Hospices Civils de Lyon, Hôpital Femme-Mère-Enfant, Centre National de Référence Pour la Maladie de Wilson, Bron, Hôpital Edouard Herriot, Lyon Cedex 03 69437, France; Ramsay Générale de Santé, Clinique de la Sauvegarde, Lyon, France.
APHP, Hôpital Lariboisière, Centre National de Référence Pour la Maladie de Wilson, Paris, France.
Clin Res Hepatol Gastroenterol. 2022 Nov;46(9):101978. doi: 10.1016/j.clinre.2022.101978. Epub 2022 Jun 14.
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.
To report the effectiveness of the use of SDD for the treatment of WD.
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.
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.
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.
每日单次剂量（SDD）是通过简化治疗来提高依从性的一种好方法。缺乏关于接受 SDD 的 Wilson 病（WD）患者的疗效数据。
报告使用 SDD 治疗 WD 的效果。
这项回顾性研究纳入了在法国国家网络中接受 SDD 维持治疗的 WD 患者。治疗失败定义为出现以下至少一个标准的复合标准：死亡、移植、转氨酶升高>2xULN、肝失代偿、神经恶化、与治疗相关的严重副作用、和/或治疗中断。
共有 26 名患者在诊断后中位数 152 个月后接受了 SDD（D-青霉胺=13，曲恩汀=8，锌=5）。一年后，有两名患者发生治疗失败：一名发生转氨酶升高，另一名因治疗依从性差而继续神经恶化。在接受 SDD 中位数 41 个月后，另外 3 名患者发生治疗失败（转氨酶升高=2，治疗中断=1）。在随访期间，无死亡、无肝移植、无肝失代偿、无与治疗相关的严重副作用。此外，与基线相比，转换后 1 年和最后一次随访时，转氨酶和血清可交换铜均无显著差异。
通过使用 SDD 简化维持治疗，可以考虑在一些 WD 患者中使用。在这项初步研究中，SDD 在 26 名患者中的 21 名（81%）有效，且无安全性问题。
