Hospital General Universitario Gregorio Marañón, Madrid, Spain.
Instituto de Investigación Gregorio Marañón, Madrid, Spain.
Am J Hematol. 2024 Dec;99(12):2328-2339. doi: 10.1002/ajh.27498. Epub 2024 Oct 12.
Avatrombopag is the newest thrombopoietin receptor agonist (TPO-RA) approved to treat immune thrombocytopenia (ITP). Real-world evidence regarding effectiveness/safety is limited. The Spanish ITP Group (GEPTI) performed a retrospective study with patients starting avatrombopag for the first time. A total of 268 ITP patients were recruited. The median (interquartile range [IQR]) follow-up time was 47.5 (30.4-58.9) weeks. Among the 193 patients with baseline platelet counts <50 × 10/L, 174 (90.1%) of them achieved response (PC ≥50 × 10/L), and 113 (87.6%) of the 129 who persisted on avatrombopag at last visit had platelet levels above such threshold. Results were similar when only those patients switching to avatrombopag due to previous treatment failure were considered (n = 104). Patients reached response in 13 (7-21) days. Among patients with baseline levels ≥50 × 10/L, 73/75 (97.3%) reported response, which was maintained by 53 (94.6%) of the 56 who continued on avatrombopag at the end of the study. Loss-of-response was always <10%. ITP duration did not influence response. Approximately 79% (34/43) of heavily pretreated (≥4 lines) patients with baseline platelet counts <50 × 10/L switching after previous failure achieved PC ≥50 × 10/L. Previous use of eltrombopag and/or romiplostim did not influence response, regardless of whether previous TPO-RA(s) succeeded or failed. Avatrombopag allowed dose-reduction/suspension of corticosteroids in 40/50 (80.0%) patients with baseline platelet counts <50 × 10/L. Overall, 40/268 (14.9%) thrombocytosis and 12/268 (4.5%) thromboembolic events were reported. Our real-world cohort supports the use of avatrombopag to manage ITP, regardless of disease severity and treatment history.
阿伐曲泊帕是最新批准用于治疗免疫性血小板减少症 (ITP) 的血小板生成素受体激动剂 (TPO-RA)。关于其有效性/安全性的真实世界证据有限。西班牙 ITP 小组 (GEPTI) 对首次使用阿伐曲泊帕的患者进行了回顾性研究。共纳入 268 例 ITP 患者。中位 (四分位距 [IQR]) 随访时间为 47.5 (30.4-58.9) 周。在基线血小板计数 <50×10/L 的 193 例患者中，174 例 (90.1%) 达到反应 (血小板计数 ≥50×10/L)，在最后一次就诊时继续使用阿伐曲泊帕的 129 例患者中，有 113 例 (87.6%) 血小板计数超过该阈值。仅考虑因先前治疗失败而改用阿伐曲泊帕的患者时，结果相似 (n=104)。患者在 13 (7-21) 天内达到反应。在基线水平≥50×10/L 的患者中，73/75 (97.3%) 报告有反应，在研究结束时继续使用阿伐曲泊帕的 56 例患者中，有 53 例 (94.6%) 保持反应。反应丢失始终<10%。ITP 持续时间不影响反应。大约 79% (34/43) 的基线血小板计数 <50×10/L、预处理≥4 线、以前失败的患者在改用阿伐曲泊帕后，血小板计数≥50×10/L。以前使用艾曲泊帕和/或罗米司亭不影响反应，无论以前的 TPO-RA(s) 是否成功。阿伐曲泊帕允许在基线血小板计数 <50×10/L 的 50 例患者中 (80.0%) 减少/停用皮质类固醇剂量。总体而言，报告了 40/268 (14.9%) 的血小板增多症和 12/268 (4.5%) 的血栓栓塞事件。我们的真实世界队列支持使用阿伐曲泊帕治疗 ITP，无论疾病严重程度和治疗史如何。
