Betaine, a promising new agent for patients with nonalcoholic steatohepatitis: results of a pilot study.

Published

Journal Article

OBJECTIVES: No effective therapy currently exists for patients with nonalcoholic steatohepatitis (NASH). Betaine, a naturally occurring metabolite of choline, has been shown to raise S-adenosylmethionine (SAM) levels that may in turn play a role in decreasing hepatic steatosis. Our aim was to determine the safety and effects of betaine on liver biochemistries and histological markers of disease activity in patients with NASH. METHODS: Ten adult patients with NASH were enrolled. Patients received betaine anhydrous for oral solution (Cystadane) in two divided doses daily for 12 months. Seven out of 10 patients completed 1 yr of treatment with betaine. RESULTS: A significant improvement in serum levels of aspartate aminotransferase (p = 0.02) and ALAT (p = 0.007) occurred during treatment. Aminotransferases normalized in three of seven patients, decreased by >50% in three of seven patients, and remained unchanged in one patient when compared to baseline values. A marked improvement in serum levels of aminotransferases (ALT -39%; AST -38%) also occurred during treatment in those patients who did not complete 1 yr of treatment. Similarly, a marked improvement in the degree of steatosis, necroinflammatory grade, and stage of fibrosis was noted at 1 yr of treatment with betaine. Transitory GI adverse events that did not require any dose reduction or discontinuation of betaine occurred in four patients. CONCLUSIONS: Betaine is a safe and well tolerated drug that leads to a significant biochemical and histological improvement in patients with NASH. This novel agent deserves further evaluation in a randomized, placebo-controlled trial.

Full Text

Duke Authors

Cited Authors

  • Abdelmalek, MF; Angulo, P; Jorgensen, RA; Sylvestre, PB; Lindor, KD

Published Date

  • September 2001

Published In

Volume / Issue

  • 96 / 9

Start / End Page

  • 2711 - 2717

PubMed ID

  • 11569700

Pubmed Central ID

  • 11569700

International Standard Serial Number (ISSN)

  • 0002-9270

Digital Object Identifier (DOI)

  • 10.1111/j.1572-0241.2001.04129.x

Language

  • eng

Conference Location

  • United States