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Progression of autosomal-dominant polycystic kidney disease in children
G M Fick-Brosnahan1, Z V Tran, A M Johnson
1Department of Medicine,University of Colorado Health Sciences Center, Denver, Colorado 80262, USA. godela.fick-brosnahan@uchsc.edu
Insights
Autosomal-dominant polycystic kidney disease (ADPKD) progression begins in childhood, marked by increased cyst number and kidney size. Children with early severe disease or high blood pressure show faster renal growth, identifying at-risk individuals for future therapies.
Area of Science:
- Pediatric Nephrology
- Genetics
- Medical Research
Background:
- Limited longitudinal data exists on childhood autosomal-dominant polycystic kidney disease (ADPKD) progression.
- Previous studies focused on case reports, lacking large-scale analysis of natural disease course and risk factors in pediatric populations.
- Understanding early ADPKD progression is crucial for timely intervention.
Purpose of the Study:
- To conduct a longitudinal study on the natural progression of ADPKD in children.
- To identify risk factors associated with more rapid renal enlargement in pediatric ADPKD patients.
- To establish baseline data for evaluating future therapeutic interventions in children with ADPKD.
Main Methods:
- Longitudinal study of 312 children from 131 families with ADPKD history since 1985.
- Inclusion of physical examinations, biochemical tests, abdominal ultrasonography, and gene linkage analysis.
- Repeated measurements of renal volumes via ultrasound over up to 15 years for 115 affected children.
Main Results:
- Affected children exhibited significantly faster renal growth compared to unaffected peers.
- Children with early severe ADPKD or hypertension demonstrated accelerated renal enlargement.
- Glomerular filtration rate remained stable in most children, with declines only in two severe early-onset cases.
Conclusions:
- ADPKD progression is evident in childhood, characterized by increased cyst formation and renal volume.
- Early identification of rapid renal enlargement in children is possible.
- This research highlights key indicators for children who may benefit most from emerging ADPKD therapies.
Background:
Although many case reports describe manifestations of autosomal-dominant polycystic kidney disease (ADPKD) in children, no longitudinal studies have examined the natural progression or risk factors for more rapid progression in a large number of children from ADPKD families.
Methods:
Since 1985, we have studied 312 children from 131 families with a history, a physical examination, blood and urine chemistries, an abdominal ultrasonography, and gene linkage analysis. One hundred fifteen of 185 affected children were studied multiple times for up to 15 years. Renal volumes were determined by ultrasound imaging. Graphs of mean renal volumes according to age were compared between affected and unaffected children, ADPKD children with and without early severe disease, and children with and without high blood pressure.
Results:
Affected children had faster renal growth than unaffected children. ADPKD children with severe renal enlargement at a young age continued to experience faster renal growth than those with mild enlargement or normal kidney size for their age, and affected children with high blood pressure had faster renal growth than those with lower blood pressure. Glomerular filtration rate did not decrease in any children except for two with unusually severe early onset disease.
Conclusions:
The progression of ADPKD clearly occurs in childhood and manifests as an increase in cyst number and renal size. This study identifies children at risk for rapid renal enlargement who may benefit the most from future therapeutic interventions.
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