Sirolimus can promote the disappearance of renal angiomyolipoma associated with tuberous sclerosis complex:a prospective cohort study  

作　　者：Shuo Dun Yang-Yang Wang Lin Wan Qiu-Hong Wang Qian Lu Xiao-Yan Yang Qi Zhang Hui-Min Chen Lu-Peng Qiu Li-Ping Zou 

机构地区：[1]Medical School of Chinese PLA,Beijing 100853,China [2]Department of Pediatrics,The First Medical Center,Chinese PLA General Hospital,Beijing 100853,China [3]Center of Epilepsy,Beijing Institute for Brain Disorders,Beijing 100069,China

出　　处：《World Journal of Pediatrics》2024年第6期602-610,共9页世界儿科杂志（英文版）

基　　金：funded by the Capital's Funds for Health Improvement and Research(No.2022-1-5081);National Key Research and Development Program of China(No.2016YFC1000707).

摘　　要：Background Renal angiomyolipoma(RAML)is the most common kidney lesion in patients with tuberous sclerosis complex(TSC),affecting about 80%of patients.It is a benign tumor that grows over time,usually bilaterally,and can easily lead to kidney complications such as acute hemorrhage.Herein,we investigated the efficacy and safety of sirolimus in children with TSC-associated RAML and explored the factors affecting tumor disappearance under sirolimus treatment through subgroup analysis.Methods A prospective cohort study was conducted.Sirolimus was initiated at 1 mg/(m^(2)×day),and dose adjustments were made by a 2-week titration period to attain a trough blood concentration of 5-10 ng/mL.The disappearance of RAML in chil-dren after sirolimus treatment was observed,and Cox regression was used to screen the factors affecting tumor disappearance.Results One hundred and twenty-six patients who met the criteria were analyzed.After 3 months,6 months,12 months,and 24 months of follow-up,tumors disappeared in 18(14.3%),30(23.8%),39(31.0%),and 42(33.3%)children,respec-tively.Tumors disappeared in 50(39.7%)children by the last visit of each individual,and 30(60%)of them occurred within 6 months.The multivariate Cox regression analysis showed that patients with a smaller maximum tumor diameter at baseline had a higher tumor disappearance rate.Thirty-six(29%)patients had stomatitis during the entire treatment period,and no serious adversereactionswereobserved.Conclusions Sirolimus could promote the disappearance of TSC-related RAML.The disappearance rate was correlated with the maximum diameter at baseline,and the smaller the tumor was,the higher the disappearance rate.It is well tolerated in the treatment of RAML associated with TSC.

关 键 词：ANGIOMYOLIPOMA mTOR inhibitor SIROLIMUS Tuberous sclerosis 

分 类 号：R737.11[医药卫生—肿瘤]