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


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 Gl 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. © 2001 by Am. Coll. of Gastroenterology.

Full Text

Duke Authors

Cited Authors

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

Published Date

  • October 3, 2001

Published In

Volume / Issue

  • 96 / 9 SUPPL.

Start / End Page

  • 2711 - 2717

International Standard Serial Number (ISSN)

  • 0002-9270

Digital Object Identifier (DOI)

  • 10.1016/S0002-9270(01)02691-0

Citation Source

  • Scopus