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Implications of early diagnosis of autosomal dominant polycystic kidney disease: a post hoc analysis of the TEMPO 3:4 trial

It is unknown whether early diagnosis of autosomal dominant polycystic kidney disease (ADPKD) can enable earlier management and improve outcomes. We conducted a post hoc analysis of data from the TEMPO 3:4 trial. Subjects were stratified by ADPKD diagnosis at age ≤18 (childhood diagnosis [CD]) or>...

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Main Authors: Janssens, Peter (Author) , Jouret, François (Author) , Bammens, Bert (Author) , Liebau, Max C. (Author) , Schaefer, Franz (Author) , Dandurand, Ann (Author) , Perrone, Ronald D. (Author) , Müller, Roman-Ulrich (Author) , Pao, Christina S. (Author) , Mekahli, Djalila (Author)
Format: Article (Journal)
Language:English
Published: 09 March 2020
In: Scientific reports
Year: 2020, Volume: 10
ISSN:2045-2322
DOI:10.1038/s41598-020-61303-9
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1038/s41598-020-61303-9
Verlag, lizenzpflichtig, Volltext: https://www.nature.com/articles/s41598-020-61303-9
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Author Notes:Peter Janssens, François Jouret, Bert Bammens, Max C. Liebau, Franz Schaefer, Ann Dandurand, Ronald D. Perrone, Roman-Ulrich Müller, Christina S. Pao & Djalila Mekahli
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Summary:It is unknown whether early diagnosis of autosomal dominant polycystic kidney disease (ADPKD) can enable earlier management and improve outcomes. We conducted a post hoc analysis of data from the TEMPO 3:4 trial. Subjects were stratified by ADPKD diagnosis at age ≤18 (childhood diagnosis [CD]) or>18 (adulthood diagnosis [AD]). Groups were compared for baseline characteristics and total kidney volume (TKV) growth and estimated glomerular filtration rate (eGFR) decline over 3 years. 294 CD and 1148 AD subjects were analyzed. At inclusion, CD subjects were younger (mean age 34.2 versus 39.8 years; p < 0.0001) and had better eGFR (mean ± SD 87.4 ± 23.9 versus 80.1 ± 20.7 mL/min/1.73 m2; p < 0.0001), while CD had more severe Mayo risk classification (p < 0.0001) and more PKD1 mutations (p = 0.003). No statistical differences were found in TKV or eGFR change. At study end, placebo-treated CD subjects had better eGFR than projected by a prediction equation (mean difference ±SD for observed versus predicted eGFR: 2.18 ± 10.7 mL/min/1.73 m2; p = 0.0475). However, these results are not confirmed when excluding stage 1 CKD. Whether CD subjects, despite their risk profile, have a slower disease course than predicted remains inconclusive. Future studies are needed to confirm that early diagnosis and management can alter the disease course of ADPKD.
Item Description:Gesehen am 29.10.2020
Physical Description:Online Resource
ISSN:2045-2322
DOI:10.1038/s41598-020-61303-9

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