PMID- 24507028
OWN - NLM
STAT- MEDLINE
DCOM- 20150115
LR  - 20140210
IS  - 1873-2623 (Electronic)
IS  - 0041-1345 (Linking)
VI  - 46
IP  - 1
DP  - 2014 Jan-Feb
TI  - Sirolimus for treatment of autosomal-dominant polycystic kidney disease: a 
      meta-analysis of randomized controlled trials.
PG  - 66-74
LID - S0041-1345(13)01091-9 [pii]
LID - 10.1016/j.transproceed.2013.10.040 [doi]
AB  - BACKGROUND: Autosomal-dominant polycystic kidney disease (ADPKD) is the most 
      common form of cystic kidney disease. The mammalian target of rapamycin (mTOR) 
      pathway is associated with progressive kidney enlargement. The drug sirolimus 
      suppresses mTOR signaling but plays an uncertain role in the treatment of ADPKD. 
      The objective of our study was to conduct a meta-analysis of randomized 
      controlled trials (RCTs) to present an objective appraisal of the efficacy and 
      safety of sirolimus therapy in patients with ADPKD. METHODS: We conducted a 
      meta-analysis of RCTs performed in adults with ADPKD, and compared the effect of 
      sirolimus on total kidney volume (TKV), glomerular filtration rate (GFR), cyst 
      volume, and daily urinary protein excretion. Safety was evaluated based on 
      analysis of blood pressure, lipid profile, complete blood count, infection, and 
      other reported adverse events. RESULTS: Four RCTs were included. The sirolimus 
      therapy group had smaller TKV than the control group. The mean difference (MD) of 
      TKV post-treatment compared with the control group was -234.74 (P = .01). 
      However, GFR did not reach a statistically significant difference between groups. 
      Standard mean difference (SMD) of GFR after therapy was 0.24 (95% confidence 
      interval [CI], 0.05-0.52; P = .11), but sirolimus seemed to increase urine 
      protein excretion (P = .002). There was no statically significant difference in 
      leukocytes, hemoglobin, platelets, and blood pressure between groups. Aphthous 
      stomatits and pharyngitis are reported more commonly in the sirolimus therapy 
      group compared with the control group (P < .000001). CONCLUSIONS: In ADPKD 
      patients, treatment with sirolimus is safe and can effectively slow kidney 
      growth, but it seems not to slow down the decrease of GFR.
CI  - Copyright (c) 2014 Elsevier Inc. All rights reserved.
FAU - Liu, Y-M
AU  - Liu YM
AD  - Department of Nephrology, Zhejiang Provincial People's Hospital, Hangzhou, 
      Zhejiang, China.
FAU - Shao, Y Q
AU  - Shao YQ
AD  - Department of Neurology, Zhejiang Provincial People's Hospital, Hangzhou, 
      Zhejiang, China.
FAU - He, Q
AU  - He Q
AD  - Department of Nephrology, Zhejiang Provincial People's Hospital, Hangzhou, 
      Zhejiang, China. Electronic address: qianghe@zju.edu.cn.
LA  - eng
PT  - Journal Article
PT  - Meta-Analysis
PL  - United States
TA  - Transplant Proc
JT  - Transplantation proceedings
JID - 0243532
RN  - 0 (Immunosuppressive Agents)
RN  - W36ZG6FT64 (Sirolimus)
SB  - IM
MH  - Blood Pressure
MH  - Glomerular Filtration Rate
MH  - Humans
MH  - Immunosuppressive Agents/*therapeutic use
MH  - Kidney/pathology
MH  - Pharyngitis/etiology
MH  - Polycystic Kidney, Autosomal Dominant/*drug therapy
MH  - Proteinuria/etiology
MH  - Randomized Controlled Trials as Topic
MH  - Signal Transduction
MH  - Sirolimus/*therapeutic use
MH  - Stomatitis/etiology
MH  - Treatment Outcome
EDAT- 2014/02/11 06:00
MHDA- 2015/01/16 06:00
CRDT- 2014/02/11 06:00
PHST- 2013/08/21 00:00 [received]
PHST- 2013/10/02 00:00 [accepted]
PHST- 2014/02/11 06:00 [entrez]
PHST- 2014/02/11 06:00 [pubmed]
PHST- 2015/01/16 06:00 [medline]
AID - S0041-1345(13)01091-9 [pii]
AID - 10.1016/j.transproceed.2013.10.040 [doi]
PST - ppublish
SO  - Transplant Proc. 2014 Jan-Feb;46(1):66-74. doi: 
      10.1016/j.transproceed.2013.10.040.