Observational studyScientific reports2021
Early childhood height-adjusted total kidney volume as a risk marker of kidney survival in ARPKD.
Observational study in Scientific reports, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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Who cites it
11 citing papers in PubMed, 26 citations in OpenAlex.
- Clinical spectrum, genetic variants, and outcomes of autosomal recessive polycystic kidney disease in Omani children: two-center experience.Pediatric nephrology (Berlin, Germany) · 2026Article
- Predictors of kidney survival in children with autosomal recessive polycystic kidney disease.Clinical and experimental nephrology · 2026Article
- Polycystic Kidney Disease in Children: The Current Status and the Next Horizon.American journal of kidney diseases : the official journal of the National Kidney Foundation · 2025Review
- Kidney volume measurement and predictive modeling in children aged 0-18 years using a computer-assisted surgery system.Scientific reports · 2025Article
- Protocol for the nationwide registry of patients with polycystic kidney disease: japanese national registry of PKD (JRP).Clinical and experimental nephrology · 2024Article
- Kidney volume normative values in Central European children aged 0-19 years: a multicenter study.Pediatric nephrology (Berlin, Germany) · 2024Article
- Polycystic Kidney Disease Drug Development: A Conference Report.Kidney medicine · 2023Article
- Design of two ongoing clinical trials of tolvaptan in the treatment of pediatric patients with autosomal recessive polycystic kidney disease.BMC nephrology · 2023Article
- Autosomal dominant and autosomal recessive polycystic kidney disease: hypertension and secondary cardiovascular effect in children.Frontiers in molecular biosciences · 2023Review
- Evaluation of galectin-3 and intestinal fatty acid binding protein as serum biomarkers in autosomal recessive polycystic kidney disease.Journal of nephrology · 2023Article
- Translational research approaches to study pediatric polycystic kidney disease.Molecular and cellular pediatrics · 2021Review
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Authors and funding
33 authors at 20 institutions in 22 countries.
Funding
No grant is acknowledged in the PubMed record.
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.
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