Djalila Mekahli1, Karlijn J van Stralen2, Marjolein Bonthuis3, Kitty J Jager2, Ayşe Balat4, Elisa Benetti5, Nathalie Godefroid6, Vidar O Edvardsson7, James G Heaf8, Augustina Jankauskiene9, Larissa Kerecuk10, Svetlana Marinova11, Flora Puteo12, Tomas Seeman13, Aleksandra Zurowska14, Jacques Pirenne15, Franz Schaefer16, Jaap W Groothoff17. 1. Department of Pediatric Nephrology, University Hospitals of Leuven, Leuven, Belgium; Department of Development and Regeneration, KU Leuven, Leuven, Belgium. 2. ESPN/ERA-EDTA Registry, Department of Medical informatics, Academic Medical Center, University of Amsterdam, Amsterdam, the Netherlands. 3. ESPN/ERA-EDTA Registry, Department of Medical informatics, Academic Medical Center, University of Amsterdam, Amsterdam, the Netherlands. Electronic address: m.bonthuis@amc.uva.nl. 4. Department of Pediatric Nephrology and Rheumatology, School of Medicine, Gaziantep University, Gaziantep, Turkey. 5. Pediatric Nephrology, Dialysis and Transplant Unit, Department of Pediatrics, University of Padova, Italy. 6. Department of Pediatrics, Université catholique de Louvain Medical School, Saint-Luc Academic Hospital, Brussels, Belgium. 7. Faculty of Medicine, School of Health Sciences, University of Iceland, Reykjavik, Iceland; Children's Medical Center, Landspitali-The National University Hospital of Iceland, Iceland. 8. Department of Medicine, Roskilde Hospital, University of Copenhagen, Copenhagen, Denmark. 9. Vilnius University Hospital, Center for Pediatrics, Vilnius, Lithuania. 10. Department of Pediatric Nephrology, Birmingham Children's Hospital, National Health Service Foundation Trust, Birmingham, United Kingdom. 11. Clinic of Pediatric Nephrology and Dialysis, University of Sofia, Sofia, Bulgaria. 12. Pediatric Nephrology and Dialysis Unit, Pediatric Hospital Giovanni XXIII, Bari, Italy. 13. University Hospital Motol, 2nd School of Medicine, Charles University Prague, Prague, Czech Republic. 14. Department for Pediatrics, Nephrology & Hypertension, Medical University of Gdansk, Gdansk, Poland. 15. Department of Abdominal Transplantation, University Hospitals of Leuven, Leuven, Belgium. 16. University of Heidelberg Center for Pediatrics and Adolescent Medicine, Heidelberg, Germany. 17. Department of Pediatric, Academic Medical Center, Amsterdam, the Netherlands.
Abstract
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.
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.