Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

Multicentre, Retrospective and Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months.

A retrospective study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them prospectively for a further 12 months.

Inclusion Criteria:

• Patients aged 1 year to 90 years of age.
• Physician established diagnosis of Wilson disease based on a Ferenci score > 3.
• Documented treatment with d-Penicillamine, withdrawal of treatment with d- Penicillamine, followed by treatment with trientine for at least 6 months at date of informed consent.
• Able/willing to provide written informed consent.
• For enrolment in the prospective part, enrolment in the retrospective part of the study is required.

Exclusion Criteria:

• Incomplete history of medication use for trientine from initial diagnosis to latest follow up.
• Unavailable outcome data for hepatic and neurological course of disease at assessment time points.
• Patients with acute liver failure and fulminant hepatic disease with fatal outcome.
• Hypersensitivity to trientine and severe anaemia.