Evidence map›Paper›PMID 39650085›Full record

ArticleMolecular genetics and metabolism reports2024

Efficacy and safety of switching therapy from chenodeoxycholic acid to cholic acid in Japanese patients with bile acid synthesis disorders.

Mitsuyoshi Suzuki, Hajime Takei, Hiromi Suzuki, Jun Mori, Satoru Sugimoto, Tatsuki Mizuochi, Akira Ohtake, Hisamitsu Hayashi, Akihiko Kimura, Hiroshi Nittono

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular genetics and metabolism reports, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. 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

10 authors.

Mitsuyoshi SuzukiDepartment of Pediatrics, Juntendo University Faculty of Medicine, 2-1-1 Hongo, Bunkyo-ku, Tokyo 113-8421, Japan.
Hajime TakeiDivision of Analysis Technology, Junshin Clinic, Bile Acid Institute, 2-1-24 Haramachi, Meguro-ku, Tokyo 152-0011, Japan.
Hiromi SuzukiDivision of Analysis Technology, Junshin Clinic, Bile Acid Institute, 2-1-24 Haramachi, Meguro-ku, Tokyo 152-0011, Japan.
Jun MoriDivision of Pediatric Endocrinology and Metabolism, Children's Medical Center, Osaka City General Hospital, 2-13-22 Miyakojima-hondori, Miyakojima-ku, Osaka 534-0021, Japan.
Satoru SugimotoDepartment of Pediatrics, Graduate School of Medical Science, Kyoto Prefectural University of Medicine, 465 Kajii-cho, Kawaramachi-Hirokoji, Kamigyo-ku, Kyoto 602-8566, Japan.
Tatsuki MizuochiDepartment of Pediatrics and Child Health, Kurume University School of Medicine, 67 Asahi-machi, Kurume, Fukuoka, Japan.
Akira OhtakeDepartment of Pediatrics and Clinical Genomics, Saitama Medical University, 38 Morohongo, Moroyama-machi, Saitama 350-0495, Japan.
Hisamitsu HayashiLaboratory of Molecular Pharmacokinetics, Graduate School of Pharmaceutical Science, The University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-0033, Japan.
Akihiko KimuraDepartment of Pediatrics, Kumamoto-Ashikita Medical Center for the Severity Disabled, 2813 Ashikita, Ashikita-cho, Ohaza, Kumamoto 869-5461, Japan.
Hiroshi NittonoDivision of Analysis Technology, Junshin Clinic, Bile Acid Institute, 2-1-24 Haramachi, Meguro-ku, Tokyo 152-0011, Japan.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Objectives: This study aimed to assess the safety and efficacy of cholic acid (CA) treatment over 74 weeks in Japanese patients with inherited enzymatic bile acid synthesis disorders (BASD). Methods: This phase 3, open-label, single-arm study enrolled four Japanese patients diagnosed with BASD, including two with 3β-hydroxy- Results: The daily CA doses ranged from 3.8 to 13.7 mg/kg/day. Laboratory values of liver-related biomarkers were maintained within normal ranges or improved. Bile acid analysis revealed CDCA replacement with CA in serum within the initial few weeks of CA treatment. Urinary concentrations of toxic bile acid metabolites associated with liver damage were higher than serum. Adverse effects from CA treatment were mild to moderate, and no treatment discontinuations were due to adverse events. Conclusions: CA treatment over 74 weeks resulted in favorable efficacy and safety outcomes in Japanese patients with BASD, consistent with previous studies. These results support the utility of CA as a therapeutic option for Japanese patients with BASD.

Indexed as

3β-hydroxy-Δ5-C27-steroid dehydrogenase/isomerase deficiencyChenodeoxycholic acidCholic acidΔ4–3-oxosteroid 5β-reductase deficiency

Identifiers

PMID39650085
PMCPMC11625240

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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.