Tier 4 — mechanistic

Postauthorization safety study of betaine anhydrous

Ulrike Mütze, Florian Gleich, Sven F Garbade, Céline Plisson, Luis Aldámiz-Echevarría, Francisco Arrieta, Diana Ballhausen, Matthias Zielonka, Danijela Petković Ramadža, Matthias R Baumgartner, Aline Cano, María Concepción García Jiménez, Carlo Dionisi-Vici, Pavel Ješina, Henk J Blom, Maria Luz Couce, Silvia Meavilla Olivas, Karine Mention, Fanny Mochel, Andrew A M Morris, Helen Mundy, Isabelle Redonnet-Vernhet, Saikat Santra, Manuel Schiff, Aude Servais, Isidro Vitoria, Martina Huemer, Viktor Kožich, Stefan Kölker
Journal of inherited metabolic disease 2022 45(4):719-733

Bibliography

PubMed
PMID 35358327
DOI
10.1002/jimd.12499
Funding
Funding was not specified in the indexed record or accessible funding metadata.
Competing interests
A competing-interest declaration was not available in the indexed record or accessible disclosure metadata.

Study snapshot

DesignProspective safety registry
ModelHuman participants
Sample130 patients with CBS deficiency, severe MTHFR deficiency or cblC disease
InterventionPrescription betaine anhydrous within disease-specific treatment
DurationMedian treatment exposure 6.8 years
EndpointsAdverse events; Prescribed dose; Homocysteine

What the study showed, in plain terms

Published in 2022, this source examined prescription betaine and inherited metabolic disorders. Design: Prospective safety registry. Sample/model: 130 patients with CBS deficiency, severe MTHFR deficiency or cblC disease. The exposure or intervention was Prescription betaine anhydrous within disease-specific treatment. Follow-up or treatment period: Median treatment exposure 6.8 years.

No new safety signal was identified despite frequent higher prescribed doses; overall homocysteine decreased with combined treatment.

Nonrandomized rare-disease registry with manufacturer participation. Absence of a new signal does not establish unrestricted safety in healthy users.

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Key findings

  • No new safety signal was identified despite frequent higher prescribed doses; overall homocysteine decreased with combined treatment.
  • The recorded outcome domains were Adverse events; Prescribed dose; Homocysteine.
  • The exposure was observed rather than assigned as an isolated supplement treatment.

What this study can and cannot tell us

  • Nonrandomized rare-disease registry with manufacturer participation. Absence of a new signal does not establish unrestricted safety in healthy users.
  • Confounding, dietary patterns, illness, medication use and reverse causation can influence an association. Blood or dietary concentrations do not identify the effect of starting a capsule.
  • Source basis: Indexed abstract and bibliographic data. Missing method details, funding information or participant denominators are not inferred.

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Cystathionine beta synthase deficiency and brain edema associated with methionine excess under betaine supplementation: Four new cases and a review of the evidence
Bernd C Schwahn et al. · JIMD reports · 2020
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Efficacy and pharmacokinetics of betaine in CBS and cblC deficiencies: a cross-over randomized controlled trial
Apolline Imbard et al. · Orphanet journal of rare diseases · 2022

Citation network

Articles citing this research paper

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Editorial review

Reviewed by the Biohack Blueprint research team

Last verified

Dr. Khaled Mahmoud, MBBChCritical Care Medicine, Longevity Science & Editorial Direction Dr. Amr Yasser, PhDCardiometabolic, Musculoskeletal, Gut & Bioavailability Dr. Abdulla Mostafa, PhDCellular Senescence, Neurological, Mitochondrial & Brain-Ageing Biology