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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.
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1 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)
2 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)
3 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)
4 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)
5 University Children’s Hospital Heidelberg, Division of Pediatric Nephrology, Heidelberg, Germany (GRID:grid.5253.1) (ISNI:0000 0001 0328 4908)
6 Otsuka Pharmaceutical Development & Commercialization Inc., Princeton, USA (GRID:grid.419943.2) (ISNI:0000 0004 0459 5953)
7 Tufts Medical Center and Tufts University School of Medicine, Division of Nephrology, Boston, USA (GRID:grid.429997.8) (ISNI:0000 0004 1936 7531)
8 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)
9 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)