西罗莫司治疗蓝色橡皮泡痣综合征的有效性和安全性分析

Efficacy and Safety of Sirolimus in the Treatment of Blue Rubber Bubble Nevus Syndrome

目的探讨西罗莫司治疗蓝色橡皮泡痣综合征的有效性和安全性,为临床应用提供数据参考。方法检索pubmed、万方数据库及中国知网所有关于西罗莫司治疗蓝色橡皮泡痣综合征的中英文文献(1961-2018.7),总结和分析西罗莫司用药情况、临床应答及不良反应等资料。结果共检索出文献13篇,包括19例患者,其中男9例,女10例。全部患者均口服给药,平均用药年龄为(10.2±5.4)岁(17月龄至25岁),目标血药浓度1～15ng/m L。中位随访时间20月(2～54月),有效率为94.7%,其中16例(94.1%)患者改善贫血,15例(78.9%)患者血管瘤病变缩小或减少,6例(85.7%)患者病变部位疼痛减轻,1例(50%)患者凝血异常改善。共7例(36.8%)患者发生不良反应,分别为黏膜炎5例,严重软组织感染1例,血象改变2例(血小板减少1例,中性粒细胞减少2例)。严重软组织感染因此停药,其余不良反应轻微,药物减量后可改善,不影响治疗。结论西罗莫司治疗蓝色橡皮泡痣综合征的作用确切,虽不良反应发生率较高,但在治疗剂量下总体是安全的。

Objective To investigate the efficacy and safety of sirolimus in the treatment of blue rubber bubble nevus syndrome and to provide data for clinical application.Methods We searched the Chinese and English articles on sirolimus in the treatment of blue rubber bubble nevus syndrome from the pubmed,Wanfang database and China Zhiwang(1961-2018.7).And we summarized and analyzed the data of cirolimus medication,clinical response and adverse reactions.Results A total of 13 articles were searched,including 19 patients,9 males and 10 females.All the patients were orally administered,the average age was 10.2±5.4 years old(17-month old-25 years old),and the target blood concentration was 1-15 ng/ml.The median follow-up time was 20 months(2-54 months)and the effective rate was 94.7%.Among them,16 cases(94.1%)had improved anemia,15 cases(78.9%)had hemangioma lesion reduced or decreased,6 cases(85.7%)had pain relief and 1 case(50%)had abnormal coagulation improvement.There were 7 cases(36.8%)with adverse reactions,including 5 cases of mucositis,1 case of severe soft tissue infection,2 cases of hematologic changes(1 case of thrombocytopenia and 2 cases of neutropenia).As a result,patients with severe soft tissue infections were stopped,and the other patients had mild adverse reactions,which could be improved after drug reduction and did not affect the treatment.Conclusion Sirolimus has a definite effect in the treatment of blue rubber bubble nevus syndrome.Although the incidence of adverse reactions is high,it is generally safe under the treatment dose.