BACKGROUND: 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 presence of neuropsychiatric involvement. This study aimed to examine the frequency of adult LT for WD in Italy, focusing on disease phenotype at time of LT. METHODS: A retrospective, observational, multicentric study was conducted across Italy exploring the frequency and characteristics of adults transplanted for WD between 2006-2016. RESULTS:Twenty-nine 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 (M/F 9/18; age at LT 29 [19-60] years; median MELD score at LT 27 [6-49]) were considered in this analysis. Isolated hepatic phenotype was the indication for LT in 17 cases (63%), while two patients (7%) underwent LT for neurological impairment on compensated liver disease. Overall, 1- and 5-yr patient survival was excellent (88% and 83%, respectively). Neuropsychiatric symptoms early after LT completely recovered only in few cases. CONCLUSIONS: WD remains an uncommon, unusual indication for LT in Italy, displaying good post-LT graft and patient survival. Since isolated neuropsychiatric involvement has represented a rare indication to LT, more data are needed to properly assess the value of LT for WD in this subset of patients.

Outcomes of liver transplant for adults with Wilson's disease

Ferrarese, Alberto;Toniutto, Pierluigi;Burra, Patrizia
2020

Abstract

BACKGROUND: 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 presence of neuropsychiatric involvement. This study aimed to examine the frequency of adult LT for WD in Italy, focusing on disease phenotype at time of LT. METHODS: A retrospective, observational, multicentric study was conducted across Italy exploring the frequency and characteristics of adults transplanted for WD between 2006-2016. RESULTS:Twenty-nine 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 (M/F 9/18; age at LT 29 [19-60] years; median MELD score at LT 27 [6-49]) were considered in this analysis. Isolated hepatic phenotype was the indication for LT in 17 cases (63%), while two patients (7%) underwent LT for neurological impairment on compensated liver disease. Overall, 1- and 5-yr patient survival was excellent (88% and 83%, respectively). Neuropsychiatric symptoms early after LT completely recovered only in few cases. CONCLUSIONS: WD remains an uncommon, unusual indication for LT in Italy, displaying good post-LT graft and patient survival. Since isolated neuropsychiatric involvement has represented a rare indication to LT, more data are needed to properly assess the value of LT for WD in this subset of patients.
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Utilizza questo identificativo per citare o creare un link a questo documento: http://hdl.handle.net/11577/3324743
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