Olivier Guillaud1, Jérôme Dumortier2, Rodolphe Sobesky3, Dominique Debray4, Philippe Wolf5, Claire Vanlemmens6, François Durand7, Yvon Calmus8, Christophe Duvoux9, Sébastien Dharancy10, Nassim Kamar11, Karim Boudjema12, Pierre Henri Bernard13, Georges-Philippe Pageaux14, Ephrem Salamé15, Jean Gugenheim16, Alain Lachaux17, Dalila Habes18, Sylvie Radenne19, Jean Hardwigsen20, Olivier Chazouillères21, Jean-Marc Trocello22, France Woimant22, Philippe Ichai3, Sophie Branchereau18, Olivier Soubrane23, Denis Castaing3, Emmanuel Jacquemin18, Didier Samuel3, Jean-Charles Duclos-Vallée3. 1. Centre National de Référence de la Maladie de Wilson/Fédération des Spécialités Digestives, Hôpital Édouard Herriot, Hospices Civils de Lyon, Lyon, France. Electronic address: olivier.guillaud@chu-lyon.fr. 2. Centre National de Référence de la Maladie de Wilson/Fédération des Spécialités Digestives, Hôpital Édouard Herriot, Hospices Civils de Lyon, Lyon, France. 3. Centre National de Référence de la Maladie de Wilson/Centre Hépato-Biliaire, Hôpital Paul Brousse, AP-HP, Villejuif, France; UMR 785, INSERM, France; UMR-S 785, Univ Paris-Sud, Villejuif, France; DHU Hepatinov, Villejuif, France. 4. Service d'Hépatologie Pédiatrique, Hôpital Necker-Enfants Malades, AP-HP, Paris, France. 5. Service de Chirurgie générale et Transplantation, Hôpital Hautefeuille, CHU Strasbourg, France. 6. Service d'Hépatologie, Hôpital Jean Minjoz, CHU Besançon, France. 7. Service d'Hépatologie, Hôpital Beaujon, AP-HP, Clichy, France. 8. Service de Chirurgie, Hôpital Cochin, AP-HP, Paris, France. 9. Service d'Hépatologie, Hôpital Henri Mondor, AP-HP, Créteil, France. 10. Service d'Hépato-Gastroentérologie, Hôpital Claude Huriez, CHRU Lille, Lille, France. 11. Service de Néphrologie-Hypertension artérielle-Dialyse-Transplantation, Hôpital Rangueil, CHU de Toulouse, France. 12. Service de Chirurgie Hépatobiliaire et Digestive, Hôpital de Pontchaillou, CHU de Rennes, France. 13. Service d'Hépatologie et de Gastroentérologie, Hôpital Pellegrin, Bordeaux, France. 14. Fédération Médico-Chirurgicale des Maladies de l'Appareil Digestif, Hôpital Saint-Eloi, Montpellier, France. 15. Service de Chirurgie Digestive, CHU Bretonneau, Tours, France. 16. Service de Chirurgie Digestive, Hôpital L'Archet (2), CHU Nice, Nice, France. 17. Centre National de Référence de la Maladie de Wilson/Service de Pédiatrie, Hôpital Femme Mère Enfant, Hospices Civils de Lyon, Bron, France. 18. Centre National de Référence de la Maladie de Wilson/Service d'Hépatologie et de Transplantation Hépatique Pédiatriques, Hôpital Bicêtre, AP-HP, Le Kremlin-Bicêtre, France; DHU Hepatinov, Villejuif, France. 19. Service d'Hépatologie, Hôpital de la Croix-Rousse, Hospices civils de Lyon, Lyon, France. 20. Service de Chirurgie Digestive, Hôpital la Conception, Marseille, France. 21. Service d'Hépatologie, Hôpital St Antoine, AP-HP, Paris, France. 22. Centre National de Référence de la Maladie de Wilson/Service de Neurologie, Hôpital Lariboisière, AP-HP, Paris, France. 23. Service de chirurgie hépatobiliaire et transplantation hépatique, Hôpital St Antoine, AP-HP, Paris, France.
Abstract
BACKGROUND & AIMS: Liver transplantation (LT) is the therapeutic option for severe complications of Wilson's disease (WD). We aimed to report on the long-term outcome of WD patients following LT. METHODS: The medical records of 121 French patients transplanted for WD between 1985 and 2009 were reviewed retrospectively. Seventy-five patients were adults (median age: 29 years, (18-66)) and 46 were children (median age: 14 years, (7-17)). The indication for LT was (1) fulminant/subfulminant hepatitis (n = 64, 53%), median age = 16 years (7-53), (2) decompensated cirrhosis (n = 50, 41%), median age = 31.5 years (12-66) or (3) severe neurological disease (n = 7, 6%), median age = 21.5 years (14.5-42). Median post-transplant follow-up was 72 months (0-23.5). RESULTS: Actuarial patient survival rates were 87% at 5, 10, and 15 years. Male gender, pre-transplant renal insufficiency, non elective procedure, and neurological indication were significantly associated with poorer survival rate. None of these factors remained statistically significant under multivariate analysis. In patients transplanted for hepatic indications, the prognosis was poorer in case of fulminant or subfulminant course, non elective procedure, pretransplant renal insufficiency and in patients transplanted before 2000. Multivariate analysis disclosed that only recent period of LT was associated with better prognosis. At last visit, the median calculated glomerular filtration rate was 93 ml/min (33-180); 11/93 patients (12%) had stage II renal insufficiency and none had stage III. CONCLUSIONS: Liver failure associated with WD is a rare indication for LT (<1%), which achieves an excellent long-term outcome, including renal function.
BACKGROUND & AIMS: Liver transplantation (LT) is the therapeutic option for severe complications of Wilson's disease (WD). We aimed to report on the long-term outcome of WDpatients following LT. METHODS: The medical records of 121 French patients transplanted for WD between 1985 and 2009 were reviewed retrospectively. Seventy-five patients were adults (median age: 29 years, (18-66)) and 46 were children (median age: 14 years, (7-17)). The indication for LT was (1) fulminant/subfulminant hepatitis (n = 64, 53%), median age = 16 years (7-53), (2) decompensated cirrhosis (n = 50, 41%), median age = 31.5 years (12-66) or (3) severe neurological disease (n = 7, 6%), median age = 21.5 years (14.5-42). Median post-transplant follow-up was 72 months (0-23.5). RESULTS: Actuarial patient survival rates were 87% at 5, 10, and 15 years. Male gender, pre-transplant renal insufficiency, non elective procedure, and neurological indication were significantly associated with poorer survival rate. None of these factors remained statistically significant under multivariate analysis. In patients transplanted for hepatic indications, the prognosis was poorer in case of fulminant or subfulminant course, non elective procedure, pretransplant renal insufficiency and in patients transplanted before 2000. Multivariate analysis disclosed that only recent period of LT was associated with better prognosis. At last visit, the median calculated glomerular filtration rate was 93 ml/min (33-180); 11/93 patients (12%) had stage II renal insufficiency and none had stage III. CONCLUSIONS:Liver failure associated with WD is a rare indication for LT (<1%), which achieves an excellent long-term outcome, including renal function.