EASL Clinical Practice Guidelines

Prognosis

Untreated Wilson's disease is universally fatal, with most patients dying from liver disease and a minority from complications of progressive neurologic disease. With chelation treatment and liver transplantation, prolonged survival has become the norm [[27], [38], [39]], although mortality has not been assessed prospectively. In general, prognosis for survival depends on the severity of liver and neurological disease and compliance with drug treatment. Liver function becomes normal over 1–2 years of treatment in most patients with no or compensated cirrhosis at presentation, and then remains stable without progressive liver disease with adherence to treatment. At the other end of the spectrum, medical therapy is rarely effective in patients presenting with acute liver failure due to Wilson's disease, mainly due to the time required to remove toxic copper from the organism. A prognostic index has been developed [40], and later modified by Dhawan et al. [41]. A score greater than 11 is always fatal without liver transplantation (Table 3). Patients presenting with neurologic symptoms fare better with respect to life expectancy, especially if liver disease is limited. However, neurologic symptoms appear to be only partly reversible with treatment and may even worsen following initiation of treatment.

Table 3
Prognostic index in Wilson's disease [40], modified by Dhawan et al. [41].



*= score points, upper limit of normal for AST = 20 IU/ml (at King's College). A score ⩾11 is associated with high probability of death without liver transplantation.

In patients undergoing orthotopic liver transplantation, survival may be slightly reduced early on, but appears normal (for transplant population) thereafter [42].