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

Abdelmalek MF, Angulo P, Jorgensen RA, Sylvestre PB, Lindor KD · The American Journal of Gastroenterology

The open-label result that launched the betaine-for-liver programme: aminotransferases improved significantly (AST P=0.02, ALT P=0.007) and steatosis, necroinflammatory grade and fibrosis stage all looked better on repeat biopsy — findings the later placebo-controlled trial did not reproduce.

Population

Adults with nonalcoholic steatohepatitis; 7 of 10 completed 12 months

Intervention

Betaine anhydrous oral solution (Cystadane) in two divided daily doses for 12 months

Comparator

None (open-label, baseline-controlled)

Limitations

Ten patients, no control group, no blinding; its histological claims were not confirmed when the same group ran a randomised placebo-controlled trial.