Urine concentrating defect as presenting sign of progressive renal failure in Bardet-Biedl syndrome patients.

GFR ciliopathy genetics kidney disease urine osmolality

Journal

Clinical kidney journal
ISSN: 2048-8505
Titre abrégé: Clin Kidney J
Pays: England
ID NLM: 101579321

Informations de publication

Date de publication:
Jun 2021
Historique:
received: 02 03 2020
accepted: 06 07 2020
entrez: 4 6 2021
pubmed: 5 6 2021
medline: 5 6 2021
Statut: epublish

Résumé

Urine concentrating defect is a common dysfunction in ciliopathies, even though its underlying mechanism and its prognostic meaning are largely unknown. This study assesses renal function in a cohort of 54 Bardet-Biedl syndrome (BBS) individuals and analyses whether renal hyposthenuria is the result of specific tubule dysfunction and predicts renal disease progression. The estimated glomerular filtration rate (eGFR), urine albumin:creatinine ratio (ACR) and maximum urine osmolality (max-Uosm) were measured in all patients. Genetic analysis was conducted in 43 patients. Annual eGFR decline (ΔeGFR) was measured in patients with a median follow-up period of 6.5 years. Urine aquaporin-2 (uAQP2) excretion was measured and the furosemide test was performed in patients and controls. At baseline, 33 (61.1%), 12 (22.2%) and 9 (16.7%) patients showed an eGFR >90, 60-90 and <60 mL/min/1.73 m Hyposthenuria is a warning sign predicting poor renal outcome in BBS. The pathophysiology of this defect is most likely beyond defective tubular function.

Sections du résumé

BACKGROUND BACKGROUND
Urine concentrating defect is a common dysfunction in ciliopathies, even though its underlying mechanism and its prognostic meaning are largely unknown. This study assesses renal function in a cohort of 54 Bardet-Biedl syndrome (BBS) individuals and analyses whether renal hyposthenuria is the result of specific tubule dysfunction and predicts renal disease progression.
METHODS METHODS
The estimated glomerular filtration rate (eGFR), urine albumin:creatinine ratio (ACR) and maximum urine osmolality (max-Uosm) were measured in all patients. Genetic analysis was conducted in 43 patients. Annual eGFR decline (ΔeGFR) was measured in patients with a median follow-up period of 6.5 years. Urine aquaporin-2 (uAQP2) excretion was measured and the furosemide test was performed in patients and controls.
RESULTS RESULTS
At baseline, 33 (61.1%), 12 (22.2%) and 9 (16.7%) patients showed an eGFR >90, 60-90 and <60 mL/min/1.73 m
CONCLUSIONS CONCLUSIONS
Hyposthenuria is a warning sign predicting poor renal outcome in BBS. The pathophysiology of this defect is most likely beyond defective tubular function.

Identifiants

pubmed: 34084454
doi: 10.1093/ckj/sfaa182
pii: sfaa182
pmc: PMC8162863
doi:

Types de publication

Journal Article

Langues

eng

Pagination

1545-1551

Informations de copyright

© The Author(s) 2020. Published by Oxford University Press on behalf of ERA-EDTA.

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Auteurs

Miriam Zacchia (M)

Department of Translational Medical Sciences, University of Campania "Luigi Vanvitelli", Naples, Italy.

Francesca Del Vecchio Blanco (FDV)

Department of Precision Medicine, University of Campania "Luigi Vanvitelli", Naples, Italy.

Annalaura Torella (A)

Department of Precision Medicine, University of Campania "Luigi Vanvitelli", Naples, Italy.

Raffaele Raucci (R)

Department of Translational Medical Sciences, University of Campania "Luigi Vanvitelli", Naples, Italy.

Giancarlo Blasio (G)

Department of Precision Medicine, University of Campania "Luigi Vanvitelli", Naples, Italy.

Maria Elena Onore (ME)

Department of Precision Medicine, University of Campania "Luigi Vanvitelli", Naples, Italy.

Emanuela Marchese (E)

Department of Mental and Physical Health and Preventive Medicine, University of Campania "Luigi Vanvitelli", Naples, Italy.
CEINGE, Advanced Biotechnologies, Naples, Italy.

Francesco Trepiccione (F)

Department of Translational Medical Sciences, University of Campania "Luigi Vanvitelli", Naples, Italy.
Biogem Scarl, Ariano Irpino, Italy.

Caterina Vitagliano (C)

Department of Translational Medical Sciences, University of Campania "Luigi Vanvitelli", Naples, Italy.

Valentina Di Iorio (VD)

Multidisciplinary Department of Medical, Eye Clinic, Surgical and Dental Sciences, University of Campania "Luigi Vanvitelli", Naples, Italy.

Perna Alessandra (P)

Department of Translational Medical Sciences, University of Campania "Luigi Vanvitelli", Naples, Italy.

Francesca Simonelli (F)

Multidisciplinary Department of Medical, Eye Clinic, Surgical and Dental Sciences, University of Campania "Luigi Vanvitelli", Naples, Italy.

Vincenzo Nigro (V)

Department of Precision Medicine, University of Campania "Luigi Vanvitelli", Naples, Italy.
Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.

Giovambattista Capasso (G)

Department of Translational Medical Sciences, University of Campania "Luigi Vanvitelli", Naples, Italy.
Biogem Scarl, Ariano Irpino, Italy.

Davide Viggiano (D)

Department of Translational Medical Sciences, University of Campania "Luigi Vanvitelli", Naples, Italy.
Department of Medicine and Health Sciences, University of Molise, Campobasso, Italy.

Classifications MeSH