Objectives: Liver transplantation (LT) in Wilson disease (WD) is a life-saving option for patients presenting with liver failure and encephalopathy. Patients without encephalopathy can avoid LT and treated successfully with chelation therapy. It is essential to predict the risk of fatal outcomes where LT is required. We aim to critically analyse the validity of the WD Index prospectively from a cohort of WD patients managed at our institution.
Methods: WD Index and other clinical data from 2005 to 2018, recorded prospectively as part of clinical management, were analysed.
Results: Over 13-year period, 52 children with WD (29 boys) with median age at diagnosis of 11.69 (range 3.92-17.26) years were studied. Of these, 17 were diagnosed as part of family screening, 17 presented with abnormal liver enzyme, and 18 with acute hepatic decompensation (AHD) as per PALF definition. Patients presented with abnormal liver enzyme and in the pre-symptomatic group had WD Index <11, and none of them required LT. WD Index is still a good predictor for LT in WD patients with AHD, providing a sensitivity of 80%, specificity of 100%, positive-predictive, and negative-predictive value of 100% and 80%, respectively. Patients presented with an index of 8-10 also required LT at median duration of 58 days (IQR 48-135 days).
Conclusions: WD patients presenting with AHD who had an index of ≥11 do require LT. Children with a WD Index of 8 to 10 within the first 2 months of admission require close monitoring as LT may become necessary.
Copyright © 2021 by European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition.