Liver transplantation in Wilson's disease: are its indications established?

The indications of progressive neurological deterioration despite no hepatic insufficiency, for liver transplant (OLT) in patients with Wilson's disease (WD) who do not improve with medical treatment is widely debated. The aim of this paper was to present our OLT experience in WD.

Fourteen patients were given a transplant after the diagnosis of WD for the following indications: in four patients, a steady neurological deterioration that failed to respond to chelation treatment (all with Child grade A liver function); in nine patients, Child grade C hepatic insufficiency, in whom medical treatment had failed (one of these patients also presented with severe neurological alterations); and in one patient, acute hepatic failure secondary to E. Coli infection of the ascitic fluid.

Two patients died, one due to severe pancreatitis in the immediate postoperative period and the other, who was transplanted for neurological involvement, experienced an acute rejection episode treated with methylprednisolone in the first postoperative month and, in the 4th month, another episode of acute rejection, failed to respond to corticoids and required OKT-3 administration. She subsequently developed bilateral bronchopneumonia due to cytomegalovirus that led to her death. During the immediate postoperative period all of the cupremia, cupriuria, and ceruloplasmin levels returned to normal. The liver function in the 12 patients currently alive was totally normal after a follow-up of 8 years (range, 1-15 years) with actuarial 5-year survival of 85.7%.

These cases demonstrated that OLT may mobilize copper sufficiently from the central nervous system to correct severe neurological deficits, a result that medical therapy alone cannot achieve. We suggest that OLT be considered for patients with WD who have crippling neurological and psychological diseases, even if liver function is stable.