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

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.
AuthorsPeter Janssens, François Jouret, Bert Bammens, Max C Liebau, Franz Schaefer, Ann Dandurand, Ronald D Perrone, Roman-Ulrich Müller, Christina S Pao, Djalila Mekahli
JournalScientific reports (Sci Rep) Vol. 10 Issue 1 Pg. 4294 (03 09 2020) ISSN: 2045-2322 [Electronic] England
PMID32152377 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
Topics
  • Adult
  • Case-Control Studies
  • Disease Management
  • Disease Progression
  • Early Diagnosis
  • Female
  • Follow-Up Studies
  • Glomerular Filtration Rate
  • Humans
  • Male
  • Polycystic Kidney, Autosomal Dominant (diagnosis, therapy)
  • Prognosis
  • Prospective Studies
  • Quality of Life

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