口服西罗莫司治疗小儿脉管异常类疾病的效果观察

Study of oral sirolimus in the treatment of vascular anomaliesin infants and children

目的初步探讨口服西罗莫司治疗小儿脉管异常类疾病的临床疗效。方法收集2019年12月至2020年12月在湖南省儿童医院皮肤科住院治疗的14例脉管异常类疾病患者作为研究对象,其中男性11例,女性3例,年龄6个月至10岁。病种:卡波西血管内皮瘤4例,Sturge-Weber综合征2例,静脉畸形3例,混合型脉管畸形3例,CLOVES综合征1例,结节性硬化症1例。住院期间给予西罗莫司每日1~1.5 mg/m^(2),分早晚两次口服,间隔12 h。住院期间监控不良反应,出院后继续口服药物,定期门诊复查,检测患者血常规、尿常规、肝肾功能、血脂全套、凝血功能、血药浓度等。结果所有患者口服西罗莫司治疗3~7个月,疗程结束后随访1~3个月。治疗效果:4例卡波西血管内皮瘤患者有效;2例Sturge-Weber综合征患者1例部分有效,1例无效;3例静脉畸形患者1例部分有效,2例无效;3例混合型脉管畸形1例部分有效,2例无效;1例CLOVES综合征患者部分有效;1例结节性硬化症患者无效。14例均未出现明显不良反应。结论西罗莫司治疗卡波西血管内皮瘤可获得较理想的疗效,无明显不良反应;西罗莫司治疗脉管畸形疗效不确定,需要进一步的研究。

Objective To investigate the efficacy of oral sirolimus in the treatment of vascular anomalies in infants and children.Methods From December 2019 to December 2020,14 infant and children with vascular anomalies were hospitalized in the dermatology department of Hunan Children's Hospital.Among them,there were 11 males and 3 females,aged from 6 months to 10 years.There were 4 cases of Kaposi hemangioendothelioma,2 cases of Sturge Weber syndrome,3 cases of venous malformation,3 cases of mixed vascular malformation,1 case of CLOVES syndrome and 1 case of tuberous sclerosis.Sirolimus 1-1.5 mg/m^(2) was given twice a day at an interval of 12 hours.The adverse reactions were monitored during hospitalization,and the drugs were taken orally after discharge.Regular outpatient examinations were conducted to detect the children's blood and urine routine,liver and kidney function,blood lipids,coagulation function and blood drug concentration.Results All children were treated with sirolimus for 3-7 months.After the whole course of treatment,they were followed up for 1-3 months.Four patients with Kaposi hemangioendothelioma were all effective,two patients with Sturge Weber syndrome,one partially effective and one ineffective.Of the 3 patients with venous malformation,one was partially effective and two were ineffective.In 3 cases of mixed vascular malformation,one case was partially effective and two cases were ineffective.Patients with CLOVES syndrome were partially effective,but patients with tuberous sclerosis were ineffective.No obvious adverse reactions occurred in all children.Conclusion Oral sirolimus has good efficacy in the treatment of Kaposi hemangioendothelioma in infants and children.There was no obvious adverse reaction.The efficacy of sirolimus in the treatment of vascular malformations is uncertain and needs further study.