Early childhood height-adjusted total kidney volume as a risk marker of kidney survival in ARPKD

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dc.authorscopusid57196234704
dc.authorscopusid57203816230
dc.authorscopusid57223689292
dc.authorscopusid56646436200
dc.authorscopusid35340836700
dc.authorscopusid57200637148
dc.authorscopusid16021105100
dc.contributor.authorBurgmaier K.
dc.contributor.authorKilian S.
dc.contributor.authorArbeiter K.
dc.contributor.authorAtmis B.
dc.contributor.authorBüscher A.
dc.contributor.authorDerichs U.
dc.contributor.authorDursun I.
dc.date.accessioned2024-08-04T20:03:30Z
dc.date.available2024-08-04T20:03:30Z
dc.date.issued2021
dc.departmentİnönü Üniversitesien_US
dc.description.abstractAutosomal recessive polycystic kidney disease (ARPKD) is characterized by bilateral fibrocystic changes resulting in pronounced kidney enlargement. Impairment of kidney function is highly variable and widely available prognostic markers are urgently needed as a base for clinical decision-making and future clinical trials. In this observational study we analyzed the longitudinal development of sonographic kidney measurements in a cohort of 456 ARPKD patients from the international registry study ARegPKD. We furthermore evaluated correlations of sonomorphometric findings and functional kidney disease with the aim to describe the natural disease course and to identify potential prognostic markers. Kidney pole-to-pole (PTP) length and estimated total kidney volume (eTKV) increase with growth throughout childhood and adolescence despite individual variability. Height-adjusted PTP length decreases over time, but such a trend cannot be seen for height-adjusted eTKV (haeTKV) where we even observed a slight mean linear increase of 4.5 ml/m per year during childhood and adolescence for the overall cohort. Patients with two null PKHD1 variants had larger first documented haeTKV values than children with missense variants (median (IQR) haeTKV 793 (450–1098) ml/m in Null/null, 403 (260–538) ml/m in Null/mis, 230 (169–357) ml/m in Mis/mis). In the overall cohort, estimated glomerular filtration rate decreases with increasing haeTKV (median (IQR) haeTKV 210 (150–267) ml/m in CKD stage 1, 472 (266–880) ml/m in stage 5 without kidney replacement therapy). Strikingly, there is a clear correlation between haeTKV in the first eighteen months of life and kidney survival in childhood and adolescence with ten-year kidney survival rates ranging from 20% in patients of the highest to 94% in the lowest quartile. Early childhood haeTKV may become an easily obtainable prognostic marker of kidney disease in ARPKD, e.g. for the identification of patients for clinical studies. © 2021, The Author(s).en_US
dc.description.sponsorshipESPN 2014.2; PKD Foundation, PKDF; Bundesministerium für Bildung und Forschung, BMBF: 01GM1515, 01GM1903; Bundesministerium für Bildung und Forschung, BMBF; Universität zu Köln, UoC; Marga und Walter Boll-Stiftung; Universitätsklinikum Kölnen_US
dc.description.sponsorshipWe thank the German Society for Pediatric Nephrology (GPN), the ESCAPE Network, and the European Society for Paediatric Nephrology (ESPN; Working Groups CAKUT and Inherited Renal Diseases) for their support. ML was supported by grants of the GPN, ESPN (Grant ESPN 2014.2), and the German PKD foundation. KB and ML were supported by the Medical Faculty of the University of Cologne (Koeln Fortune program), and the Marga and Walter Boll-Foundation. FS and ML are supported by the German Federal Ministry of Research and Education (BMBF grant 01GM1515 and 01GM1903). This work was generated within the European Reference Network for Rare Kidney Disorders (ERKNet).en_US
dc.identifier.doi10.1038/s41598-021-00523-z
dc.identifier.issn2045-2322
dc.identifier.issue1en_US
dc.identifier.pmid34737334en_US
dc.identifier.scopus2-s2.0-85118765226en_US
dc.identifier.scopusqualityQ1en_US
dc.identifier.urihttps://doi.org/10.1038/s41598-021-00523-z
dc.identifier.urihttps://hdl.handle.net/11616/91872
dc.identifier.volume11en_US
dc.indekslendigikaynakScopusen_US
dc.indekslendigikaynakPubMeden_US
dc.language.isoenen_US
dc.publisherNature Researchen_US
dc.relation.ispartofScientific Reportsen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/openAccessen_US
dc.subjectbiological markeren_US
dc.subjectcell surface receptoren_US
dc.subjectPKHD1 protein, humanen_US
dc.subjectadolescenten_US
dc.subjectchilden_US
dc.subjectchronic kidney failureen_US
dc.subjectcohort analysisen_US
dc.subjectdisease exacerbationen_US
dc.subjectechographyen_US
dc.subjectfemaleen_US
dc.subjectgeneticsen_US
dc.subjectglomerulus filtration rateen_US
dc.subjecthumanen_US
dc.subjectinfanten_US
dc.subjectkidneyen_US
dc.subjectkidney polycystic diseaseen_US
dc.subjectliver cirrhosisen_US
dc.subjectlongitudinal studyen_US
dc.subjectmaleen_US
dc.subjectmetabolismen_US
dc.subjectmortalityen_US
dc.subjectorgan sizeen_US
dc.subjectpathophysiologyen_US
dc.subjectphysiologyen_US
dc.subjectpreschool childen_US
dc.subjectprognosisen_US
dc.subjectAdolescenten_US
dc.subjectBiomarkersen_US
dc.subjectChilden_US
dc.subjectChild, Preschoolen_US
dc.subjectCohort Studiesen_US
dc.subjectDisease Progressionen_US
dc.subjectFemaleen_US
dc.subjectGlomerular Filtration Rateen_US
dc.subjectHumansen_US
dc.subjectInfanten_US
dc.subjectKidneyen_US
dc.subjectLiver Cirrhosisen_US
dc.subjectLongitudinal Studiesen_US
dc.subjectMaleen_US
dc.subjectOrgan Sizeen_US
dc.subjectPolycystic Kidney, Autosomal Recessiveen_US
dc.subjectPrognosisen_US
dc.subjectReceptors, Cell Surfaceen_US
dc.subjectRenal Insufficiency, Chronicen_US
dc.subjectUltrasonographyen_US
dc.titleEarly childhood height-adjusted total kidney volume as a risk marker of kidney survival in ARPKDen_US
dc.typeArticleen_US

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