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

Burgmaier K, Kilian S, Arbeiter K, Atmis B, Büscher A, Derichs U, Dursun I, Duzova A, Eid LA, Galiano M, Gessner M, Gokce I, Haeffner K, Hooman N, Jankauskiene A, Körber F, Longo G, Massella L, Mekahli D, Miloševski-Lomić G, Nalcacioglu H, Rus R, Shroff R, Stabouli S, Weber LT, Wygoda S, Yilmaz A, Zachwieja K, Zagozdzon I, Dötsch J, Schaefer F, Liebau MC, Consortium TA (2021)

Publication Type: Journal article

Publication year: 2021

Journal

Book Volume: 11

Article Number: 21677

Journal Issue: 1

DOI: 10.1038/s41598-021-00523-z

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.

Additional Organisation(s)

Involved external institutions

Azienda Ospedale Università Padova / University Hospital of Padova Italy (IT) University College London (UCL) United Kingdom (GB) AHEPA University General Hospital / Πανεπιστημιακό Νοσοκομείο ΑΧΕΠΑ Greece (GR) Universitätsklinikum Köln Germany (DE) Çukurova Üniversitesi Turkey (TR) Johannes Gutenberg-Universität Mainz (JGU) Germany (DE) Hacettepe University Turkey (TR) Dubai Hospital United Arab Emirates (AE) Marmara University Turkey (TR) Universitätsklinikum Freiburg Germany (DE) Katholieke Universiteit Leuven (KUL) / Catholic University of Leuven Belgium (BE) University Children's Hospital / Univerzitetska dečja klinika u Beogradu (Tiršova) Serbia (RS) Ljubljana University Medical Centre (Ljubljana UMC) / Univerzitetni klinični center Ljubljana Slovenia (SI) Klinikum St. Georg Germany (DE) Ospedale Pediatrico Bambino Gesu Italy (IT) Jagiellonian University / Uniwersytet Jagielloński (UJ) Poland (PL) Medical University Gdansk / Gdański Uniwersytet Medyczny Poland (PL) Universitätsklinikum Heidelberg Germany (DE) Ondokuz Mayıs University Turkey (TR) Istanbul University / İstanbul Üniversitesi Turkey (TR) Ruprecht-Karls-Universität Heidelberg Germany (DE) Medizinische Universität Wien Austria (AT) Universitätsklinikum Essen Germany (DE) Erciyes University / Erciyes Üniversitesi Turkey (TR) Universitätsklinikum Tübingen Germany (DE) Ali Asghar Children Hospital / (مرکز آموزشی درمانی حضرت علی اصغر (ص Iran, Islamic Republic of (IR) Vilnius University / Vilniaus universitetas Lithuania (LT)

How to cite

APA:

Burgmaier, K., Kilian, S., Arbeiter, K., Atmis, B., Büscher, A., Derichs, U.,... Consortium, T.A. (2021). Early childhood height-adjusted total kidney volume as a risk marker of kidney survival in ARPKD. Scientific Reports, 11(1). https://doi.org/10.1038/s41598-021-00523-z

MLA:

Burgmaier, Kathrin, et al. "Early childhood height-adjusted total kidney volume as a risk marker of kidney survival in ARPKD." Scientific Reports 11.1 (2021).

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