介入栓塞联合西罗莫司治疗难治性Kasabach-Merritt综合征的疗效及安全性

Treatment of refractory Kasabach-Merritt syndrome with transcatheter arterial embolization plus sirolimus therapy

目的 探讨介入栓塞联合西罗莫司治疗难治性婴幼儿Kasabach-Merritt综合征（KMS）的疗效及其安全性.方法 回顾性分析2015年12月至2016年10月接受治疗的12例难治性婴幼儿KMS患儿临床资料.12例均对激素等传统治疗无效、接受介入栓塞术,术后予西罗莫司口服.每隔4周常规监测血常规、凝血功能、肝肾功能、血脂及西罗莫司血药浓度等指标.根据血药浓度、血小板变化、凝血功能、瘤体缩小及不良反应情况调整用药计划.术后随访3-13个月,观察疗效和不良反应.结果 12例患儿均成功实施介入栓塞术,治疗后2-15 d,平均（7±5）d患儿血小板均上升至≥100×10^9/L,治疗后7-25 d,平均（15±7）d血小板均稳定在150×10^9/L以上.其中2例治疗前纤维蛋白原正常;10例治疗后3-15 d,平均（9±4）d天纤维蛋白原第1次上升至≥2.0 g/L,治疗后13-34 d,平均（19±7）d纤维蛋白原稳定在2.0 g/L以上.治疗过程中1例出现瘤体溃烂、1例肺部感染,给予对症处理后恢复良好.结论 介入栓塞术联合西罗莫司治疗婴幼儿KMS是一种安全有效的方法,能够在短时间内迅速提升血小板及改善凝血功能.

Objective To investigate the efficacy and safety of transcather arterial embolization （TAE） plus sirolimus for the treatment of refractory Kasabach-Merritt syndrome （KMS） in infants. Methods Clinical data of twelve infants with refractory KMS treated between December 2015 and October 2016 in a single hospital were retrospectively analyzed. TAE were performed in all patients after failed traditional multiple therapies, followed by oral sirolimus administration. The dose of sirolimus was modulated according to the level of sirolimus, the count of platelet, the shrinkage of the lesion and the side effects, which were monitored regularly during the study. Results All 12 patients were treated with TAE plus sirolimus therapies successfully. The platelet count for all patients increased to≥100×10^9/L for the first time at （7±5） days. Stabilization of platelet level was obtained in （15±7） days averagely. Before the treatment, two infants had a normal fibrinogen level and the fibrinogen level in the other 10 infants was found to be increased to≥2.0 g/L at （9 ± 4）days for the first time and was then stabilized at levels〉2.0 g/L at （19 ± 7）days after treatment. One patient showed skin fester （GradeⅡ）, one patient had a fever with acute pulmonary infection （Grade Ⅲ） and both patients improved well after symptomatic treatment. There were no serious complications in the other ten patients. Conclusions TAE plus sirolimus can rapidly improve levels of platelets and fibrinogen, and it is a safe, useful and effective method for treatment of refractory KMS in infants.