荟萃分析西罗莫司对获得性纯红细胞再生障碍性贫血的疗效及安全性  

A meta-analysis of efficacy and safety of sirolimus in acquired pure red cell aplasia(aPRCA)

作　　者：穆耶赛尔·玉苏普 何广胜[3] 秦玉婷[1,2] 尼罗帕尔·吐尔逊 郝建萍[1,2] Muyesaier Yusupu;HE Guangsheng;QIN Yuting;Niluopaer Tuerxun;HAO Jianping(Second Department of Hematology Center,the First Affiliated Hospital of Xinjiang Medical University,Urumqi 830054,China;Xinjiang Uyghur Autonomous Region Hematology Clinical Medical Research Center,Urumqi 830000,China;Department of Hematology,Jiangsu Provincial People's Hospital,Nanjing 210000,China)

机构地区：[1]新疆医科大学第一附属医院血液病中心血液二科,乌鲁木齐830054 [2]新疆维吾尔自治区血液病临床医学研究中心,乌鲁木齐830000 [3]江苏省人民医院血液科,南京210000

出　　处：《新疆医科大学学报》2025年第2期175-183,共9页Journal of Xinjiang Medical University

基　　金：新疆维吾尔自治区科技支疆项目(2022E02132)。

摘　　要：目的评价西罗莫司对获得性纯红细胞再生障碍性贫血(acquired Pure Red Cell Aplasia,aPRCA)的疗效及安全性。方法以“西罗莫司”、“aPRCA”和“治疗”作为关键词,按自由词和主题词结合的检索策略分别在PubMed、Cochrane Library、Embase、Web of Science等英文数据库和中国知网、万方等国内医学数据库进行检索,检索时限为自数据库建库以来至2024年8月。严格按照纳排标准筛选西罗莫司治疗aPRCA相关文献,采用STATA/MP16进行荟萃分析。根据异质性检验I2和Q检验结果,用固定效应模型或随机效应模型分析不同用药时间内西罗莫司在aPRCA中的有效率、完全缓解率以及相关并发症的发生率。最后用Begg′s和Egger′s检验和漏斗图评估纳入文献的发表偏倚。结果共纳入6篇文献,荟萃分析结果提示西罗莫司对aPRCA的有效率为73.4%(95%CI:0.651~0.811),完全缓解率为55.7%(95%CI:0.415~0.696);排除因缓解后、严重并发症停药等患者,在10月用药时间内西罗莫司的有效率高达79.8%(95%CI:0.720~0.868),完全缓解率为60.2%(95%CI:0.490~0.709);西罗莫司治疗aPRCA不良反应发生率15.1%(95%CI:0.043~0.294),因不良反应危及生命及停药情况非常少见。结论西罗莫司对aPRCA的治疗具有良好的疗效和安全性。Objective To evaluate the efficacy and safety of sirolimus in acquired pure red cell aplasia(aPRCA).Methods The keywords"sirolimus","aPRCA","treatment"were searched in English databases such as PubMed,Cochrane Library,Embase,Web of Science and domestic medical databases such as China Knowledge and Wanfang.According to the search strategy of combining free words and subject terms.The time frame of the search was from the establishment of the database to August 2024.Literature related to sirolimus treatment for aPRCA was screened strictly according to the inclusion and exclusion criteria,and meta-analysis was performed using STATA/MP16.Based on the results of the heterogeneity test I2 and Q test,the effectiveness rate,complete remission rate and the incidence of related complications of sirolimus in aPRCA at different dosing times were analyzed by fixed-effects model or randomeffects model.Finally,Begg's and Egger's tests and funnel plots were used to assess the publication bias of the included literature.Results A total of 6 papers were included in the meta-analysis.The results suggested that the effective rate of sirolimus in aPRCA was 73.4%(95%CI:0.651-0.811),and the complete remission rate was 55.7%(95%CI:0.415-0.696);excluding the patients who discontinued the drug due to remission and serious complications,the effective rate of sirolimus in 10-month exposure time for drug was up to 79.8%(95%CI:0.720-0.868),and the complete remission rate was 60.2%(95%CI:0.490-0.709);the incidence of adverse effects of sirolimus in aPRCA was 15.1%(95%CI:0.043-0.294)and life-threatening and discontinuation of medication were very rare.Conclusion Sirolimus has good efficacy and safety in the treatment of aPRCA.

关 键 词：纯红细胞再生障碍性贫血 西罗莫司 荟萃分析 疗效 安全性 

分 类 号：R556.5[医药卫生—血液循环系统疾病]