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Pharmacological Interventions for Nonalcoholic Fatty Liver Disease in Adults and in Children: A Systematic Review

Socha, Piotr*; Horvath, Andrea; Vajro, Pietro; Dziechciarz, Piotr; Dhawan, Anil§; Szajewska, Hania

Journal of Pediatric Gastroenterology and Nutrition: May 2009 - Volume 48 - Issue 5 - p 587–596
doi: 10.1097/MPG.0b013e31818e04d1
Original Articles: Hepatology and Nutrition

Background: Uncertainty exists regarding the treatment of patients with nonalcoholic fatty liver disease (NAFLD) who are unable to lose weight and/or change lifestyle. The present study assesses the effectiveness and safety of pharmacological and dietary supplement interventions for NAFLD.

Methods: MEDLINE, EMBASE, and the Cochrane Library were searched for randomized controlled trials (RCTs) both in adults and in children.

Results: Fifteen (2 pediatric patients and 13 adults) RCTs met the inclusion criteria. A significant effect on normalization of alanine transaminase was found in patients treated with metformin compared with vitamin E, and in those treated with high-dose (3 g) carnitine vs diet. In contrast, there was no difference in patients treated with pioglitazone combined with vitamin E versus vitamin E alone, ursodeoxycholic acid (UDCA) combined with vitamin E or alone versus placebo, or UDCA versus combination of vitamin E and vitamin C, and in patients treated with vitamin E, probucol, N-acetylcysteine, low doses of carnitine, or Yo Jyo Shi Ko compared with placebo. Aspartate aminotransferase normalization was significantly higher in those treated with UDCA combined with vitamin E versus UDCA alone or placebo, and in those treated with metformin. Small number of subjects, high drop-out rates, and numerous interventions in 1 study limit the value of many studies. Only 7 RCTs analyzed biopsy specimens, but most of them have significant methodological limitations. Pioglitazone had reduced liver necrosis and inflammation in 1 large study.

Conclusions: Limited data do not allow one to draw firm conclusions on the efficacy of various treatments for NAFLD.

*Department of Gastroenterology, Hepatology and Immunology, Children's Memorial Health Institute, Poland

2nd Department of Paediatrics, Medical University of Warsaw, Warsaw, Poland

Department of Pediatrics, University of Naples “Federico II,” Naples, Italy

§Paediatric Liver Centre, Institute of Liver Studies, Variety Club Children's Hospital, King's College School of Medicine at King's College Hospital, London, UK

Received 10 April, 2008

Accepted 15 September, 2008

Address correspondence and reprint requests to Dr Piotr Socha, Department of Gastroenterology, Hepatology and Immunology, Children's Memorial Health Institute, Al. Dzieci Polskich 20, 04-730 Warsaw, Poland (e-mail:

The authors report no conflicts of interest.

© 2009 Lippincott Williams & Wilkins, Inc.