A phase 2 clinical trial of metformin as a treatment for non-diabetic paediatric non-alcoholic steatohepatitis.

摘要: Summary Background : Children with non-alcoholic steatohepatitis are insulin-resistant and metformin has been proposed as a potential therapy. However, paediatric safety efficacy data absent. Aim : To test the hypothesis that therapy will safely improve markers of liver disease in steatohepatitis. Methods : Single-arm open-label pilot study 500 mg twice daily for 24 weeks non-diabetic children biopsy-proven steatohepatitis. Results : Ten obese (mean body mass index 30.4) enrolled completed trial. Mean alanine aminotransferase aspartate (AST) improved significantly (P < 0.01) from baseline (184, 114 U/L) to end treatment (98, 68 U/L). Alanine normalized 40% AST 50% subjects. Children demonstrated significant improvements fat measured by magnetic resonance spectroscopy (30–23%, P < 0.01); insulin sensitivity quantitative check (0.294–0.310, P < 0.05); quality life pediatric inventory 4.0 (69–81, P < 0.01). Conclusion : Open-label was notable improvement chemistry, fat, life. A large randomized-controlled trial is needed definitively determine steatohepatitis.