Efficacy and safety of oral chelators in treatment of patients with Wilson disease.

Weiss, Karl Heinz, Thurik, Florentine, Gotthardt, Daniel Nils, Schäfer, Mark, Teufel, Ulrike, Wiegand, Franziska, Merle, Uta, Ferenci-Foerster, Daniela, Maieron, Andreas, Stauber, Rudolf, Zoller, Heinz, Schmidt, Hartmut H, Reuner, Ulrike, Hefter, Harald, Trocello, Jean Marc, Houwen, Roderick H J, Ferenci, Peter and Stremmel, Wolfgang (2013) Efficacy and safety of oral chelators in treatment of patients with Wilson disease. Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association, 11 (8). 1028-35.e1-2. ISSN 1542-7714

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BACKGROUND & AIMS Wilson disease is a genetic copper storage disorder that causes hepatic and neurologic symptoms. Chelating agents (D-penicillamine, trientine) are used as first-line therapies for symptomatic patients, but there are few data from large cohorts. We assessed the safety of D-penicillamine and trientine therapy and outcomes of patients with Wilson disease. METHODS We performed a retrospective analysis of data on 380 patients with Wilson disease from tertiary care centers in Germany and Austria, and 25 additional patients from the EUROWILSON registry. Chelator-based treatment regimens were analyzed for their effect on neurologic and hepatic symptoms and for adverse events that led to discontinuation of therapy (Kaplan-Meier estimation; data were collected for a mean of 13.3 y after therapy began). RESULTS Changes in medication were common, resulting in analysis of 471 chelator monotherapies (326 patients receiving D-penicillamine and 141 receiving trientine). Nine of 326 patients treated with D-penicillamine and 3 of 141 patients given trientine underwent liver transplantation. Adverse events leading to discontinuation of treatment were more frequent among those receiving D-penicillamine than trientine (P = .039). Forty-eight months after therapy, hepatic deterioration was reported in only 4 of 333 patients treated initially with a chelating agent. Hepatic improvements were observed in more than 90%, and neurologic improvements were observed in more than 55%, of therapy-naive patients, and values did not differ significantly between treatments. However, neurologic deterioration was observed less frequently in patients given D-penicillamine first (6 of 295) than those given trientine first (4 of 38; P = .018). CONCLUSIONS Chelating agents are effective therapies for most patients with Wilson disease; D-penicillamine and trientine produce comparable outcomes, although D-penicillamine had a higher rate of adverse events. Few patients receiving chelation therapy had neurologic deterioration, which occurred more frequently in patients who received trientine.
Item Type: Article
Divisions: Ordensklinikum Linz Barmherzige Schwestern > Interne IV: Gastroenterologie & Hepatologie, Endokrinologie und Stoffwechsel, Ernährungsmedizin
Depositing User: Prof. Dr. Rainer Schöfl
Date Deposited: 16 Nov 2021 11:40
Last Modified: 16 Nov 2021 11:40

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