Evidence map›Paper›PMID 39005507›Full record

ArticleFrontiers in pediatrics2024

Glycodeoxycholic acid as alternative treatment in 3β-hydroxy-Δ5-C

S Majait, F M Vaz, E Marleen Kemper, A H Bootsma, A K Groen, M Nieuwdorp, Maarten R Soeters

Abstract readCase Reports
In one paragraph

Article in Frontiers in pediatrics, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors.

S MajaitDepartment of Pharmacy and Clinical Pharmacology, Amsterdam UMC Location University of Amsterdam, Amsterdam, Netherlands.
F M VazDepartment of Clinical Chemistry and Pediatrics, Laboratory Genetic Metabolic Diseases, Emma Children's Hospital, Amsterdam UMC Location University of Amsterdam, Amsterdam, Netherlands.
E Marleen KemperDepartment of Experimental Vascular Medicine, Amsterdam University Medical Center, Amsterdam, Netherlands.
A H BootsmaDepartment of Clinical Chemistry and Pediatrics, Laboratory Genetic Metabolic Diseases, Emma Children's Hospital, Amsterdam UMC Location University of Amsterdam, Amsterdam, Netherlands.
A K GroenDepartment of Experimental Vascular Medicine, Amsterdam University Medical Center, Amsterdam, Netherlands.
M NieuwdorpDepartment of Vascular Medicine, Amsterdam UMC Location University of Amsterdam, Amsterdam, Netherlands.
Maarten R SoetersDepartment of Endocrinology and Metabolism, Amsterdam UMC Location University of Amsterdam, Amsterdam, Netherlands.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

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.

Indexed as

3β-HSDbile acid defectFXR ligandglycine-conjugated deoxycholic acidpostprandial

Identifiers

PMID39005507
PMCPMC11239425

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.