A risk score to predict kidney survival in patients with autosomal recessive polycystic kidney disease at the age of two months

Burgmaier K, Kilian S, Arbeiter K, Atmis B, Boyer O, Buescher A, Dursun I, Erger F, Fila M, Galiano M, Gokce I, Haeffner K, Haffner D, Hooman N, Klaus G, König J, Lange-Sperandio B, Marlais M, Massella L, Mekahli D, Miklaszewska M, Miloševski-Lomić G, Obrycki L, Ranchin B, Seitz B, Stabouli S, Tabel Y, Taranta-Janusz K, Weber LT, Weitz M, Wühl E, Yilmaz A, Dötsch J, Schaefer F, Liebau MC (2025)

Publication Language: English

Publication Type: Journal article

Publication year: 2025

Journal

Book Volume: 107

Pages Range: 903-915

Journal Issue: 5

DOI: 10.1016/j.kint.2025.01.023

Abstract

Autosomal recessive polycystic kidney disease (ARPKD) is a severe hepatorenal fibrocystic disorder. Its rareness and the variability of disease courses have been major obstacles for the establishment of clinical trials on treatment of kidney disease in ARPKD. In this observational study we characterized kidney disease progression in a very large cohort of up to 658 patients with the clinical diagnosis of ARPKD and identified risk factors associated with rapid kidney disease progression. The estimated probability of kidney failure by the age of 20 years was 50.1% (95% confidence interval 42.2%‒57.0%), with earlier kidney failure in specific subgroups. Mean yearly estimated glomerular filtration rate decline after the first year of life was 1.3 ml/min per 1.73 m² during childhood and adolescence in the overall cohort, ranging from 0.5 to 2.2 ml/min per 1.73 m² in various subgroups. Furthermore, we developed prediction models for the relative risk of early kidney failure to be applied at the age of two months in daily clinical life. The finally chosen predictor set for a score based on a Cox model encompassed five factors: gestational age at oligo- or anhydramnios, gestational age at birth, functional genotype, serum creatinine (mg/dl) as well as documentation of arterial hypertension at the age of two months. The derived simple prognostic score showed good prediction performance, especially in the first three years of life. It reliably identified patients who are not at risk of early kidney failure and may be helpful to identify patients at risk of more rapid disease progression that could benefit from novel therapeutic interventions.

Involved external institutions

Universitätsklinikum Köln Germany (DE) Ruprecht-Karls-Universität Heidelberg Germany (DE) University College London (UCL) United Kingdom (GB) Medizinische Universität Wien Austria (AT) Çukurova Üniversitesi Turkey (TR) Université Sorbonne Paris Cité France (FR) Universitätsklinikum Essen Germany (DE) Erciyes University / Erciyes Üniversitesi Turkey (TR) Centre Hospitalier Universitaire de Montpellier (CHU/CHRU MTP) France (FR) Marmara University Turkey (TR) Albert-Ludwigs-Universität Freiburg Germany (DE) Medizinische Hochschule Hannover (MHH) / Hannover Medical School Germany (DE) Ali Asghar Children Hospital / (مرکز آموزشی درمانی حضرت علی اصغر (ص Iran, Islamic Republic of (IR) Philipps-Universität Marburg Germany (DE) Universitätsklinikum Münster Germany (DE) Klinikum der Universität München Germany (DE) Ospedale Pediatrico Bambino Gesu Italy (IT) Katholieke Universiteit Leuven (KUL) / Catholic University of Leuven Belgium (BE) Jagiellonian University Medical College Poland (PL) University Children's Hospital / Univerzitetska dečja klinika u Beogradu (Tiršova) Serbia (RS) Children's Memorial Health Institute / Instytut "Pomnik - Centrum Zdrowia Dziecka" Poland (PL) Hospices Civils de Lyon (CHU) France (FR) Inonu Univeristy Turkey (TR) Medical University of Bialystok Poland (PL) Universitätsklinikum Tübingen Germany (DE) Universitätsklinikum Heidelberg Germany (DE) Istanbul University / İstanbul Üniversitesi Turkey (TR) AHEPA University General Hospital / Πανεπιστημιακό Νοσοκομείο ΑΧΕΠΑ Greece (GR) Klinikum Schwabing Germany (DE)

How to cite

APA:

Burgmaier, K., Kilian, S., Arbeiter, K., Atmis, B., Boyer, O., Buescher, A.,... Liebau, M.C. (2025). A risk score to predict kidney survival in patients with autosomal recessive polycystic kidney disease at the age of two months. Kidney International, 107(5), 903-915. https://doi.org/10.1016/j.kint.2025.01.023

MLA:

Burgmaier, Kathrin, et al. "A risk score to predict kidney survival in patients with autosomal recessive polycystic kidney disease at the age of two months." Kidney International 107.5 (2025): 903-915.

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