Hong Tian, Lu Zhou, Jia-Wen Dai, Yun Li, Cheng-Yuan Gu, Dan-Qing Kong, Zi-Qiang Yu, Xiao-Fan Liu, Jie Yin
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引用次数: 0
摘要
由于在中国应用阿伐溴铂治疗免疫性血小板减少症(ITP)的实际研究有限,我们对阿伐溴铂在临床实践中的有效性和安全性进行了评估。我们纳入了三个医疗中心的 121 名接受 AVA 治疗的成年 ITP 患者。根据选择 AVA 的原因,这些患者被分为艾曲波帕(ELT)/赫曲波帕(HET)不耐受组(IG)和艾曲波帕/赫曲波帕无应答组(UG)。与 UG 组相比,IG 组中有更多患者有肝病史,且在 AVA 前接受的治疗次数较少。在所有患者中,83%的患者在 AVA 后血小板有反应(≥30 × 109/L),62%的患者达到完全反应(≥100 × 109/L,CR)。62%的IG患者和56%的UG患者能够停用一种以上的ITP并发症药物。共有 17 名患者多次更换血小板生成素受体激动剂(TPO-RA),血小板应答率为 88%。63 名患者停用了 AVA,其中 27% 是由于负担不起。大多数患者对 AVA 耐受良好。在 ITP 患者中,事实证明 AVA 有效且安全,尤其是对 ELT/HET 不耐受或无反应的患者。患者从 TPO-RAs 转换中获益,尤其是那些接受多次转换的患者。然而,许多患者在 AVA 的长期经济负担中挣扎。
Real-world evidence of avatrombopag for the treatment of immune thrombocytopenia intolerant or ineffective to eltrombopag/hetrombopag.
Due to the limited real-world research on the application of avatrombopag (AVA) for immune thrombocytopenia (ITP) in China, we evaluated the effectiveness and safety of AVA in clinical practice. We included 121 adult ITP patients treated with AVA across three medical centres. Based on the reasons for choosing AVA, these patients were divided into eltrombopag (ELT)/hetrombopag (HET) intolerance group (IG), and ELT/HET unresponsive group (UG). Compared with UG, more patients in IG had a history of liver disease and received fewer treatments before AVA. Amongst all patients, 83% had platelet response (≥30 × 109/L) after AVA and 62% achieved complete response (≥100 × 109/L, CR). Sixty-two percent in IG and 56% in UG were able to discontinue more than one concomitant ITP medication. A total of 17 patients underwent multiple switches of thrombopoietin receptor agonists (TPO-RAs), resulting in an 88% platelet response rate. Sixty-three patients discontinued AVA, 27% were due to unaffordability. AVA was well tolerated in most patients. In the ITP population, AVA proved effective and safe, particularly in patients intolerant or unresponsive to ELT/HET. Patients benefited from TPO-RAs switches, particularly those undergoing multiple switches. However, many patients struggled with the long-term financial burden of AVA.
期刊介绍:
The British Journal of Haematology publishes original research papers in clinical, laboratory and experimental haematology. The Journal also features annotations, reviews, short reports, images in haematology and Letters to the Editor.