Original Contribution | Published:

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

American Journal of Gastroenterology volume 96, pages 27112717 (2001) | Download Citation

M.F.A.'s current address: Section of Hepatobiliary Diseases, University of Florida, P.O. Box 100214, Gainesville, FL 32610

Subjects

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 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.

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Author information

Affiliations

  1. Division of Gastroenterology and Hepatology, Mayo Clinic and Foundation, Rochester, Minnesota, USA

    • Manal F Abdelmalek
    • , Paul Angulo
    • , Roberta A Jorgensen
    •  & Keith D Lindor
  2. Division of Surgical Pathology, Mayo Clinic and Foundation, Rochester, Minnesota, USA

    • Pamela B Sylvestre

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Correspondence to Keith D Lindor.

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DOI

https://doi.org/10.1111/j.1572-0241.2001.04129.x