Outcomes of Liver Transplant for Adults With Wilson’s Disease

Abstract

Wilson’s disease (WD) is a rare genetic disorder with protean manifestations. Even if liver transplantation (LT) could represent an effective therapeutic option for patients with end‐stage liver disease, it has remained controversial in the presence of neuropsychiatric involvement. This study aimed to examine the frequency of adult LT for WD in Italy, focusing on the disease phenotype at the time of LT. A retrospective, observational, multicenter study was conducted across Italy exploring the frequency and characteristics of adults transplanted for WD between 2006 and 2016. A total of 29 adult WD patients underwent LT during the study period at 11 Italian LT centers (accounting for 0.4% of all LTs performed), and 27 of them were considered in this analysis (male/female, n = 9/18; age at LT, 29 years [19‐60 years]; median Model for End‐Stage Liver Disease score at LT, 27 [6‐49]). Isolated hepatic phenotype was the indication for LT in 17 (63%) patients, whereas 2 (7%) patients underwent LT for neurological impairment on compensated liver disease. Overall 1‐ and 5‐year patient survival was excellent (88% and 83%, respectively). Neuropsychiatric symptoms early after LT completely recovered in only a few patients. In conclusion, WD remains an uncommon, unusual indication for LT in Italy, displaying good post‐LT graft and patient survival. Because isolated neuropsychiatric involvement represents a rare indication to LT, more data are needed to properly assess the value of LT for WD in this subset of patients.

Liver Transplantation in Adults With Wilson’s Disease for the Neuropsychiatric Phenotype: Are We There Yet?

“…(5) With a median follow-up of 6 years, overall 1-and 5-year patient survivals were 88% and 83%, respectively. (5) These observations are consistent with other reports of 79%-88% and 75%-86%, respectively. (6,7) LT outcomes are reported to be better for decompensated cirrhosis compared with ALF.…”

“…The authors used the validated Unified Wilson's Disease Rating Scale to examine the response on neuropsychiatric features. (5) Of the 2 patients receiving LT for the isolated neuropsychiatric phenotype, the posttransplant course of a female patient with severe ataxia, dystonia, and tremor was notable for prolonged hospitalization with multiple complications. In the second case, a bedbound male patient with severe dystonia and tremor had only modest improvement in neurological symptoms.…”

“…Of the 10 patients with neuropsychiatric features (6 with severe neurological and 4 with severe psychiatric manifestations), posttransplant patient survival was similar to the hepatic phenotype. (5) However, LT recipients for the mixed hepatic and neuropsychiatric phenotype experienced more frequent complications (75% versus 59%), especially due to infections. LT also did not provide a benefit in improving the neuropsychiatric symptoms or signs.…”

“…These data are consistent with other reports on the lack of benefit of LT on neuropsychiatric features. (5) Some studies have also reported paradoxical neurological deterioration after LT for WD with neuropsychiatric manifestations. (3,4) The authors of the study suggest that the lack of improvement of neuropsychiatric symptoms could be due to insufficient follow-up after LT. (5) In the current study, WD accounted for 0.4% of all LTs in Italy, (5) a frequency lower than that reported by others.…”

“…(5) Some studies have also reported paradoxical neurological deterioration after LT for WD with neuropsychiatric manifestations. (3,4) The authors of the study suggest that the lack of improvement of neuropsychiatric symptoms could be due to insufficient follow-up after LT. (5) In the current study, WD accounted for 0.4% of all LTs in Italy, (5) a frequency lower than that reported by others. (6,7) For example, in 2 other European studies, WD was implicated in 1.0%-1.2% of all LTs.…”