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. 2015 Jul 14;2015(7):CD010294. doi: 10.1002/14651858.CD010294.pub2

Melemadathil 2013.

Methods
  • Study design: parallel, open‐label RCT

  • Duration of study: not reported

  • Follow‐up: 1 year

  • ADPKD assessment: magnetic nuclear imaging

Participants
  • Country: India

  • Setting: not reported

  • ADPKD type 1 after genetic typing; aged 18 to 60 years; GFR > 40 mL/min/1.73 m2; proteinuria < 0.5 g/24 h; informed consent

  • Number: treatment group (40); control group (20)

  • Mean age ± SD (years): not reported

  • Sex (M/F): not reported

  • Exclusion criteria: proteinuria > 0.5 g/24 h or abnormal urinalysis; DM; malignancy; psychiatric disorder; hepatitis B, C; HIV; pregnancy and lactation; increased liver enzymes; dyslipidaemia; granulocytopenia or thrombocytopenia; co‐medication with strong inhibitor of CYP3A4; hypersensitivity

Interventions Treatment group
  • Sirolimus: 2 mg/d


Control group
  • Standard treatment


Duration of intervention
  • 6 months extended to 1 year

Outcomes
  • Kidney volume

  • Cyst volume

  • Parenchymal volume

  • Proteinuria and other laboratory data

  • Adverse events

Notes
  • Abstract‐only publication

  • Funding source: not reported

Risk of bias
Bias Authors' judgement Support for judgement
Random sequence generation (selection bias) Unclear risk Randomised 2:1. Sequence generation not defined
Allocation concealment (selection bias) Unclear risk Insufficient information to permit judgement
Blinding of participants and personnel (performance bias) 
 All outcomes High risk Open‐label study
Blinding of outcome assessment (detection bias) 
 All outcomes High risk Open‐label study
Incomplete outcome data (attrition bias) 
 All outcomes High risk 6/40 (15%) patients in the mTOR group dropped or were lost to follow up. Unclear whether the study was analysed on ITT or PP basis
Selective reporting (reporting bias) Unclear risk Insufficient information to permit judgement
Other bias Unclear risk Insufficient information to permit judgement