title: Efficacy and safety of sirolimus for renal angiomyolipoma in patients with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis: a systematic review.
abstract: OBJECTIVE
  We evaluate the efficacy and safety of sirolimus in the treatment of renal angiomyolipoma in patients with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis.
  
  
  METHODS
  A systematic search of MEDLINE®, Embase®, ACP (American College of Physicians) Journal Club, Cochrane CENTRAL (Central Register of Controlled Trials) and Cochrane Database of Systematic Reviews was performed. A secondary hand search was performed in relevant journals, references and the grey literature. The screening, quality assessment and data extraction of the retrieved articles were independently performed by 2 reviewers in duplicate. Studies that reported an angiomyolipoma response or adverse events after the treatment of sirolimus were included in the analysis.
  
  
  RESULTS
  Four prospective nonrandomized studies involving 94 patients were included in the study. The overall response rate of angiomyolipoma was 46.8% (44 of 94) in the first year. In the second year the angiomyolipoma response rate for those patients still being treated with sirolimus was 43.5% (20 of 46) and the response rate of the patients whose sirolimus treatment was discontinued was 5% (2 of 40). The most common sirolimus related adverse reactions were stomatitis, respiratory infection, skin lesions and hyperlipidemia, while serious adverse reactions were rarely observed.
  
  
  CONCLUSIONS
  This study shows that renal angiomyolipoma shrank during sirolimus therapy but tended to regrow after the therapy was stopped. In general, sirolimus is an effective and safe therapy for renal angiomyolipoma in patients with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis.
authors.affiliation: Department of Urology, West China Hospital, Sichuan University, Chengdu, Sichuan, China.
authors.forename: Zhu-feng
authors.initials: ZF
authors.lastname: Peng
authors.affiliation: Department of Urology, West China Hospital, Sichuan University, Chengdu, Sichuan, China.
authors.forename: Lu
authors.initials: L
authors.lastname: Yang
authors.affiliation: Department of Neurology, West China Hospital, Sichuan University, Chengdu, Sichuan, China.
authors.forename: Ting-ting
authors.initials: TT
authors.lastname: Wang
authors.affiliation: Department of Urology, West China Hospital, Sichuan University, Chengdu, Sichuan, China.
authors.forename: Ping
authors.initials: P
authors.lastname: Han
authors.affiliation: Department of Urology, West China Hospital, Sichuan University, Chengdu, Sichuan, China.
authors.forename: Zhen-hua
authors.initials: ZH
authors.lastname: Liu
authors.affiliation: Department of Urology, West China Hospital, Sichuan University, Chengdu, Sichuan, China. Electronic address: weiqiang163163@163.com.
authors.forename: Qiang
authors.initials: Q
authors.lastname: Wei
mesh_terms: D018207:Angiomyolipoma / Q000150:complications / Q000188:drug therapy*; D000903:Antibiotics, Antineoplastic / Q000627:therapeutic use; D006801:Humans; D007680:Kidney Neoplasms / Q000150:complications / Q000188:drug therapy*; D018192:Lymphangioleiomyomatosis / Q000150:complications / Q000188:drug therapy*; D020123:Sirolimus / Q000627:therapeutic use*; D016896:Treatment Outcome; D014402:Tuberous Sclerosis / Q000150:complications*
chemical_list: D000903:Antibiotics, Antineoplastic; D020123:Sirolimus
keywords: angiomyolipoma; drug-related side effects and adverse reactions; sirolimus; therapeutics