We have treated 9 patients who presented with hepatic decompensation resulting from
Wilson's disease with a combination of
trientine and
zinc, generally for at least 4 months, followed by transition to
zinc maintenance
therapy. All of these patients had
hypoalbuminemia, all but 1 had
hyperbilirubinemia, and 7 had
ascites. All of these patients would have been candidates for
liver transplantation on the basis of their initial Child-Turcotte-Pugh (
CTP) scores. The minimal listing criteria for transplant candidates is a score greater than 7. Eight of the 9 patients had demonstrated a
CTP score of 10 or higher. The other scoring system that has been used in
Wilson's disease to determine need for
transplantation is the prognostic index of Nazer, in which a score over 6 indicates that the patient is unlikely to survive without a transplant if treated with
penicillamine. Two of our patients had Nazer scores higher than 6. With our medical
therapy, all 9 of these patients have recovered normal liver function as reflected by normalization of their
CTP scores to 5. Because of coexisting neurologic disease, 1 of our 9 patients was initiated on a neurologic protocol and by chance randomized to receive
tetrathiomolybdate (TM) and
zinc after 2 weeks of
trientine/
zinc treatment. This patient's liver function recovered much more rapidly than did that of the other 8 patients, all of whom were treated with
trientine/
zinc, suggesting that TM
therapy offers a further advantage. In summary, we were able to take 9 patients who presented with
liver failure -8 of whom had
CTP scores indicating a potential need for
liver transplantation and 2 of whom had Nazer prognostic scores indicating that they were not likely to survive if treated only with
penicillamine - and treat them medically, with recovery in all 9. We believe the
trientine/
zinc combination
therapy should be the standard for initial treatment of
liver failure in
Wilson's disease because its efficacy is equal or slightly superior to that of
penicillamine and because it has a much lower incidence of side effects. Moreover, TM warrants study to determine whether
therapy for hepatic
Wilson's disease can be further improved.