https://www.ncbi.nlm.nih.gov/pubmed/30021057 Wilson disease

Liver Transpl. 2018 Jul 18. doi: 10.1002/lt.25308. [Epub ahead of print].Predictive factors for survival in children liver transplanted for Wilson disease: a cohort study using ELTR data.
Pfister ED1, Karch A2,3, Polak WG4, Karam V5,6, Adam R5,6, Duvoux C7, Mirza D8, O Grady J9, Klempnauer J10, Reding R11, Kalicinski P12, Coker A13, Trunecka P14, Astarcioglu I15, Duclos-Vallée JC6, Pratschke J16, Paul A17, Popescu I18, Schneeberger S19, Boillot O20, Nashan B21, Mikolajczyk RT2,3, Baumann U1.

Abstract

Liver transplantation (LTx) is a rescue therapy for life threatening complications of Wilson disease (WD). However, data on the outcome of WD patients post LTx are scarce. The aim of our study was to analyse a large pediatric WD cohort with the aim of investigating the long-term outcome of pediatric WD patients after LTx, and to identify predictive factors for patient and transplant survival. This is a retrospective cohort study using data of all children (<18 years) liver transplanted for WD enrolled in the European Liver Transplant Registry (ELTR) from 1968 until December 2013. In total, 338 patients (57.6% female), transplanted at 80 different European centers (1-26 patients per center) were included in this study. The median age at transplantation was 14 (IQR: 11-16) years; patients were followed up for a median of 5.4 (IQR: 1.0-10.9) years after LTx. Overall patient survival rates were high with 87% (1-year survival), 84% (5-year survival) and 81% (10-year survival); survival rates increased considerably with calendar year (p<0.001). Early age at LTx, living donation, and HTK preservation liquid were identified as risk factors for poor patient survival in the multivariable analysis. LTx is an excellent treatment option for pediatric patients with WD and associated end stage liver disease. Long-term outcome in these patients is similar to other pediatric causes for LTx. Overall patient and graft survival rates improved considerably over the last decades. To improve future research in the field, the vast variability of allocation strategies should be harmonised and a generally accepted definition or discrimination of acute versus chronic WD needs to be found. This article is protected by copyright. All rights reserved.