title: Efficacy and safety of a mammalian target of rapamycin inhibitor in pediatric patients with tuberous sclerosis complex: A systematic review and meta-analysis.
abstract: Inhibitors of mammalian target of rapamycin (mTOR) are increasingly used as therapy for pediatric patients with tuberous sclerosis complex (TSC). The uncertainty over the efficacy and safety of mTOR inhibitor therapy for the treatment of pediatric patients with TSC emphasizes the necessity for a synthesis of existing evidence. The aim of this study was to assess the efficacy and safety of mTOR inhibitor therapy for the treatment of pediatric patients with TSC. The PubMed, EmBase and Cochrane Library electronic databases were searched, and studies of mTOR inhibitor therapy and non-mTOR inhibitor therapy in pediatric patients with TSC (<18 years old) were selected. Eleven studies met the inclusion criteria. There was evidence of a significantly increased response rate in pediatric patients with TSC treated with mTOR inhibitor therapy compared with those treated with non-mTOR inhibitor therapy (odds ratio, 24.71; 95% confidence interval, 7.46-81.72; P<0.001). The majority of studies reported few adverse events. There was an increased incidence of mouth ulceration, stomatitis, convulsion and pyrexia in pediatric patients with TSC treated with mTOR inhibitor therapy. In conclusion, mTOR inhibitor therapy is an efficacious and safe treatment for pediatric patients with TSC.
authors.affiliation: Department of Pediatrics, Chinese PLA General Hospital, Beijing 100853, P.R. China.
authors.forename: Guang
authors.initials: G
authors.lastname: Yang
authors.affiliation: Special Care Medical Center, Navy General Hospital of PLA, Beijing 100048, P.R. China.
authors.forename: Lu
authors.initials: L
authors.lastname: Yang
authors.affiliation: Department of Pediatrics, Chinese PLA General Hospital, Beijing 100853, P.R. China.
authors.forename: Xiaofan
authors.initials: X
authors.lastname: Yang
authors.affiliation: Department of Pediatrics, Chinese PLA General Hospital, Beijing 100853, P.R. China.
authors.forename: Xiuyu
authors.initials: X
authors.lastname: Shi
authors.affiliation: Department of Pediatrics, Chinese PLA General Hospital, Beijing 100853, P.R. China.
authors.forename: Jing
authors.initials: J
authors.lastname: Wang
authors.affiliation: Department of Pediatrics, Chinese PLA General Hospital, Beijing 100853, P.R. China.
authors.forename: Yujie
authors.initials: Y
authors.lastname: Liu
authors.affiliation: Department of Pediatrics, Chinese PLA General Hospital, Beijing 100853, P.R. China.
authors.forename: Jun
authors.initials: J
authors.lastname: Ju
authors.affiliation: Department of Pediatrics, Chinese PLA General Hospital, Beijing 100853, P.R. China ; Beijing Institute for Brain Disorders, Beijing 100069, P.R. China.
authors.forename: Liping
authors.initials: L
authors.lastname: Zou
keywords: mammalian target of rapamycin inhibitor; meta-analysis; pediatric; tuberous sclerosis complex