BACKGROUND: The choice for either kidney or combined liver-kidney transplantation in young people with kidney failure and liver fibrosis due to autosomal recessive polycystic kidney disease (ARPKD) can be challenging. We aimed to analyze the characteristics and outcomes of transplantation type in these children, adolescents, and young adults. STUDY DESIGN: Cohort study. SETTING & PARTICIPANTS: We derived data for children, adolescents, and young adults with ARPKD with either kidney or combined liver-kidney transplants for 1995 to 2012 from the ESPN/ERA-EDTA Registry, a European pediatric renal registry collecting data from 36 European countries. FACTOR: Liver transplantation. OUTCOMES & MEASUREMENTS: Transplantation and patient survival. RESULTS: 202 patients with ARPKD aged 19 years or younger underwent transplantation after a median of 0.4 (IQR, 0.0-1.4) years on dialysis therapy at a median age of 9.0 (IQR, 4.1-13.7) years. 32 (15.8%) underwent combined liver-kidney transplantation, 163 (80.7%) underwent kidney transplantation, and 7 (3.5%) were excluded because transplantation type was unknown. Age- and sex-adjusted 5-year patient survival posttransplantation was 95.5% (95% CI, 92.4%-98.8%) overall: 97.4% (95% CI, 94.9%-100.0%) for patients with kidney transplantation in contrast to 87.0% (95% CI, 75.8%-99.8%) with combined liver-kidney transplantation. The age- and sex-adjusted risk for death after combined liver-kidney transplantation was 6.7-fold (95% CI, 1.8- to 25.4-fold) greater than after kidney transplantation (P=0.005). Five-year death-censored kidney transplant survival following combined liver-kidney and kidney transplantation was similar (92.1% vs 85.9%; P=0.4). LIMITATIONS: No data for liver disease of kidney therapy recipients. CONCLUSIONS: Combined liver-kidney transplantation in ARPKD is associated with increased mortality compared to kidney transplantation in our large observational study and was not associated with improved 5-year kidney transplant survival. Long-term follow-up of both kidney and liver involvement are needed to better delineate the optimal transplantation strategy.

Clinic of Pediatric Nephrology and Dialysis University of Sofia Sofia Bulgaria

Department for Pediatrics Nephrology and Hypertension Medical University of Gdansk Gdansk Poland

Department of Abdominal Transplantation University Hospitals of Leuven Leuven Belgium

Department of Medicine Roskilde Hospital University of Copenhagen Copenhagen Denmark

Department of Pediatric Academic Medical Center Amsterdam the Netherlands

Department of Pediatric Nephrology and Rheumatology School of Medicine Gaziantep University Gaziantep Turkey

Department of Pediatric Nephrology Birmingham Children's Hospital National Health Service Foundation Trust Birmingham United Kingdom

Department of Pediatric Nephrology University Hospitals of Leuven Leuven Belgium; Department of Development and Regeneration KU Leuven Leuven Belgium

Department of Pediatrics Université catholique de Louvain Medical School Saint Luc Academic Hospital Brussels Belgium

ESPN ERA EDTA Registry Department of Medical informatics Academic Medical Center University of Amsterdam Amsterdam the Netherlands

Faculty of Medicine School of Health Sciences University of Iceland Reykjavik Iceland; Children's Medical Center Landspitali The National University Hospital of Iceland Iceland

Pediatric Nephrology and Dialysis Unit Pediatric Hospital Giovanni XXIII Bari Italy

Pediatric Nephrology Dialysis and Transplant Unit Department of Pediatrics University of Padova Italy

University Hospital Motol 2nd School of Medicine Charles University Prague Prague Czech Republic

University of Heidelberg Center for Pediatrics and Adolescent Medicine Heidelberg Germany

Vilnius University Hospital Center for Pediatrics Vilnius Lithuania

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Design of two ongoing clinical trials of tolvaptan in the treatment of pediatric patients with autosomal recessive polycystic kidney disease