title: Long-Term Outcomes in Patients with Very-Early Onset Autosomal Dominant Polycystic Kidney Disease.
abstract: Long-term clinical outcomes in children with very-early onset (VEO; diagnosis in utero or within the first 18 months of life) autosomal dominant polycystic kidney disease (ADPKD) are currently not well understood. We conducted a longitudinal retrospective cohort study to assess the association between VEO status and adverse clinical outcomes.
  
  
  
  Seventy patients with VEO-ADPKD matched (by year of birth, sex and race/ethnicity) to 70 patients with non-VEO-ADPKD who participated in research at the University of Colorado were studied. Kaplan-Meier survival analysis was performed. The predictor was VEO status, and outcomes were progression to end-stage renal disease (ESRD), development of hypertension, progression to estimated glomerular filtration rate (eGFR <90 ml/min/1.73 m2), glomerular hyperfiltration (eGFR ≥140 ml/min/1.73 m2) and height-adjusted total kidney volume (htTKV) measured by MRI ≥600 ml/m.
  
  
  
  Median follow-up was until 16.0 years of age. There were only 4 ESRD events during the follow-up period, all in the VEO group (p < 0.05). VEO patients were more likely to develop hypertension (hazard ratio, HR 3.15, 95% CI 1.86-5.34; p < 0.0001) and to progress to eGFR <90 ml/min/1.73 m2 (HR 1.97, 95% CI 1.01-3.84; p < 0.05) than non-VEO patients. There was no difference between groups in the development of glomerular hyperfiltration (HR 0.89, 95% CI 0.56-1.42; p = 0.62). There were only 7 patients who progressed to htTKV ≥600 ml/m, 4 in the VEO group and 3 in the non-VEO group (p < 0.01).
  
  
  
  Several clinical outcomes are worse in patients with VEO-ADPKD compared to non-VEO ADPKD. Children with VEO-ADPKD represent a particularly high-risk group of ADPKD patients.
authors.affiliation: Division of Renal Diseases and Hypertension, University of Colorado Anschutz Medical Campus, Aurora, Colo., USA.
authors.forename: Kristen L
authors.initials: KL
authors.lastname: Nowak
authors.forename: Melissa A
authors.initials: MA
authors.lastname: Cadnapaphornchai
authors.forename: Michel B
authors.initials: MB
authors.lastname: Chonchol
authors.forename: Robert W
authors.initials: RW
authors.lastname: Schrier
authors.forename: Berenice
authors.initials: B
authors.lastname: Gitomer
mesh_terms: D000293:Adolescent; D017668:Age of Onset; D016022:Case-Control Studies; D002648:Child; D018450:Disease Progression; D005260:Female; D005919:Glomerular Filtration Rate; D006801:Humans; D006973:Hypertension / Q000209:etiology*; D007223:Infant; D007231:Infant, Newborn; D053208:Kaplan-Meier Estimate; D007668:Kidney / Q000000981:diagnostic imaging / Q000473:pathology*; D007676:Kidney Failure, Chronic / Q000209:etiology* / Q000503:physiopathology; D008137:Longitudinal Studies; D008297:Male; D009929:Organ Size; D016891:Polycystic Kidney, Autosomal Dominant / Q000150:complications* / Q000175:diagnosis* / Q000503:physiopathology; D011296:Prenatal Diagnosis; D012189:Retrospective Studies; D013997:Time Factors; D055815:Young Adult