Department of Hematology, Oncology, Hemostaseology, and Stem Cell Transplantation, Faculty of Medicine, RWTH Aachen University, Aachen, Germany.
Center for Integrated Oncology, Aachen Bonn Cologne Düsseldorf (CIO ABCD), Aachen, Germany.
Ann Hematol. 2023 Dec;102(12):3383-3399. doi: 10.1007/s00277-023-05458-1. Epub 2023 Oct 4.
Ruxolitinib (RUX) is a Janus kinase 1/2 inhibitor (JAKi) approved in the EU for treating disease‑related splenomegaly or symptoms in adults patients with myelofibrosis (MF). This is an interim analysis of JAKoMo, a prospective, non‑interventional, phase IV study in MF. Between 2012-2019 (cutoff March 2021), 928 patients (JAKi-naïve and -pretreated) enrolled from 122 German centers. This analysis focuses on JAKi-naïve patients. RUX was administered according to the Summary of Product Characteristics. Compared to the COMFORT-I, -II, and JUMP trials, patients in JAKoMo were older (median 73 years), had poorer Eastern Cooperative Oncology Group (ECOG) performance statuses (16.5% had ECOG ≥ 2), and were more transfusion dependent (48.5%). JAKoMo represents the more challenging patients with MF encountered outside of interventional studies. However, patients with low-risk International Prognostic Scoring System (IPSS) scores or without palpable splenomegaly were also included. Following RUX treatment, 82.5% of patients experienced rapid (≤ 1 month), significant decreases in palpable spleen size, which remained durable for 24 months (60% patients). Symptom assessment scores improved significantly in Month 1 (median -5.2) up to Month 12 (-6.2). Common adverse events (AEs) were anemia (31.2%) and thrombocytopenia (28.6%). At cutoff, 54.3% of patients had terminated the study due to, death, AEs, or deterioration of health. No new safety signals were observed. Interim analysis of the JAKoMo study confirms RUX safety and efficacy in a representative cohort of real-world, elderly, JAKi-naïve patients with MF. Risk scores were used in less than half of the patients to initiate RUX treatment.Trial registration: NCT05044026; September 14, 2021.
芦可替尼（RUX）是一种 Janus 激酶 1/2 抑制剂（JAKi），在欧盟被批准用于治疗成人骨髓纤维化（MF）患者与疾病相关的脾肿大或症状。这是一项前瞻性、非干预性、IV 期 JAKoMo 研究的中期分析。在 2012-2019 年（截止到 2021 年 3 月）期间，来自 122 个德国中心的 928 名（JAKi 初治和预处理）患者入组。本分析重点关注 JAKi 初治患者。RUX 根据产品特性摘要进行给药。与 COMFORT-I、-II 和 JUMP 试验相比，JAKoMo 中的患者年龄更大（中位年龄 73 岁），东部合作肿瘤学组（ECOG）表现状态更差（16.5%的患者 ECOG≥2），并且更依赖输血（48.5%）。JAKoMo 代表了在干预性研究之外遇到的更具挑战性的 MF 患者。然而，也包括低风险国际预后评分系统（IPSS）评分或无可触及脾肿大的患者。在 RUX 治疗后，82.5%的患者出现快速（≤1 个月）、显著的可触及脾肿大缩小，这种缩小在 24 个月时仍然持久（60%的患者）。症状评估评分在第 1 个月（中位数-5.2）显著改善，直至第 12 个月（-6.2）。常见的不良反应（AE）是贫血（31.2%）和血小板减少症（28.6%）。截止时，由于死亡、AE 或健康状况恶化，54.3%的患者终止了研究。未观察到新的安全性信号。JAKoMo 研究的中期分析证实了 RUX 在代表性的真实世界、老年、JAKi 初治 MF 患者中的安全性和疗效。不到一半的患者使用风险评分来启动 RUX 治疗。
NCT05044026；2021 年 9 月 14 日。
