Liver transplantation for Wilson's disease

Area of Science:

• Hepatology
• Genetics
• Transplantation

Background:

• Wilson disease (WD) is a genetic disorder of copper metabolism.
• Standard treatments include copper chelation and zinc salts.
• Some WD patients have inadequate responses to medical therapy, necessitating alternative interventions.

Purpose of the Study:

• To evaluate the role and outcomes of liver transplantation in managing Wilson disease.
• To identify patient populations within WD who benefit most from liver transplantation.

Main Methods:

• Review of clinical data for WD patients undergoing liver transplantation.
• Analysis of indications for transplantation, including acute liver failure and end-stage liver disease.
• Assessment of post-transplantation outcomes and copper excretion.

Main Results:

• Liver transplantation is indicated for approximately 5% of WD patients presenting with acute liver failure or end-stage liver disease.
• Transplantation is most common in the second to fourth decades of life.
• Outcomes are excellent, regardless of donor type (cadaveric or living).

Conclusions:

• Liver transplantation is a highly effective treatment for select Wilson disease patients with severe hepatic manifestations.
• The procedure restores normal copper excretion and improves long-term survival.
• Excellent outcomes support liver transplantation as a definitive treatment option for medically refractory WD.