Therapeutic Management of Wilson Disease in Clinical Practice: Multicenter Retrospective Real-World Data from Germany

Wilson disease (WD) is a rare disorder of copper metabolism. Chelating agents available include D-penicillamine (DPA) and trientine (TETA-2HCl and TETA-4HCl). Real-world data on treatment sequencing and formulation switches are limited. This study aimed to retrospectively analyze real-world treatment patterns in WD patients receiving trientine therapy in Germany.

We retrospectively collected data from patients treated with trientine at six specialized centers between 01/2012 and 12/2021. Inclusion criteria were documented initiation of trientine and at least one follow-up visit. We analyzed treatment duration, number of treatment lines, and dosing during switches. Reasons for switching therapy and clinical outcome data were not systematically collected.

A total of 143 patients were included. Monitoring across centers involved non-ceruloplasmin-bound copper, 24-hour urinary copper excretion, and transient elastography. Data on prior DPA therapy were available for 109 patients (mean duration: 11 years). Fifty percent were switched to trientine within 7 years of DPA therapy, while the remaining switches occurred after up to 48 years. Patients received a mean of 3.3 lines of therapy (range: 1-6). Mean switch factors were 0.75 for DPA to TETA-2HCl and 0.6 for DPA to TETA-4HCl. For switching between TETA-2HCl and TETA-4HCl, factors were 0.84 and 1.27, respectively.

These real-world data describe WD treatment management at specialized German centers. The observed dose changes during formulation switches are descriptive and hypothesis-generating, but prospective studies with clinical outcome data are needed before any switch factor can be recommended for routine practice.