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

• Verfasst von: Weiss, Karl Heinz [VerfasserIn]
• Verfasst von: Thurik, Florentine [VerfasserIn]
• Verfasst von: Gotthardt, Daniel [VerfasserIn]
• Verfasst von: Schäfer, Mark [VerfasserIn]
• Verfasst von: Teufel-Schäfer, Ulrike [VerfasserIn]
• Verfasst von: Wiegand, Franziska [VerfasserIn]
• Verfasst von: Merle, Uta [VerfasserIn]
• Verfasst von: Ferenci-Foerster, Daniela [VerfasserIn]
• Verfasst von: Maieron, Andreas [VerfasserIn]
• Verfasst von: Stauber, Rudolf [VerfasserIn]
• Verfasst von: Zoller, Heinz [VerfasserIn]
• Verfasst von: Schmidt, Hartmut H. [VerfasserIn]
• Verfasst von: Reuner, Ulrike [VerfasserIn]
• Verfasst von: Hefter, Harald [VerfasserIn]
• Verfasst von: Trocello, Jean Marc [VerfasserIn]
• Verfasst von: Houwen, Roderick H. J. [VerfasserIn]
• Verfasst von: Ferenci, Peter [VerfasserIn]
• Verfasst von: Stremmel, Wolfgang [VerfasserIn]
• Titel: Efficacy and safety of oral chelators in treatment of patients with Wilson disease
• Verf.angabe: Karl Heinz Weiss, Florentine Thurik, Daniel Nils Gotthardt, Mark Schäfer, Ulrike Teufel, Franziska Wiegand, Uta Merle, Daniela Ferenci-Foerster, Andreas Maieron, Rudolf Stauber, Heinz Zoller, Hartmut H. Schmidt, Ulrike Reuner, Harald Hefter, Jean Marc Trocello, Roderick H. J. Houwen, Peter Ferenci, Wolfgang Stremmel
• E-Jahr: 2013
• Jahr: 28 March 2013
• Umfang: 10 S.
• Fussnoten: Gesehen am 28.10.2021
• Titel Quelle: Enthalten in: Clinical gastroenterology and hepatology
• Ort Quelle: New York, NY : Elsevier Science, 2003
• Jahr Quelle: 2013
• Band/Heft Quelle: 11(2013), 8, Seite 1028-1035.e2
• ISSN Quelle: 1542-7714
• DOI: doi:10.1016/j.cgh.2013.03.012
• Datenträger: Online-Ressource
• Sprache: eng
• Sach-SW: ATP7B
• Sach-SW: Metabolic Disorder
• Sach-SW: Wilson's Disease
• Sach-SW: Wilsons disease
• K10plus-PPN: 177566757X

Abstract

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.