Tarantino Michael D, Mosalpuria Kailash, Kolodny Scott, Zhang Jessica, Vredenburg Michael, Jamieson Brian D
Bleeding and Clotting Disorders Institute, Peoria, IL.
Nebraska Hematology Oncology Cancer Center, Lincoln, NE.
Blood Adv. 2025 Jun 10;9(11):2733-2743. doi: 10.1182/bloodadvances.2024015635.
This phase 4, multicenter, open-label study was conducted to evaluate the safety, efficacy, and treatment satisfaction of switching to avatrombopag from another thrombopoietin receptor agonist (TPO-RA) in patients with immune thrombocytopenia (ITP). Adults who had received ≥90 days of treatment with eltrombopag or romiplostim and had a response (2 platelet counts [PCs] ≥50 × 109/L) switched to avatrombopag with no protocol-defined washout period. The primary end point was the incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs. Secondary end points were the proportion of patients who had a PC between ≥50 × 109/L and ≤200 × 109/L (days 15, 30, 60, and 90) and change from baseline in each domain of the self-administered Treatment Satisfaction Questionnaire for Medication (TSQM) to day 90. Among 60 enrolled patients, 58.3% experienced TEAEs and 10.0% experienced serious TEAEs (1 related to avatrombopag [thrombocytopenia that resolved]; 5 unrelated [1 unrelated death]). A PC ≥50 × 109/L to ≤200 × 109/L was reported for 51.7%, 31.7% (mean PC, 256.2 × 109/L [standard deviation, 176.7 × 109/L]), 55.0%, 60.0%, and 55.0% at baseline and on days 15, 30, 60, and 90, respectively. TSQM scores increased from baseline to day 90 across all domains (mean change: convenience, +13.5; effectiveness, +14.4; global satisfaction, +14.2; side effects, +8.3). There was no correlation between stable avatrombopag dose (day 90) and previous TPO-RA dose (high or low). Patients with ITP may safely switch from another TPO-RA to avatrombopag and maintain adequate PCs while experiencing improved treatment satisfaction. This trial was registered at www.ClinicalTrials.gov as #NCT04638829.
这项4期、多中心、开放标签研究旨在评估免疫性血小板减少症（ITP）患者从另一种血小板生成素受体激动剂（TPO-RA）转换为阿伐曲泊帕的安全性、有效性和治疗满意度。接受艾曲泊帕或罗米司亭治疗≥90天且有反应（2次血小板计数[PC]≥50×10⁹/L）的成人患者，在无方案定义的洗脱期情况下转换为阿伐曲泊帕。主要终点是治疗中出现的不良事件（TEAE）和严重TEAE的发生率。次要终点是PC在≥50×10⁹/L至≤200×10⁹/L之间的患者比例（第15、30、60和90天）以及从基线到第90天自我管理的药物治疗满意度问卷（TSQM）各领域的变化。在60名入组患者中，58.3%经历了TEAE，10.0%经历了严重TEAE（1例与阿伐曲泊帕相关[血小板减少症已缓解]；5例不相关[1例不相关死亡]）。在基线、第15、30、60和90天，报告PC≥50×10⁹/L至≤200×10⁹/L的患者比例分别为51.7%、31.7%（平均PC，256.2×10⁹/L[标准差，176.7×10⁹/L]）、55.0%、60.0%和55.0%。TSQM评分在所有领域从基线到第90天均有所增加（平均变化：便利性，+13.5；有效性，+14.4；总体满意度，+14.2；副作用，+8.3）。稳定的阿伐曲泊帕剂量（第90天）与先前的TPO-RA剂量（高或低）之间无相关性。ITP患者可以安全地从另一种TPO-RA转换为阿伐曲泊帕，并在治疗满意度提高的同时维持足够的PC。该试验在www.ClinicalTrials.gov上注册为#NCT04638829。
