Abstract

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.

Details

Title
Implications of early diagnosis of autosomal dominant polycystic kidney disease: A post hoc analysis of the TEMPO 3:4 trial
Author
Janssens, Peter 1 ; Jouret François 2 ; Bammens Bert 3 ; Liebau, Max C 4   VIAFID ORCID Logo  ; Schaefer, Franz 5 ; Dandurand, Ann 6 ; Perrone, Ronald D 7 ; Roman-Ulrich, Müller 8   VIAFID ORCID Logo  ; Pao, Christina S 6 ; Djalila, Mekahli 9 

 University Hospitals Leuven, PKD Research Group, Laboratory of Pediatrics, Leuven, Belgium (GRID:grid.410569.f) (ISNI:0000 0004 0626 3338); University Hospitals Brussels, Department of Nephrology, Brussels, Belgium (GRID:grid.410569.f) (ISNI:0000 0004 0626 3338) 
 University of Liège Hospital (ULiège CHU), Division of Nephrology, Liège, Belgium (GRID:grid.4861.b) (ISNI:0000 0001 0805 7253); Cardiovascular Sciences, Groupe Interdisciplinaire de Génoprotéomique Appliquée (GIGA), ULiège, Belgium (GRID:grid.4861.b) 
 Department of Microbiology & Immunology, KU Leuven, Belgium (GRID:grid.4861.b); University Hospitals Leuven, Department of Nephrology, Dialysis and Renal Transplantation, Leuven, Belgium (GRID:grid.410569.f) (ISNI:0000 0004 0626 3338) 
 University of Cologne, Faculty of Medicine and University Hospital of Cologne, Department of Pediatrics and Center for Molecular Medicine, Cologne, Germany (GRID:grid.6190.e) (ISNI:0000 0000 8580 3777); Faculty of Medicine and University Hospital of Cologne, Department II of Internal Medicine and Center for Molecular Medicine Cologne, University of Cologne, Cologne, Germany (GRID:grid.411097.a) (ISNI:0000 0000 8852 305X); University of Cologne, Faculty of Medicine and University Hospital of Cologne, Cologne Excellence Cluster on Cellular Stress Responses in Ageing-Associated Diseases (CECAD) and Systems Biology of Ageing Cologne (Sybacol), Cologne, Germany (GRID:grid.6190.e) (ISNI:0000 0000 8580 3777) 
 University Children’s Hospital Heidelberg, Division of Pediatric Nephrology, Heidelberg, Germany (GRID:grid.5253.1) (ISNI:0000 0001 0328 4908) 
 Otsuka Pharmaceutical Development & Commercialization Inc., Princeton, USA (GRID:grid.419943.2) (ISNI:0000 0004 0459 5953) 
 Tufts Medical Center and Tufts University School of Medicine, Division of Nephrology, Boston, USA (GRID:grid.429997.8) (ISNI:0000 0004 1936 7531) 
 Faculty of Medicine and University Hospital of Cologne, Department II of Internal Medicine and Center for Molecular Medicine Cologne, University of Cologne, Cologne, Germany (GRID:grid.411097.a) (ISNI:0000 0000 8852 305X); University of Cologne, Faculty of Medicine and University Hospital of Cologne, Cologne Excellence Cluster on Cellular Stress Responses in Ageing-Associated Diseases (CECAD) and Systems Biology of Ageing Cologne (Sybacol), Cologne, Germany (GRID:grid.6190.e) (ISNI:0000 0000 8580 3777) 
 University Hospitals Leuven, PKD Research Group, Laboratory of Pediatrics, Leuven, Belgium (GRID:grid.410569.f) (ISNI:0000 0004 0626 3338); University Hospitals Leuven, Department of Pediatric Nephrology, Leuven, Belgium (GRID:grid.410569.f) (ISNI:0000 0004 0626 3338) 
Publication year
2020
Publication date
2020
Publisher
Nature Publishing Group
e-ISSN
20452322
Source type
Scholarly Journal
Language of publication
English
ProQuest document ID
2375480882
Copyright
This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.