Abstract
BackgroundAs outcome data for prune belly syndrome (PBS) complicated by end-stage renal disease are scarce, we analyzed characteristics and outcomes of children with PBS using the European Society for Pediatric Nephrology/European Renal Association-European Dialysis and Transplant Association (ESPN/ERA-EDTA) Registry data.MethodsData were available for 88 male PBS patients aged <20 years who started renal replacement therapy (RRT) between 1990 and 2013 in 35 European countries. Patient characteristics, survival, and transplantation outcomes were compared with those of male patients requiring RRT due to congenital obstructive uropathy (COU) and renal hypoplasia or dysplasia (RHD).ResultsMedian age at onset of RRT in PBS was lower [7.0; interquartile range (IQR) 0.9–12.2 years] than in COU (9.6; IQR: 3.0–14.1 years) and RHD (9.4; IQR: 2.7–14.2 years). Unadjusted 10-year patient survival was 85% for PBS, 94% for COU, and 91% for RHD. After adjustment for country, period, and age, PBS mortality was similar to that of RHD but higher compared with COU [hazard ratio (HR) 1.96, 95% confidence interval (CI) 1.03–3.74]. Seventy-four PBS patients (84%) received a first kidney transplant after a median time on dialysis of 8.4 (IQR 0.0–21.1) months. Outcomes with respect to time on dialysis before transplantation, chance of receiving a first transplant within 2 years after commencing RRT, and death-censored, adjusted risk of graft loss were similar for all groups.ConclusionsThis study in the largest cohort of male patients with PBS receiving RRT to date demonstrates that outcomes are comparable with other congenital anomalies of the kidney and urinary tract, except for a slightly higher mortality risk compared with patients with COU.
Highlights
Prune belly syndrome (PBS) is a very rare congenital disorder that consists of a deficiency in the development of anterior abdominal wall muscles, variable amounts of urinary tract dilatation, and cryptorchidism
This study in the largest cohort of male patients with prune belly syndrome (PBS) receiving renal replacement therapy (RRT) to date demonstrates that outcomes are comparable with other congenital anomalies of the kidney and urinary tract, except for a slightly higher mortality risk compared with patients with congenital obstructive uropathy (COU)
We aimed to describe the incidence, clinical, and demographic characteristics of boys with PBS requiring RRT using the European Society of Pediatric Nephrology/European Renal Association–European Dialysis and Transplant Association (ESPN/ERA–EDTA) Registry
Summary
Prune belly syndrome (PBS) is a very rare congenital disorder that consists of a deficiency in the development of anterior abdominal wall muscles, variable amounts of urinary tract dilatation, and cryptorchidism. Other fetal malformations that may be associated with PBS include gastrointestinal, cardiac, pulmonary, and limb abnormalities [1]. Reports of more than one PBS case in the same family have suggested a genetic contribution. Several gene loci have been defined, a clear genetic basis for PBS has not yet been established [4,5,6,7]. It is thought that PBS arises from a defect in the intermediate and lateral plate mesoderm development during the 6th to 10th week of gestation, resulting in clinical abnormalities [2, 8]. As outcome data for prune belly syndrome (PBS) complicated by end-stage renal disease are scarce, we analyzed characteristics and outcomes of children with PBS using the European Society for Pediatric Nephrology/ European Renal Association-European Dialysis and Transplant Association (ESPN/ERA-EDTA) Registry data
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