Deng Siying, Wang Bei, Hu Ziwei, Cai Shaozhe, Dong Lingli
Department of Rheumatology and Immunology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Arthritis Res Ther. 2025 Jan 10;27(1):7. doi: 10.1186/s13075-024-03472-w.
The objective of this study was to investigate the therapeutic effectiveness and safety profile of Eltrombopag, a thrombopoietin receptor agonist, as prolonged therapy in refractory CTD-ITP patients.
We conducted a pilot observational study of Eltrombopag in CTD-ITP patients who were unresponsive to or intolerant of conventional medications. Eltrombopag was administered orally at 25-75 mg/qd and adjusted on the basis of tolerance and efficacy until a minimum dosage of 25 mg/qd was reached. Clinical and laboratory data were collected and analysed monthly. The therapeutic response, relapse, and adverse events during the follow-up were also reviewed and evaluated.
Fifty-two patients were enrolled and followed monthly for a median of 6 months. Thirty-six (90%) patients achieved durable overall remission. The remission rates were 67.5% at month 1, 87.5% at month 2, 97.5% at month 3, and 95% at month 6. The platelet count of the patients improved significantly, with the median reaching 50 × 10/L within 2 weeks (p = 0.003). Disease activity indices were reduced in SLE and pSS patients (p = 0.016), allowing glucocorticoid tapering (p = 0.004). One patient had no response, four relapsed, and fifteen (28.8%) experienced clinically relevant adverse events. In the analyses, protopathy, comorbidity, and prior treatment were associated with efficacy.
For refractory CTD-ITP patients, Eltrombopag demonstrated significant clinical improvement, safety, and a steroid-sparing effect with prolonged use. Patient characteristics at baseline may affect treatment efficacy.
本研究的目的是调查血小板生成素受体激动剂艾曲泊帕作为难治性结缔组织病相关性免疫性血小板减少症（CTD-ITP）患者的长期治疗药物的疗效和安全性。
我们对常规药物治疗无效或不耐受的CTD-ITP患者进行了一项关于艾曲泊帕的初步观察性研究。艾曲泊帕口服给药，剂量为25-75毫克/天，并根据耐受性和疗效进行调整，直至达到最低剂量25毫克/天。每月收集并分析临床和实验室数据。还对随访期间的治疗反应、复发情况和不良事件进行了回顾和评估。
52例患者入组，每月进行随访，中位随访时间为6个月。36例（90%）患者实现了持久的完全缓解。缓解率在第1个月为67.5%，第2个月为87.5%，第3个月为97.5%，第6个月为95%。患者的血小板计数显著改善，中位数在2周内达到50×10⁹/L（p = 0.003）。系统性红斑狼疮（SLE）和原发性干燥综合征（pSS）患者的疾病活动指数降低（p = 0.016），糖皮质激素得以减量（p = 0.004）。1例患者无反应，4例复发，15例（28.8%）出现临床相关不良事件。在分析中，原发病、合并症和既往治疗与疗效相关。
对于难治性CTD-ITP患者，长期使用艾曲泊帕显示出显著的临床改善、安全性和激素节省效应。基线时的患者特征可能会影响治疗效果。
