Postauthorization safety study of betaine anhydrous

Mütze U, Gleich F, Garbade SF, et al. · Journal of Inherited Metabolic Disease

The longest-duration human safety dataset betaine has: in 130 people treated for a median of 6.8 years, with 49% of those over 10 years old exceeding the 6 g/day recommended maximum, no new or potential risk was identified, and combined treatment cut mean total homocysteine from 203 to 81 umol/L.

Population

Individuals with cystathionine beta-synthase deficiency, MTHFR deficiency or cobalamin C disease in an international registry, median 6.8 years of betaine treatment

Intervention

Betaine anhydrous (Cystadane), frequently above the recommended 6 g/day maximum

Comparator

None (prospective non-interventional registry)

Limitations

A rare-disease population on combined disease-specific therapy, so betaine's contribution cannot be isolated; non-interventional pharmacovigilance reporting rather than systematic adverse-event ascertainment; run as a partnership with the marketing authorisation holder, which funds the registry and has an employee among the authors.