Systematic review of phenotypes and genotypes of patients with gastrointestinal defects and immunodeficiency syndrome-1 (GIDID1) (related to TTC7A).

TTC7A enteropathy gastrointestinal defect and immunodeficiency-1 immune deficiency intestinal atresia

Journal

Intractable & rare diseases research
ISSN: 2186-3644
Titre abrégé: Intractable Rare Dis Res
Pays: Japan
ID NLM: 101586847

Informations de publication

Date de publication:
31 May 2024
Historique:
received: 22 11 2023
revised: 02 02 2024
accepted: 31 03 2024
medline: 5 6 2024
pubmed: 5 6 2024
entrez: 5 6 2024
Statut: ppublish

Résumé

The objective was to conduct a comprehensive review of the morbidity and mortality observed in published patients with gastrointestinal defects and immunodeficiency syndrome-1 (GIDID1) related to TTC7A abnormalities. This included phenotypic, genotypic, and therapeutic aspects. Twenty-seven articles were included, which represented a total of 83 patients. Mortality was of 65.8% of the cases with a mean death at 11.8 months. The mortality rate was 197.1 per 1,000 patients-years, which is significantly higher than other enteropathy types caused by defects in epithelial trafficking and polarity (such as

Identifiants

pubmed: 38836179
doi: 10.5582/irdr.2023.01109
pmc: PMC11145403
doi:

Types de publication

Journal Article Review

Langues

eng

Pagination

89-98

Informations de copyright

2024, International Research and Cooperation Association for Bio & Socio - Sciences Advancement.

Déclaration de conflit d'intérêts

None.The authors have no conflicts of interest to disclose.

Auteurs

Amelie Busolin (A)

APHM, Multidisciplinary Pediatrics Departement, La Timone Children's Hospital, Marseille, France.

Frederic Vely (F)

Aix Marseille University, CNRS, INSERM, CIML, Marseille, France.
APHM, Hôpital de la Timone, Immunology Department, Marseille Immunopôle, Marseille, France.

Gilles Eymard-Duvernay (G)

Transmissions Department, Airbus, Vitrolles, France.

Vincent Barlogis (V)

Pediatric Haematology Department, Timone Enfant, APHM, Aix-Marseille University, Marseille, France.

Alexandre Fabre (A)

APHM, Multidisciplinary Pediatrics Departement, La Timone Children's Hospital, Marseille, France.

Classifications MeSH