| Autor: |
Peter Ott, Thomas Sandahl, Aftab Ala, David Cassiman, Eduardo Couchonnal-Bedoya, Rubens Gisbert Cury, Anna Czlonkowska, Gerald Denk, Renata D’Inca, Francisco de Assis Aquino Gondim, Joanna Moore, Aurelia Poujois, Carlos Alexandre Twardowschy, Karl Heinz Weiss, Massimo Zuin, C.Omar F. Kamlin, Michael L. Schilsky |
| Jazyk: |
angličtina |
| Rok vydání: |
2024 |
| Předmět: |
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| Zdroj: |
JHEP Reports, Vol 6, Iss 8, Pp 101115- (2024) |
| Druh dokumentu: |
article |
| ISSN: |
2589-5559 |
| DOI: |
10.1016/j.jhepr.2024.101115 |
| Popis: |
Background & Aims: Wilson disease (WD) is caused by accumulation of copper primarily in the liver and brain. During maintenance therapy of WD with D-penicillamine, current guidelines recommend on-treatment ranges of urinary copper excretion (UCE) of 200-500 μg/24 h and serum non-ceruloplasmin-bound copper (NCC) of 50-150 μg/L. We compared NCC (measured by two novel assays) and UCE from patients with clinically stable WD on D-penicillamine therapy with these recommendations. Methods: This is a secondary analysis of data from the Chelate trial (NCT03539952) that enrolled physician-selected patients with clinically stable WD on D-penicillamine maintenance therapy (at an unaltered dose for at least 4 months). We analyzed laboratory samples from the first screening visit, prior to interventions. NCC was measured by either protein speciation (NCC-Sp) using anion exchange high-performance liquid chromatography protein speciation followed by copper determination with inductively coupled plasma mass spectroscopy or as exchangeable copper (NCC-Ex). NCC-Sp was also analyzed in healthy controls (n = 75). Results: In 76 patients with WD with 21.3±14.3 average treatment-years, NCC-Sp (mean±SD: 56.6±26.2 μg/L) and NCC-Ex (mean±SD: 57.9±24.7 μg/L) were within the 50-150 μg/L target in 61% and 54% of patients, respectively. In addition, 36% and 31%, respectively, were even below the normal ranges (NCC-Sp: 46-213 μg/L, NCC-Ex: 41-71 μg/L). NCC-Ex positively correlated with NCC-Sp (r2 = 0.66, p |
| Databáze: |
Directory of Open Access Journals |
| Externí odkaz: |
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