Janssens, Peter, Jouret, Francois, Bammens, Bert, Liebau, Max C., Schaefer, Franz, Dandurand, Ann, Perrone, Ronald D., Mueller, Roman-Ulrich, Pao, Christina S. and Mekahli, Djalila ORCID: 0000-0003-0954-6088 (2020). Implications of early diagnosis of autosomal dominant polycystic kidney disease: A post hoc analysis of the TEMPO 3:4 trial. Sci Rep, 10 (1). LONDON: NATURE PUBLISHING GROUP. ISSN 2045-2322

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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 m(2); 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 m(2); 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 Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Janssens, PeterUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Jouret, FrancoisUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bammens, BertUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Liebau, Max C.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schaefer, FranzUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Dandurand, AnnUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Perrone, Ronald D.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Mueller, Roman-UlrichUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Pao, Christina S.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Mekahli, DjalilaUNSPECIFIEDorcid.org/0000-0003-0954-6088UNSPECIFIED
URN: urn:nbn:de:hbz:38-341283
DOI: 10.1038/s41598-020-61303-9
Journal or Publication Title: Sci Rep
Volume: 10
Number: 1
Date: 2020
Publisher: NATURE PUBLISHING GROUP
Place of Publication: LONDON
ISSN: 2045-2322
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
GLOMERULAR HYPERFILTRATION; TOLVAPTAN; VOLUME; PROGRESSION; CHILDRENMultiple languages
Multidisciplinary SciencesMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/34128

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