Long-Term Transplantation Outcomes in Patients With Primary Hyperoxaluria Type 1 Included in the European Hyperoxaluria Consortium (OxalEurope) Registry.
Metry, Elisabeth L; Garrelfs, Sander F; Peters-Sengers, Hesselet al.
2022 • In Kidney International Reports, 7 (2), p. 210-220
[en] INTRODUCTION: In primary hyperoxaluria type 1 (PH1), oxalate overproduction frequently causes kidney stones, nephrocalcinosis, and kidney failure. As PH1 is caused by a congenital liver enzyme defect, combined liver-kidney transplantation (CLKT) has been recommended in patients with kidney failure. Nevertheless, systematic analyses on long-term transplantation outcomes are scarce. The merits of a sequential over combined procedure regarding kidney graft survival remain unclear as is the place of isolated kidney transplantation (KT) for patients with vitamin B6-responsive genotypes. METHODS: We used the OxalEurope registry for retrospective analyses of patients with PH1 who underwent transplantation. Analyses of crude Kaplan-Meier survival curves and adjusted relative hazards from the Cox proportional hazards model were performed. RESULTS: A total of 267 patients with PH1 underwent transplantation between 1978 and 2019. Data of 244 patients (159 CLKTs, 48 isolated KTs, 37 sequential liver-KTs [SLKTs]) were eligible for comparative analyses. Comparing CLKTs with isolated KTs, adjusted mortality was similar in patients with B6-unresponsive genotypes but lower after isolated KT in patients with B6-responsive genotypes (adjusted hazard ratio 0.07, 95% CI: 0.01-0.75, P = 0.028). CLKT yielded higher adjusted event-free survival and death-censored kidney graft survival in patients with B6-unresponsive genotypes (P = 0.025, P < 0.001) but not in patients with B6-responsive genotypes (P = 0.145, P = 0.421). Outcomes for 159 combined procedures versus 37 sequential procedures were comparable. There were 12 patients who underwent pre-emptive liver transplantation (PLT) with poor outcomes. CONCLUSION: The CLKT or SLKT remains the preferred transplantation modality in patients with PH1 with B6-unresponsive genotypes, but isolated KT could be an alternative approach in patients with B6-responsive genotypes.
Disciplines :
Urology & nephrology Pediatrics
Author, co-author :
Metry, Elisabeth L; Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC,
Garrelfs, Sander F; Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC,
Peters-Sengers, Hessel; Center for Experimental and Molecular Medicine, Amsterdam UMC, University of
Hulton, Sally-Anne; Department of Nephrology, Birmingham Women's and Children's Hospital NHS
Acquaviva, Cecile; Service de Biochimie et Biologie Moléculaire, UM Pathologies Héréditaires du
Bacchetta, Justine; Centre de Référence des Maladies Rares Néphrogones, Hospices Civils de Lyon &
Beck, Bodo B; Institute of Human Genetics, Center for Molecular Medicine Cologne, University ; Center for Rare and Hereditary Kidney Disease Cologne, University Hospital of
Collard, Laure ; Centre Hospitalier Universitaire de Liège - CHU > > Service de pédiatrie
Deschênes, Georges; Department of Pediatric Nephrology, Assistance Publique-Hôpitaux de Paris
Franssen, Casper; Department of Internal Medicine, University Medical Center Groningen, University
Kemper, Markus J; Division of Pediatric Nephrology, University Children's Hospital, Hamburg,
Lipkin, Graham W; Department of Nephrology, University Hospitals Birmingham NHS Foundation Trust,
Mandrile, Giorgia; Medical Genetics Unit, Department of Clinical and Biological Sciences, University ; Thalassemia Unit, San Luigi University Hospital, Orbassano, Italy.
Mohebbi, Nilufar; Division of Nephrology, University Hospital Zurich, Zurich, Switzerland.
Moochhala, Shabbir H; UCL Department of Renal Medicine, Royal Free Hospital, London, UK.
Oosterveld, Michiel J S; Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC,
Prikhodina, Larisa; Department of Inherited and Acquired Kidney Diseases, Research and Clinical
Hoppe, Bernd; Department of Pediatric Nephrology, Children's Hospital of the University of
Cochat, Pierre; Centre de Référence des Maladies Rares Néphrogones, Hospices Civils de Lyon &
Groothoff, Jaap W; Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC,
Long-Term Transplantation Outcomes in Patients With Primary Hyperoxaluria Type 1 Included in the European Hyperoxaluria Consortium (OxalEurope) Registry.
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