The prevalence of non-alcoholic fatty liver disease and non-alcoholic steatohepatitis (NAFLD/NASH) is increasing. NAFLD/NASH may progress to cirrhosis and hepatocellular carcinoma. However, most patients with NAFLD/NASH will die from a vascular cause. There are no currently approved pharmacological treatments for NASH/NAFLD. A large number of clinical trials have been, or are being, undertaken; however, the challenge is the assessment of the clinical endpoint.
Scope of review
The main objective of this narrative review was to evaluate the efficacy of drugs used in clinical trials for the treatment of NAFLD/NASH that included a liver biopsy as the gold standard. A literature search was carried out using 3 databases (PubMed, Scopus, and Google Scholar) to identify the clinical trials that included liver biopsy assessment before and after treatment. Interventional clinical trials (n=33) involving 18 different agents, alone and in combination, were identified.
Pioglitazone is the only agent that has shown consistent benefit and efficacy in a number of clinical trials. Pentoxifylline, rosiglitazone and ursodeoxycholic acid had both positive and negative results from clinical trials. There is also evidence for vitamin E and metformin. Other drugs including bicyclol, cysteamine bitartrate, L-carnitine, liraglutide, obeticholic acid, oligofructose, selonsertib, silymarin, and statins each had a single clinical study. There is an urgent need for further research in this field.