Wilson’s disease: a review of treatment options with a focus on zinc therapy

J Hui,¹ NL Tang<sup>2

1</sup>Department of Pediatrics, ²Department of Chemical Pathology, Prince of Wales Hospital, The Chinese University of Hong Kong, Shatin, Hong Kong

Abstract: Wilson’s disease, an inherited disorder of copper metabolism, is well known for its remarkable heterogeneous clinical presentation and variable organ involvement. Since its first introduction almost five decades ago, zinc – with its relative lack of side effects and safety profile – has gained a lot of attention and favor over other therapeutic agents for the treatment of Wilson’s disease. Traditional chelating agents like D-penicillamine and trientine, although proven to be efficacious, compared less favorably to zinc because of their long list of potential side effects. The medical literature regarding the use of zinc generally supports its primary role in the treatment of presymptomatic/asymptomatic, pediatric, and pregnant patients. However, for symptomatic patients – especially the symptomatic hepatic patients, long-term follow-up studies concluded that zinc is less effective than chelating agents in preventing hepatic deterioration. Other treatment concepts like combination therapy using zinc in conjunction with a chelating agent or sequential treatment with initial chelating agents followed by zinc have been tried with variable success. Rigorously designed studies and safety data on these newer treatment concepts are not yet available. Independent of the chosen medical regimen, nonadherence or discontinuation with noncompliance is the biggest threat adversely affecting the long-term outcome in all treated patients with Wilson’s disease. Close monitoring as well as long-term follow-up for the development of new neurologic or hepatic symptoms are essential for all patients, irrespective of how long they have been on treatment and whether treatment has remained static for years. In this paper, the authors will discuss the current opinion and role of zinc in the treatment of various manifestations and different stages of Wilson’s disease.

Keywords: Wilson’s disease, treatment, zinc