Maintenance therapy simplification using a single daily dose: A preliminary real-life feasibility study in patients with Wilson disease - 05/11/22
, France Woimant c, Eduardo Couchonnal a, Jérôme Dumortier a, d, Chloe Laurencin a, Laurence Lion-François a, Abdelouahed Belmalih a, Muriel Bost a, Erwan Morvan e, Nouzha Oussedik-Djebrani c, Alain Lachaux a, d, Aurélia Poujois eHighlights |
• | Wilson disease is treatable with multiple daily doses and lifelong treatment. |
• | Multiple daily doses can be an obstacle for adherence and treatment efficacy. |
• | In our study, a single daily dose is effective in most of stabilized WD patients. |
• | SDD could represent a very interesting option to consider in some WD patients. |
• | Our results encourage to set up a validation in a prospective study. |
Abstract |
Background |
Single daily dose (SDD) is a good way to improve adherence by simplifying treatment. Efficacy data concerning patients with Wilson disease (WD) taking an SDD are lacking.
Aim |
To report the effectiveness of the use of SDD for the treatment of WD.
Methods |
This retrospective study included WD patients followed in the French National Network who received an SDD in maintenance phase. The treatment failure was defined as a composite criterion with the occurrence of at least one of the following criterion: death, transplantation, increase of transaminases >2xULN, hepatic decompensation, neurological aggravation, severe side effects related to treatment, and/or discontinuation of treatment.
Results |
A total of 26 patients received an SDD (D-penicillamine=13, trientine=8, zinc=5) after a median interval of 152 months after diagnosis. After one year, two patients had treatment failure: transaminitis in one, continuation of neurological deterioration in the other related to a poor compliance. After a median duration of 41 months on SDD, 3 other patients had treatment failure (transaminitis=2, treatment discontinuation=1). There was no death, no liver transplantation, no hepatic decompensation, and no severe side effects related to treatment during the follow-up. Moreover, transaminases and serum exchangeable copper were not significantly different 1 year post-switch and at last follow-up compared to baseline.
Conclusions |
Maintenance therapy simplification through the use of an SDD could be considered in some WD patients. In this pilot study, SDD was effective in 21/26 patients (81%) without any concern regarding safety.
Le texte complet de cet article est disponible en PDF.Keywords : Wilson disease, Wilson's disease, Single daily dose, Adherence, Compliance, Persistence, Long-term care
Abbreviations : Wilson disease, single daily dose, upper limit of normal, Unified Wilson's Disease Rating Scale, Aspartate aminotransferase, Alanine aminotransferase, gammaglutamyl transferase, alkaline phosphatase, serum exchangeable copper, liver tests, Kayser-Fleischer, relative exchangeable copper
Plan
Vol 46 - N° 9
Article 101978- novembre 2022 Retour au numéro
Article précédent
- Prevalence and characteristics of cystic fibrosis liver disease: a study highlighting the lack of histological diagnosis
- Zaina Issa, Sophie Gohy, Francis Zech, Pamela Baldin, Bénédicte Delire, Géraldine Dahlqvist
- Prophylactic sequential antibiotic therapy for recurrent liver/biliary sepsis
- Jérôme Dumortier, Olivier Guillaud, Pierre-Jean Valette, Christian Partensky, Pierre Paliard, Olivier Boillot, Domitille Erard
Bienvenue sur EM-consulte, la référence des professionnels de santé.
L’accès au texte intégral de cet article nécessite un abonnement.
Déjà abonné à cette revue ?