Chinese expert consensus on mTOR inhibitors for treating immune hematologic diseases
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摘要: 该共识由中华医学会血液学分会血栓与止血学组、中华医学会血液学分会红细胞疾病(贫血)学组发起，国内多位血液病临床专家联合商讨制定，旨在为mTOR抑制剂(如西罗莫司、依维莫司等)在免疫性血液病中的临床应用提供循证医学建议。共识系统总结了mTOR抑制剂的作用机制、药代动力学特性及在多种免疫性血液病中的疗效与安全性，包括免疫性血小板减少症(ITP)、自身免疫性溶血性贫血(AIHA)、纯红细胞再生障碍(PRCA)、自身免疫性淋巴细胞增生性综合征(ALPS)和移植物抗宿主病(GVHD)等。基于循证级别，共识推荐西罗莫司作为难治性ITP、AIHA、PRCA、ALPS及GVHD的二线或优选治疗方案，并强调需通过血药浓度监测平衡疗效与不良反应。此外，共识提出需关注mTOR抑制剂的药物相互作用、肝肾功能影响及感染预防，呼吁未来开展大样本研究以优化用药策略。Abstract: This consensus was initiated by the Thrombosis and Hemostasis Group and the Red Cell Diseases (Anemia) Group of the Society of Hematology, Chinese Medical Association, and was formulated through joint discussions by multiple domestic clinical hematology experts, to provide evidence-based recommendations for the clinical use of mTOR inhibitors(e. g., Sirolimus, Everolimus) in immune hematologic diseases. It comprehensively reviews the mechanisms of action, pharmacokinetics, efficacy and safety of mTOR inhibitors in treating conditions such as immune thrombocytopenia(ITP), autoimmune hemolytic anemia(AIHA), pure red cell aplasia(PRCA), autoimmune lymphoproliferative syndrome(ALPS) and graft-versus-host disease(GVHD). Supported by evidence levels, the consensus recommends sirolimus as a second-line or preferred option for refractory ITP, AIHA, PRCA, ALPS and GVHD, emphasizing the importance of therapeutic drug monitoring to balance efficacy and adverse effects. Additionally, the consensus highlights the need to monitor drug interactions, effects on liver and kidney function, and prevent infections when using mTOR inhibitors. It also calls for large-scale studies to refine medication strategies in the future.
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表 1 英国牛津大学循证医学中心证据分级和推荐标准(2009版)
推荐强度 证据级别 描述 A 1a 同质随机对照试验的系统评价 1b 单个随机对照试验(可信区间窄) 1c “全或无”的病例系列研究 B 2a 同质队列研究的系统评价 2b 单个队列研究(包括低质量随机对照试验如随访率 < 80%) 2c 结果研究或生态学研究 3a 同质病例-对照研究的系统评价 3b 单个病例-对照研究 C 4 病例系列研究(包括低质量队列或病例-对照研究) D 5 基于经验未经严格论证的专家意见或评论或基础实验 注：A~D表示推荐强度逐渐减弱；证据级别: 1~5级，分值越大证据越弱。 
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