Autosomal recessive congenital ichthyosis due to homozygous variants in NIPAL4 with a dramatic response to ustekinumab.


Journal

Pediatric dermatology
ISSN: 1525-1470
Titre abrégé: Pediatr Dermatol
Pays: United States
ID NLM: 8406799

Informations de publication

Date de publication:
Nov 2019
Historique:
pubmed: 19 9 2019
medline: 6 5 2020
entrez: 19 9 2019
Statut: ppublish

Résumé

Autosomal recessive congenital ichthyosis is a genetically and phenotypically heterogenous group of scaling skin disorders. We describe a patient with ARCI caused by homozygous variants in NIPAL4, in whom the dermatologic phenotype and an associated arthropathy, markedly improved with ustekinumab.

Identifiants

pubmed: 31532840
doi: 10.1111/pde.13995
doi:

Substances chimiques

Dermatologic Agents 0
NIPAL4 protein, human 0
Receptors, Cell Surface 0
Ustekinumab FU77B4U5Z0

Types de publication

Case Reports Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

1002-1003

Informations de copyright

© 2019 Wiley Periodicals, Inc.

Références

Fischer J. Autosomal recessive congenital ichthyosis. J Invest Dermatol. 2009;129:1319-1321.
Li H, Vahlquist A, Törmä H. Interactions between FATP4 and ichthyin in epidermal lipid processing may provide clues to the pathogenesis of autosomal recessive congenital ichthyosis. J Dermatol Sci. 2013;69:195-201.
Alavi A, Shahshahani MM, Klotzle B, Fan J-B, Ronaghi M, Elahi E. Manifestation of diffuse yellowish keratoderma on the palms and soles in autosomal recessive congenital ichthyosis patients may be indicative of mutations in NIPAL4. J Dermatol. 2012;39:375-381.
Paller AS, Renert-Yuval Y, Suprun M, et al. An IL-17-dominant immune profile is shared across the major orphan forms of ichthyosis. J Allergy Clin Immunol. 2017;139:152-165.
Paller AS, Czarnowicki T, Renert-Yuval Y, et al. The spectrum of manifestations in desmoplakin gene (DSP) spectrin repeat 6 domain mutations: immunophenotyping and response to ustekinumab. J Am Acad Dermatol. 2018;78(498-505):e2.

Auteurs

Cathryn Poulton (C)

Genetic Services of Western Australia, King Edward Memorial Hospital, Perth, Australia.
Department of Paediatrics and Neonatology, Fiona Stanley Hospital, Perth, Australia.

Dylan Gration (D)

Genetic Services of Western Australia, King Edward Memorial Hospital, Perth, Australia.
The Western Australian Register of Developmental Anomalies, King Edward Memorial Hospital, Perth, Australia.

Kevin Murray (K)

Department of Rheumatology, Perth Children's Hospital, Perth, Australia.

Gareth Baynam (G)

Genetic Services of Western Australia, King Edward Memorial Hospital, Perth, Australia.
The Western Australian Register of Developmental Anomalies, King Edward Memorial Hospital, Perth, Australia.
The Undiagnosed Diseases Program, King Edward Memorial Hospital, Perth, Australia.

Anne Halbert (A)

Department of Dermatology, Perth Children's Hospital, Perth, Australia.

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Classifications MeSH