Tier 4 — mechanistic

Cerebral edema associated with betaine treatment in classical homocystinuria

A M Devlin, L Hajipour, A Gholkar, H Fernandes, V Ramesh, A A M Morris
The Journal of pediatrics 2004 144(4):545-548

Bibliography

PubMed
PMID 15069409
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

DesignCase report
ModelHuman participants
SampleOne child with classical homocystinuria
InterventionBetaine treatment
DurationEdema 4-6 weeks after treatment began
EndpointsCerebral edema; Adverse-event management

What the study showed, in plain terms

Published in 2004, this source examined prescription betaine and inherited metabolic disorders. Design: Case report. Sample/model: One child with classical homocystinuria. The exposure or intervention was Betaine treatment. Follow-up or treatment period: Edema 4-6 weeks after treatment began.

The child developed cerebral edema and recovered after emergency treatment and withdrawal of betaine.

Single rare-disease case; causal mechanism and incidence cannot be established from this report alone.

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Key findings

  • The child developed cerebral edema and recovered after emergency treatment and withdrawal of betaine.
  • The recorded outcome domains were Cerebral edema; Adverse-event management.

What this study can and cannot tell us

  • Single rare-disease case; causal mechanism and incidence cannot be established from this report alone.
  • A case report or series cannot estimate how often an event occurs or establish causality without alternative explanations and a suitable comparator.
  • Source basis: Indexed abstract and bibliographic data. Missing method details, funding information or participant denominators are not inferred.

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