Resolution of decompensated cirrhosis from Wilson's disease with zinc monotherapy: a potential therapeutic option?

BACKGROUND & AIMS: Wilson's disease is a genetic autosomal-recessive copper deposition disorder often presenting with neurologic or hepatic symptoms. In cases of hepatic presentation, treatment usually is initiated with potentially toxic copper chelators, such as D-penicillamine or trientine. Although multiple studies have introduced zinc as a low-toxicity and low-cost Wilson's disease treatment, its use has been limited to adjunctive or single-agent maintenance options. In this report, we describe the use of zinc monotherapy in a patient with severe hepatic presentation of Wilson's disease. METHODS: Zinc has not been evaluated as a single-agent treatment option for active hepatic Wilson's disease. Zinc monotherapy was initiated for a single patient with fulminant hepatic failure caused by Wilson's disease while awaiting liver transplantation. RESULTS: Over a 1-year period with zinc monotherapy, this patient experienced normalization of hepatic synthetic function and resolution of hypoalbuminemia and coagulopathy. Clinical stabilization of variceal bleeds, ascites, and lower-extremity edema also were observed. The patient is no longer a transplant candidate as a result of clinical recovery and improvement of Model for End-stage Liver Disease and Child-Turcotte-Pugh scores. CONCLUSIONS: This case highlights the potential use of zinc as a low-toxicity and low-cost single-agent treatment in severely decompensated hepatic Wilson's disease. Despite promising results in this case, further clinical evaluation will be necessary to assess fully the clinical efficacy of zinc monotherapy.

Duke Scholars

Author Carl Berg Medicine, Gastroenterology