Burgmaier, Kathrin, Kilian, Samuel, Arbeiter, Klaus, Atmis, Bahriye, Buescher, Anja, Derichs, Ute, Dursun, Ismail, Duzova, Ali, Eid, Loai Akram, Galiano, Matthias, Gessner, Michaela, Gokce, Ibrahim, Haeffner, Karsten, Hooman, Nakysa, Jankauskiene, Augustina, Koerber, Friederike, Longo, Germana, Massella, Laura, Mekahli, Djalila, Milosevski-Lomic, Gordana, Nalcacioglu, Hulya ORCID: 0000-0002-0686-9714, Rus, Rina, Shroff, Rukshana ORCID: 0000-0001-8501-1072, Stabouli, Stella, Weber, Lutz T., Wygoda, Simone, Yilmaz, Alev, Zachwieja, Katarzyna, Zagozdzon, Ilona, Doetsch, Joerg, Schaefer, Franz and Liebau, Max Christoph (2021). Early childhood height-adjusted total kidney volume as a risk marker of kidney survival in ARPKD. Sci Rep, 11 (1). BERLIN: NATURE PORTFOLIO. ISSN 2045-2322

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Abstract

Autosomal 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.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Burgmaier, KathrinUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kilian, SamuelUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Arbeiter, KlausUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Atmis, BahriyeUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Buescher, AnjaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Derichs, UteUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Dursun, IsmailUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Duzova, AliUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Eid, Loai AkramUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Galiano, MatthiasUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Gessner, MichaelaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Gokce, IbrahimUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Haeffner, KarstenUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Hooman, NakysaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Jankauskiene, AugustinaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Koerber, FriederikeUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Longo, GermanaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Massella, LauraUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Mekahli, DjalilaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Milosevski-Lomic, GordanaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Nalcacioglu, HulyaUNSPECIFIEDorcid.org/0000-0002-0686-9714UNSPECIFIED
Rus, RinaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Shroff, RukshanaUNSPECIFIEDorcid.org/0000-0001-8501-1072UNSPECIFIED
Stabouli, StellaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Weber, Lutz T.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Wygoda, SimoneUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Yilmaz, AlevUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Zachwieja, KatarzynaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Zagozdzon, IlonaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Doetsch, JoergUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schaefer, FranzUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Liebau, Max ChristophUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
URN: urn:nbn:de:hbz:38-606884
DOI: 10.1038/s41598-021-00523-z
Journal or Publication Title: Sci Rep
Volume: 11
Number: 1
Date: 2021
Publisher: NATURE PORTFOLIO
Place of Publication: BERLIN
ISSN: 2045-2322
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
DISEASEMultiple languages
Multidisciplinary SciencesMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/60688

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