Glycodeoxycholic acid as alternative treatment in 3β-hydroxy-Δ5-C27-steroid-oxidoreductase: a case report

Front Pediatr. 2024 Jun 28:12:1418963. doi: 10.3389/fped.2024.1418963. eCollection 2024.

Abstract

Background: 3β-hydroxy-Δ5-C27-steroid-oxidoreductase (3β-HSD) deficiency is a bile acid synthesis disorder that leads to the absence of normal primary bile acids and the accumulation of abnormal bile acids. This results in cholestatic jaundice, fat-soluble vitamin deficiency, acholic or fatty stools and failure to thrive. Bile acid supplementation is used to treat 3β-HSD-deficiency and its symptoms.

Methods: This report details the case of a 28-year-old woman diagnosed with 3β-HSD-deficiency, who was treated with glycine-conjugated deoxycholic acid (gDCA).

Results: gDCA treatment successfully restored normal bile acid levels, improved body weight by reducing fat malabsorption, and was well-tolerated with no observed liver problems or side effects.

Conclusions: As a potent FXR ligand, gDCA might exert its action through FXR activation leading to bile acid synthesis regulation.

Keywords: 3β-HSD; FXR ligand; bile acid defect; glycine-conjugated deoxycholic acid; postprandial.

Publication types

  • Case Reports

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The author(s) declare financial support was received for the research, authorship, and/or publication of this article.