TRISTAN – a Secondary Use of Data (SUD), retrospective real world data acquisition for therapy management of Wilson Disease Patients in Germany

Zeitschrift Fur Gastroenterologie(2023)

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摘要
Introduction Wilson disease (WD) is a rare autosomal recessively inherited disorder of copper metabolism with pathological copper accumulation mainly in the liver and brain. Chelators are mainly used as treatment for WD. In Germany D-penicillamine is approved as first-line treatment and Trientine (Trientine dihydrochloride (TETA-2HCl) and Trientine tetrahydrochloride (TETA-4HCL)), is available for patients who are intolerant to D-penicillamine. Data on therapy management of patients, specifically with respect to treatment switch and dosing, is rare.
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关键词
wilson disease patients,world data,therapy management
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