摘要
目的:探索西罗莫司治疗各类脉管性疾病的有效性和安全性。方法:采用单中心前瞻性设计,纳入各类脉管性疾病患儿,给予0.8 mg/m^(2)西罗莫司单药口服,每12 h 1次,目标血药浓度为5~15μg/L,28 d为1个治疗周期。通过影像学评估疗效,监测血药浓度和用药期间不良反应。结果:27例脉管性疾病患儿中,18例血管瘤和9例脉管畸形。在治疗的前6个周期内,瘤体体积呈持续缩小趋势,后续治疗缩小速率减缓。治疗6个周期后,24例(88.9%)患儿达到客观缓解(ORR),其中13例完全缓解(CR)、11例部分缓解(PR);治疗12个周期后ORR提升至96.3%(26/27),其中13例达到CR、13例达到PR。瘤体平均缩小(63.1±27.0)%,平均起效时间为(4.4±2.8)个治疗周期。15例患儿出现至少1次西罗莫司相关不良事件。更高的西罗莫司血药浓度(≥10μg/L)疗效更好,但不良事件发生率更高。结论:西罗莫司在各类脉管性疾病中有较好的疗效且耐受性良好。推荐治疗时间为6个疗程。西罗莫司血药浓度为10~15μg/L时疗效相对更好。
Objective:To evaluate the efficacy and safety of sirolimus in the treatment of various vascular anomalies in children.Methods:Sirolimus was administered orally on a continuous dosing schedule,from 0.8 mg/m^(2)every 12 hours to a target serum trough concentration of 5-15μg/L.Each treatment course lasted 28 days.Efficacy was assessed via imaging examinations.Serum trough concentrations and adverse events were monitored throughout the treatment period.Results:Twenty-seven patients were evaluated for efficacy and tolerance at the end of the treatment.Among them,18 were diagnosed with hemangiomas and 9 with vascular malformations.During the first 6 treatment cycles,the volume of vascular lesions showed a progressive reduction,but the rate of shrinkage decreased in subsequent cycles.After 6 cycles,the objective response rate(ORR)was 88.9%(24/27),including 13 complete responses(CR)and 11 partial responses(PR).By the 12th cycle,the ORR rose to 96.3%(26/27),with CR in 13 and PR in 13 cases.The average lesion volume reduction was(63.1±27.0)%after 12 courses,and the median time to response was 4.4±2.8 courses.Fifteen patients experienced at least one sirolimus-associated adverse event.The group with higher sirolimus trough concentrations(≥10μg/L)exhibited a greater lesion volume reduction,but also had a higher incidence of adverse events.Conclusion:Sirolimus is an effective and well-tolerated therapeutic option for children with various vascular anomalies.A treatment of six courses is recommended.A sirolimus trough concentration of 10-15μg/L is associated with a stronger efficacy,though it requires careful monitoring for adverse events.
作者
吴莉
陈凛凛
曾颖华
关建维
周铭
林丹娜
余莉华
王曦培
张旺明
杨丽华
WU Li;CHEN Linlin;ZENG Yinghua;GUAN Jianwei;ZHOU Ming;LIN Danna;YU Lihua;WANG Xipei;ZHANG Wangming;YANG Lihua(Department of Pediatric Hematology,Zhujiang Hospital,Southern Medical University,Guangzhou 510220,China;Department of Pediatrics,Jiangmen Maternal and Child Health Hospital,Jiangmen 529000,China;Department of Pediatric Neurosurgery,Zhujiang Hospital,Southern Medical University,Guangzhou 510220,China;Medical Research Center,Guangdong Provincial People's Hospital,Guangdong Academy of Medical Sciences,Guangzhou 510000,China)
出处
《临床皮肤科杂志》
北大核心
2025年第12期720-724,共5页
Journal of Clinical Dermatology
