摘要
目的初步评估临床上应用雷帕霉素治疗激素抵抗型婴幼儿Kasabach-Merritt综合征的临床疗效及安全性。方法2015年2月至2016年4月共收集8例在我科接受治疗的Kasabaeh-Merritt综合征患儿,其中男5例,女3例,病灶位于颌面部3例、颈部1例、四肢2例,胸腹壁2例,8例患儿均表现为激素抵抗,对激素抵抗型Kasabach-Merritt综合征采用mTOR抑制剂雷帕霉素治疗。雷帕霉素服用方法为每次0.8mg/m^2,2次/d,间隔12h,血药浓度维持10-15ng/ml。定期监测血常规、凝血功能、肝肾功能、血脂及雷帕霉素血药浓度等指标。根据血药浓度、血小板变化、凝血功能、瘤体缩小情况及副作用可适当调整用药计划。结果8例患儿经雷帕霉素治疗后血小板均恢复正常,瘤体萎缩,并逐步撤离了激素,有效率达到100%。雷帕霉素平均起效时间(6.8±2.7)d,平均血小板稳定时间(19.1±8.5)d,目前雷帕霉素总的用药时间为4~10个月,平均(6.0±2.2)个月,开始雷帕霉素单药治疗时间为2~8个月,平均(4.5±1.9)个月。8例患儿仍在服药进行中,均无血小板下降和病灶复发。药物使用中不良反应主要为口腔黏膜炎及口腔溃疡(2例,GradeⅡ)、呕吐(1例,GradeⅠ)、腹泻(2例,GradeⅠ/GradeⅡ)、发热(2例,GradeⅡ)、皮疹(1例,GradeⅠ)、疼痛(1例,GradeI)、短暂性转氨酶及血脂异常(1例,GradeⅠ/GradeⅡ),予对症治疗后好转,无严重不良事件,无病例退出。结论mTOR抑制剂雷帕霉素治疗激素抵抗型Kasabaeh-Merritt综合征具有一定的疗效及安全性,值得临床进一步推广应用。
Objective To explore the efficacy and safety of rapamycin in the treatment of steroid-resistant Kasabach-Merritt syndrome in infants. Methods Eight infants with steroid-resistant Kasabach-Merritt syndrome between June 2015 and April 2016 at our hospital were enrolled. There were 5 males and 3 females. The lesions were located in maxillofacial region (n = 3), neck region (n = 1), extremities (n = 2) and trunk (n = 2). Patients with steroid-resistant Kasabach-Merritt received the therapy of rapamycin. Rapamycin was started at a dose of 0. 8 mg/m^2 , administered twice daily at approximately 12-hour intervals for maintaining a level of 10-15 ng/ml. The dose of rapamycin could be modulated according to the level of rapamycin, count of platelet, shrinkage of lesion and side effects. Results All 8 cases were diagnosed as steroid-resistant Kasabach-Merritt syndrome. There were significant improvements in clinical status, includin4g platelet elevation, coagulation function improvement and tumor shrinkage. Steroids were withdrawn quickly. Time to initial response was (6. 8± 2. 7) days, average stabilization time of platelet (19. 1± 8. 5) days, average duration of rapamycin treatment (6. 0 ±2. 2) months and average time for rapamycin alone (4. 5 ± 1.9) months. There was no symptomatic relapse. The side effects were tolerable, including oral mucositis/ulcer (n = 2, grade Ⅱ ), vomiting (n = 1, grade Ⅰ ), diarrhea (n = 2, grade Ⅰ / Ⅱ), fever (n = 2, grade Ⅱ), skin rash (n = 1, grade Ⅰ), pain (n = 1, grade Ⅰ), transient elevation of serum transaminase and cholesterin (n = 1, grade Ⅰ / Ⅱ ). Conclusions Rapamycin therapy for infants with steroid-resistant Kasabach-Merritt syndrome is safe and efficacious so that it is worthy of wider clinical application.
作者
谭小云
张靖
周少毅
申刚
李海波
陈昆山
夏杰军
Tan Xiaoyun Zhang Jing Zhou Shaoyi Shen Gang Li Haibo Chen Kunshan Xia Jiejun(Department of lnterwentional Therapy & Vascular Anomalies, Guangzhou Women & Children's Medical Center, Guangzhou 510120, China)
出处
《中华小儿外科杂志》
CSCD
2017年第1期20-24,共5页
Chinese Journal of Pediatric Surgery
基金
广州市妇女儿童医疗中心儿科研究所内部基金(YIP-2016-019)