Medical Center of Hematology, The Xinqiao Hospital of Army Medical University, Chongqing, People's Republic of China.
Department of Hematology, Affiliated Hospital of North Sichuan Medical College, Nanchong, People's Republic of China.
Hematology. 2022 Dec;27(1):1282-1289. doi: 10.1080/16078454.2022.2151230.
The complex pathogenesis of relapsed and refractory (R/R) immune thrombocytopenia (ITP) contributes to the varied efficacy and tolerability of current treatment regimens. Rapamycin, an immunomodulatory agent, was originally used in the prevention of organ rejection after organ transplantation. Additional evidence now shows that rapamycin can successfully treat R/R ITP. Here, we summarize recent clinical progress on the role and potential mechanism of rapamycin in the treatment of ITP.
PubMed, Web of Science and CNKI database were searched to identify eligible studies, and the clinical data and preclinical studies on the use of mTOR inhibitors in ITP treatment were reviewed. The key results (efficacy and safety) of the most recent clinical reports were summarized.
Case series provide evidence of the effectiveness and tolerable safety profile of rapamycin in ITP, including primary and some secondary ITP. Mechanistic explorations indicate that rapamycin can regulate immune cell subsets (Th1, Th2, Th17, Treg, Breg, MDSC, etc.), modulate cytokine secretion (IL-6, IL-10, TGF-β, BAFF, etc.) and promote platelet autophagy.
Emerging clinical data and basic studies suggest that rapamycin, as a multifaceted regulator, could provide a new promising option for the therapy of ITP. Additional research is needed to identify those patients which may benefit the most, as well as therapeutic regimens with which rapamycin may be combined.
复发性和难治性（R/R）免疫性血小板减少症（ITP）的复杂发病机制导致了当前治疗方案疗效和耐受性的差异。雷帕霉素是一种免疫调节剂，最初用于器官移植后预防器官排斥。现在有更多的证据表明雷帕霉素可以成功治疗 R/R ITP。在这里，我们总结了雷帕霉素在 ITP 治疗中的作用和潜在机制的最新临床进展。
通过搜索 PubMed、Web of Science 和 CNKI 数据库，确定了合格的研究，并对 mTOR 抑制剂在 ITP 治疗中的临床数据和临床前研究进行了回顾。总结了最近临床报告中最关键的结果（疗效和安全性）。
病例系列提供了雷帕霉素在 ITP 中的有效性和可耐受安全性的证据，包括原发性和一些继发性 ITP。机制探索表明，雷帕霉素可以调节免疫细胞亚群（Th1、Th2、Th17、Treg、Breg、MDSC 等），调节细胞因子分泌（IL-6、IL-10、TGF-β、BAFF 等）并促进血小板自噬。
新兴的临床数据和基础研究表明，雷帕霉素作为一种多效调节剂，可能为 ITP 的治疗提供一种新的有前途的选择。需要进一步研究以确定哪些患者可能受益最大，以及雷帕霉素可能与哪些治疗方案联合使用。
