Whole-exome sequencing (WES) increases the diagnostic rate of genetic kidney disorders, but accessibility, interpretation of results, and costs limit use in daily practice.
Univariable analysis of a historical cohort of 392 patients who underwent WES for kidney diseases showed that resistance to treatments, familial history of kidney disease, extrarenal involvement, congenital abnormalities of the kidney and urinary tract and CKD stage ≥G2, two or more cysts per kidney on ultrasound, persistent hyperechoic kidneys or nephrocalcinosis on ultrasound, and persistent metabolic abnormalities were most predictive for genetic diagnosis. We prospectively applied these criteria to select patients in a network of nephrology centers, followed by centralized genetic diagnosis by WES, reverse phenotyping, and multidisciplinary board discussion.
We applied this multistep workflow to 476 patients with eight clinical categories (podocytopathies, collagenopathies, CKD of unknown origin, tubulopathies, ciliopathies, congenital anomalies of the kidney and urinary tract, syndromic CKD, metabolic kidney disorders), obtaining genetic diagnosis for 319 of 476 patients (67.0%) (95% in 21 patients with disease onset during the fetal period or at birth, 64% in 298 pediatric patients, and 70% in 156 adult patients). The suspected clinical diagnosis was confirmed in 48% of the 476 patients and modified in 19%. A modeled cost analysis showed that application of this workflow saved 20% of costs per patient when performed at the beginning of the diagnostic process. Real cost analysis of 66 patients randomly selected from all categories showed actual cost reduction of 41%.
A diagnostic workflow for genetic kidney diseases that includes WES is cost-saving, especially if implemented early, and is feasible in a real-world setting.
Copyright © 2023 by the American Society of Nephrology.
About The Expert
Francesca Becherucci
Samuela Landini
Viviana Palazzo
Luigi Cirillo
Valentina Raglianti
Gianmarco Lugli
Lucia Tiberi
Elia Dirupo
Stefania Bellelli
Tommaso Mazzierli
Jacopo Lomi
Fiammetta Ravaglia
Giulia Sansavini
Marco Allinovi
Domenico Giannese
Chiara Somma
Giuseppe Spatoliatore
Debora Vergani
Rosangela Artuso
Alberto Rosati
Calogero Cirami
Pietro Claudio Dattolo
Gesualdo Campolo
Letizia De Chiara
Laura Papi
Augusto Vaglio
Elena Lazzeri
Hans-Joachim Anders
Benedetta Mazzinghi
Paola Romagnani
References
PubMed
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