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

Manal F. Abdelmalek, Paul Angulo, Roberta A. Jorgensen, Pamela B. Sylvestre, Keith D. Lindor

Research output: Contribution to journalArticle

381 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)2711-2717
Number of pages7
JournalAmerican Journal of Gastroenterology
Volume96
Issue number9 SUPPL.
DOIs
StatePublished - Oct 3 2001
Externally publishedYes

ASJC Scopus subject areas

  • Hepatology
  • Gastroenterology

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