Familial hypomagnesemia with hypercalciuria and nephrocalcinosis.

CLDN16 CLDN19 Claudin FHHNC Hypomagnesemia-hypercalciuria-nephrocalcinosis Macular colobomata Tight junctions

Journal

Pediatric nephrology (Berlin, Germany)
ISSN: 1432-198X
Titre abrégé: Pediatr Nephrol
Pays: Germany
ID NLM: 8708728

Informations de publication

Date de publication:
10 2021
Historique:
received: 22 09 2020
accepted: 25 01 2021
revised: 14 01 2021
pubmed: 18 2 2021
medline: 3 3 2022
entrez: 17 2 2021
Statut: ppublish

Résumé

Familial hypomagnesemia with hypercalciuria and nephrocalcinosis (FHHNC; OMIM 248250) is a rare autosomal recessive kidney disease caused by mutations in the CLDN16 or CLDN19 genes encoding the proteins claudin-16 and claudin-19, respectively. These are involved in paracellular magnesium and calcium transport in the thick ascending limb of Henle's loop and account for most of the magnesium reabsorption in the tubules. FHHNC is characterized by hypomagnesaemia, hypercalciuria, and nephrocalcinosis, and progresses to kidney failure, requiring dialysis and kidney transplantation mainly during the second to third decades of life. Patients carrying CLDN19 mutations frequently exhibit associated congenital ocular defects leading to variable visual impairment. Despite this severe clinical course, phenotype variability even among siblings has been described in this disease, suggesting unidentified epigenetic mechanisms or other genetic or environmental modifiers. Currently, there is no specific therapy for FHHNC. Supportive treatment with high fluid intake and dietary restrictions, as well as magnesium salts, thiazides, and citrate, are commonly used in an attempt to retard the progression of kidney failure. A kidney transplant remains the only curative option for kidney failure in these patients. In this review, we summarize the current knowledge about FHHNC and discuss the remaining open questions about this disorder.

Identifiants

pubmed: 33595712
doi: 10.1007/s00467-021-04968-2
pii: 10.1007/s00467-021-04968-2
doi:

Substances chimiques

Claudins 0
Magnesium I38ZP9992A

Types de publication

Journal Article Research Support, Non-U.S. Gov't Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

3045-3055

Commentaires et corrections

Type : CommentIn

Informations de copyright

© 2021. IPNA.

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Auteurs

Mònica Vall-Palomar (M)

Fisiopatologia Renal, Centro de Investigaciones en Bioquímica y Biología Molecular (CIBBIM), Vall d'Hebron. Institut de Recerca (VHIR), Barcelona, Spain.

Leire Madariaga (L)

Biocruces Bizkaia Health Research Institute, Barakaldo, Bizkaia, Spain.
Pediatric Nephrology Department, Cruces University Hospital, UPV/EHU, Barakaldo, Spain.

Gema Ariceta (G)

Fisiopatologia Renal, Centro de Investigaciones en Bioquímica y Biología Molecular (CIBBIM), Vall d'Hebron. Institut de Recerca (VHIR), Barcelona, Spain. gariceta@vhebron.net.
Pediatric Nephrology Department, Hospital Universitari Vall d'Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain. gariceta@vhebron.net.
Servei Nefrología Pediátrica, Hospital Vall d' Hebron, Passeig de la Vall d'Hebron, 119-129, 08035, Barcelona, Spain. gariceta@vhebron.net.

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