59%) 下降至 7% (95% CI, 3-15%)，60% 的患者达到分子缓解。总之，联合治疗提高了细胞计数；骨髓细胞构成和纤维化；并降低了 JAK2V617F VAF；对于真性红细胞增多症患者具有可接受的毒性。 EudraCT2018-004150-13.版权所有 © 2024 美国血液学会。

We report the two-year end-of-study results from the phase 2 COMBI II clinical trial (#EudraCT2018-004150-13) investigating the combination treatment of ruxolitinib and low-dose pegylated interferon-α2a in patients with newly diagnosed polycythemia vera. The primary outcome was safety and key secondary endpoints were efficacy, based on hematological parameters, quality of life measurements, and JAK2V617F variant allele frequency (VAF). We used the 2013 ELN and IWG-MRT remission criteria. The remission criteria included remissions in symptoms, splenomegaly, peripheral blood counts, and bone marrow. We included 25 patients with PV with a median age of 70 years; 5 of those had prior thromboembolic events and three had CT-verified splenomegaly. Two patients stopped both study drugs, one of these due to progression to post-PV myelofibrosis; only that patient had a grade 3 infection. No events of herpes zoster infections were observed. No patients discontinued treatment due to psychiatric symptoms. The peripheral blood cell count remission rate was 92% at 24 months. Using the 2013 ELN and IWG-MRT remission criteria, 14 (56%) achieved remission at 24 months; 3 (12%) achieved complete remission, and 11 (44%) achieved partial remission. The following items from the Myeloproliferative Neoplasm Symptom Total Symptom Score were significantly reduced: abdominal discomfort, night sweats, itching, and bone pain. The median JAK2V617F VAF decreased from 47% (95%CI, 35-59%) to 7% (95%CI, 3-15%), and 60% of patients achieved molecular remission. In conclusion, combination treatment improved cell counts; bone marrow cellularity, and fibrosis; and decreased JAK2V617F VAF; with acceptable toxicity in patients with polycythemia vera. EudraCT2018-004150-13.Copyright © 2024 American Society of Hematology.