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Table 1 Different preclinical studies evidencing the efficacy of pioglitazone in ADPKD

From: Autosomal dominant polycystic kidney disease and pioglitazone for its therapy: a comprehensive review with an emphasis on the molecular pathogenesis and pharmacological aspects

Primary citation of study Preclinical model ^^Dose of pioglitazone Duration of treatment Observation Mechanism
Yoshihara et al. (2012) PCK rat 10 mg/kg 16 weeks Decreased cell proliferation and reduced PKD progression Gene sets responsible for cell cycle and proliferation were downregulated
Raphael et al. (2009) PC-PKD1-KO mice 30 mg/kg 3 weeks Pioglitazone increased the survival ^Blood pressure reduction due to improved endothelial function and/or reduction of inflammation
Blazer-Yost et al. (2010) PCK rat 4 mg/kg and 20 mg/kg 7 and 14 weeks With 20 mg/kg cystic burden¥ reduced after 7 weeks in male rats and 14 weeks in female rats and 4 mg/kg was effective in both the sexes after 7 weeks A decrease in the synthesis of CFTR ion channel
Muto et al. (2002) PKD1−/− mice embryo 80 mg/kg Embryonic day (7–11) Decreased cardiac defects and renal cystogenesis ^Tyrosine phosphorylation of GAB1 and EGFR
Flaig et al. (2016) WPK−/− rat 2 and 0.2 mg/kg 13 days Inhibition of cyst growth by 0.2 mg/kg ^Decrease in the CFTR expression
Kanhai et al. (2020) PKD mice 30 mg/kg 14 weeks Inhibition of cystic growth, and increase of survival Inhibition of cAMP pathway
  1. ADPKD autosomal dominant polycystic kidney disease, CFTR cystic fibrosis transmembrane conductance receptor, PCK polycystic kidney disease, WPK wistar polycystic kidney, EGFR epidermal growth factor receptor
  2. ^^Doses are in per kilogram of body weight and frequency of dose is per day
  3. ^This is not the exact mechanism, but the suggested mechanism as mentioned in the study
  4. ¥Cystic burden can also be termed as cystic size