PMID- 30107845
OWN - NLM
STAT- MEDLINE
DCOM- 20190318
LR  - 20211204
IS  - 1750-1172 (Electronic)
IS  - 1750-1172 (Linking)
VI  - 13
IP  - 1
DP  - 2018 Aug 14
TI  - The efficacy and adverse events of mTOR inhibitors in lymphangioleiomyomatosis: 
      systematic review and meta-analysis.
PG  - 134
LID - 10.1186/s13023-018-0874-7 [doi]
LID - 134
AB  - BACKGROUND: Lymphangioleiomyomatosis (LAM) is a rare lung disease and the 
      mammalian target of the rapamycin (mTOR) inhibitors has been used as an effective 
      therapy. Here we conducted a systematic review and meta-analysis with the aims to 
      quantify the efficacy and safety of mTOR inhibitors in LAM patients. METHODS: The 
      following databases were searched for clinical trials regarding LAM patients 
      treated with mTOR inhibitors until December 2017: Pubmed, Embase, Cochrane 
      Library and OVID medicine. Random effect models were used for the quantitative 
      analysis. RESULTS: Nine eligible studies were included in our systematic review, 
      7 of which were used for the meta-analysis. In LAM patients, mTOR inhibitors 
      improved forced expiratory volume in 1 s (FEV(1)) and forced vital capacity (FVC) 
      significantly, with the weighted mean difference (WMD) 0.15 L (95%CI: 0.08 to 
      0.22, P < 0.01, I(2) = 0%) and 0.22 L (95%: 0.11 to 0.32, P < 0.01, I(2) = 0%) 
      respectively. There was no significant change in neither the diffusing capacity 
      for carbon monoxide (WMD: 0.51 ml/mm Hg/min, 95%CI: -0.48 to 1.49, P = 0.31, 
      I(2) = 0%) nor 6-min walking distance (WMD: 5.29 m, 95%CI: -18.01 to 28.59, 
      P = 0.66, I(2) = 1%). The weighted partial response rate was 0.68 (95%CI: 0.53 to 
      0.84, P < 0.01, I(2) = 72%) for renal angiomylipoma. The cumulative incidence 
      rates of common safety events were 50, 40, 23, 20 and 19% for oral mucositis, 
      hyperlipidemia, headache, bone marrow suppression, and diarrhea, respectively. 
      And most events were low grade and tolerant. CONCLUSIONS: In LAM patients, there 
      are improvements of FEV(1) and FVC after the application of mTOR inhibitors and 
      over a half achieved the shrinkage of renal angiomyolipoma. TRIAL REGISTRATION: 
      PROSPERO registration number: CRD42018085470. Registered 22 January 2018.
FAU - Gao, Nannan
AU  - Gao N
AD  - Department of Respiratory Medicine, Peking Union Medical College Hospital, Peking 
      Union Medical College, Chinese Academy of Medical Sciences, Beijing, China.
FAU - Zhang, Tengyue
AU  - Zhang T
AD  - Department of Respiratory Medicine, Peking Union Medical College Hospital, Peking 
      Union Medical College, Chinese Academy of Medical Sciences, Beijing, China.
FAU - Ji, Jiadong
AU  - Ji J
AD  - School of Statistics, Shandong University of Finance and Economics, Jinan, China.
FAU - Xu, Kai-Feng
AU  - Xu KF
AD  - Department of Respiratory Medicine, Peking Union Medical College Hospital, Peking 
      Union Medical College, Chinese Academy of Medical Sciences, Beijing, China.
FAU - Tian, Xinlun
AU  - Tian X
AUID- ORCID: 0000-0003-4307-6665
AD  - Department of Respiratory Medicine, Peking Union Medical College Hospital, Peking 
      Union Medical College, Chinese Academy of Medical Sciences, Beijing, China. 
      xinlun_t@sina.com.
LA  - eng
GR  - 2016YFC0901502/State Key Project, Clinical Cohort Study of Rare Diseases. Rare 
      Lung Disease Research Group in China/International
GR  - Z151100003915126/The Capital of the Public Health Program: Study on the 
      Evaluation of Diagnosis and Treatment on Seven Treatable Rare 
      Diseases/International
PT  - Journal Article
PT  - Meta-Analysis
PT  - Research Support, Non-U.S. Gov't
PT  - Systematic Review
DEP - 20180814
PL  - England
TA  - Orphanet J Rare Dis
JT  - Orphanet journal of rare diseases
JID - 101266602
RN  - 0 (Antineoplastic Agents)
RN  - EC 2.7.11.1 (TOR Serine-Threonine Kinases)
SB  - IM
MH  - Animals
MH  - Antineoplastic Agents/*therapeutic use
MH  - Humans
MH  - Lymphangioleiomyomatosis/*drug therapy/*metabolism
MH  - TOR Serine-Threonine Kinases/*antagonists & inhibitors
PMC - PMC6092843
OTO - NOTNLM
OT  - Lymphangioleiomyomatosis
OT  - Meta-analysis
OT  - mTOR inhibitors
COIS- ETHICS APPROVAL AND CONSENT TO PARTICIPATE: Not applicable. CONSENT FOR 
      PUBLICATION: Not applicable. COMPETING INTERESTS: The authors declare that they 
      have no competing interests. PUBLISHER'S NOTE: Springer Nature remains neutral 
      with regard to jurisdictional claims in published maps and institutional 
      affiliations.
EDAT- 2018/08/16 06:00
MHDA- 2019/03/19 06:00
PMCR- 2018/08/14
CRDT- 2018/08/16 06:00
PHST- 2018/05/05 00:00 [received]
PHST- 2018/07/10 00:00 [accepted]
PHST- 2018/08/16 06:00 [entrez]
PHST- 2018/08/16 06:00 [pubmed]
PHST- 2019/03/19 06:00 [medline]
PHST- 2018/08/14 00:00 [pmc-release]
AID - 10.1186/s13023-018-0874-7 [pii]
AID - 874 [pii]
AID - 10.1186/s13023-018-0874-7 [doi]
PST - epublish
SO  - Orphanet J Rare Dis. 2018 Aug 14;13(1):134. doi: 10.1186/s13023-018-0874-7.