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英国成人免疫性血小板减少症患者使用血小板生成素受体激动剂的真实世界数据:TRAIT 研究结果。

Real-world use of thrombopoietin receptor agonists for the management of immune thrombocytopenia in adult patients in the United Kingdom: Results from the TRAIT study.

机构信息

Imperial College Healthcare NHS Trust, London, UK.

Haematology Programme, NIHR UCLH/UCL BRC, London, UK.

出版信息

Br J Haematol. 2024 Jun;204(6):2442-2452. doi: 10.1111/bjh.19345. Epub 2024 Mar 1.

DOI:10.1111/bjh.19345
PMID:38429869
Abstract

Few studies have reported the real-world use of both romiplostim and eltrombopag in immune thrombocytopenia (ITP). TRAIT was a retrospective observational study aimed to evaluate the platelet responses and adverse effects associated with the use of these thrombopoietin receptor agonists (TPO-RAs) in adult patients with ITP in the United Kingdom. Of 267 patients (median age at diagnosis, 48 years) with ITP (primary ITP [n = 218], secondary ITP [n = 49]) included in the study, 112 (42%) received eltrombopag and 155 (58%) received romiplostim as the first prescribed TPO-RA. A platelet count ≥30 × 10/L was achieved in 89% of patients with the first TPO-RA treatments, while 68% achieved a platelet count ≥100 × 10/L. Treatment-free response (TFR; platelet count ≥30 × 10/L, 3 months after discontinuing treatment) was achieved by 18% of the total patients. Overall, 61 patients (23%) switched TPO-RAs, most of whom achieved platelet counts ≥30 × 10/L with the second TPO-RA (23/25 who switched from eltrombopag to romiplostim [92%]; 28/36 who switched from romiplostim to eltrombopag [78%]). TFR was associated with secondary ITP, early TPO-RA initiation after diagnosis, the presence of comorbidity and no prior splenectomy or treatment with steroids or mycophenolate mofetil. Both TPO-RAs had similar efficacy and safety profiles to those reported in clinical studies.

摘要

很少有研究报告过罗米司亭和艾曲泊帕在免疫性血小板减少症(ITP)中的真实世界应用。TRAIT 是一项回顾性观察性研究,旨在评估血小板反应和不良反应与这两种血小板生成素受体激动剂(TPO-RA)在英国成人 ITP 患者中的应用相关。在这项研究中,纳入了 267 名(诊断时的中位年龄为 48 岁)ITP 患者(原发性 ITP [n=218],继发性 ITP [n=49]),其中 112 名(42%)接受艾曲泊帕治疗,155 名(58%)接受罗米司亭作为首选用药 TPO-RA。第一次 TPO-RA 治疗后,89%的患者血小板计数达到≥30×10/L,而 68%的患者血小板计数达到≥100×10/L。总共有 18%的患者达到了无治疗反应(TFR;停药后 3 个月血小板计数≥30×10/L)。总体而言,61 名患者(23%)更换了 TPO-RA,其中大多数患者在接受第二种 TPO-RA 治疗后达到了血小板计数≥30×10/L(25 名从艾曲泊帕换用罗米司亭的患者中有 23 名[92%];36 名从罗米司亭换用艾曲泊帕的患者中有 28 名[78%])。TFR 与继发性 ITP、诊断后早期开始 TPO-RA 治疗、合并症存在以及无脾切除术史或接受过皮质类固醇或霉酚酸酯治疗有关。两种 TPO-RA 的疗效和安全性与临床试验报告的相似。

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