Published online May 16, 2021. doi: 10.12998/wjcc.v9.i14.3273
Peer-review started: January 12, 2021
First decision: February 11, 2021
Revised: February 28, 2021
Accepted: March 17, 2021
Article in press: March 17, 2021
Published online: May 16, 2021
Acute liver failure (ALF) can be a primary presentation of Wilson disease (WD). Mortality rates are high in WD with ALF (WDALF). Predictions of mortality in WDALF vary by model and are sometimes contradictory, perhaps because few patients are studied or WD diagnoses are questionable.
To determine the outcomes among well-documented WDALF patients and assess mortality model performance in this cohort.
We reviewed the medical records of our pediatric WDALF patients (n = 41 over 6-years-old, single-center retrospective study) and compared seven prognostic models (King’s College Hospital Criteria, model for end-stage liver disea
Among the 41 Han Chinese patients with ALF, WD was established by demonstrating ATP7B variants in 36. In 5 others, Kayser-Fleischer rings and Coombs-negative hemolytic anemia permitted diagnosis. Three died during hospitalization and three underwent liver transplantation (LT) within 1 mo of presentation and survived (7.3% each); 35 (85.4%) survived without LT when given enteral D-penicillamine and zinc-salt therapy with or without urgent plasmapheresis. Parameters significantly correlated with mortality included encephalopathy, coagulopathy, and gamma-glutamyl transpeptidase activity, bilirubin, ammonia, and serum sodium levels. Area under the receiver operating curves varied among seven prognostic models from 0.981 to 0.748 with positive predictive values from 0.214 to 0.429.
WDALF children can survive and recover without LT when given D-peni
Core Tip: This study determined outcomes among 41 recently seen pediatric patients with well-documented Wilson disease (WD) with acute liver failure (ALF) (WDALF) (most confirmed genetically, WD presenting as ALF) and assessed mortality model performances. Mortality was low, showing that WDALF children, including those with milder encephalopathy, can survive and recover without liver transplant (LT) when given D-penicillamine and zinc-salt therapy with or without plasmapheresis. Sensiti