Second Department of Neurology, Institute of Psychiatry and Neurology, Warsaw, Poland; Department of Experimental and Clinical Pharmacology, Medical University, Warsaw, Poland.
Eur J Neurol. 2014 Apr;21(4):599-606. doi: 10.1111/ene.12348. Epub 2014 Jan 21.
To compare the course of treatment in patients with symptomatic Wilson's disease (WD) receiving either D-penicillamine (DPA) or zinc sulfate (ZS) as first-line therapy.
In all, 143 consecutive patients diagnosed with symptomatic WD from January 2005 to December 2009, followed until December 2010, were included. The decision about first-line therapy was made individually after discussion with the patient. Physicians had no clear preference of one drug over the other. Data were analyzed in subgroups with predominantly neurological (DPA, 35; ZS, 21) and hepatic (DPA, 36; ZS, 51) presentation of WD.
According to Kaplan-Meier analysis, neurological WD patients scheduled for DPA had a similar probability of not remaining on first-line therapy as patients receiving ZS (20% vs. 24% at the end of follow-up), with adjusted odds ratio (OR) of 0.9 (95% CI 0.2-3.5). In patients with hepatic WD, this probability was significantly higher for DPA (31% vs. 12%; adjusted OR 3.0, 95% CI 0.9-9.9), especially in the first 6 months. Early worsening occurred only in neurological WD patients, with no differences between both treatment groups (35% vs. 19%; OR 2.8, 95% CI 0.7-10.8). Neurological improvement and decrease of liver enzymes were achieved with similar frequency. Compliance with DPA was better in hepatic (97% vs. 80%) but not in neurological patients (91% vs. 81%). Drug adverse effects were more common on DPA (15% vs. 3%).
DPA and ZS are effective in the majority of WD patients. Neither therapy appears to be clearly superior. Therefore ZS may be considered a reasonable alternative to DPA as a first-line therapy.
比较接受 D-青霉胺（DPA）或硫酸锌（ZS）一线治疗的有症状威尔逊病（WD）患者的治疗过程。
共纳入 143 例 2005 年 1 月至 2009 年 12 月期间诊断为有症状 WD 的连续患者，随访至 2010 年 12 月。一线治疗决策在与患者讨论后个别做出。医生对一种药物没有明显的偏好。根据主要表现为神经（DPA，35；ZS，21）和肝脏（DPA，36；ZS，51）的 WD 患者进行数据亚组分析。
根据 Kaplan-Meier 分析，计划接受 DPA 的神经 WD 患者未继续接受一线治疗的可能性与接受 ZS 的患者相似（随访结束时分别为 20%和 24%），调整后的比值比（OR）为 0.9（95%CI 0.2-3.5）。在肝 WD 患者中，DPA 的可能性明显更高（31%对 12%；调整后的 OR 3.0，95%CI 0.9-9.9），尤其是在前 6 个月。仅在神经 WD 患者中出现早期恶化，两组之间无差异（35%对 19%；OR 2.8，95%CI 0.7-10.8）。神经改善和肝脏酶降低的频率相似。DPA 的肝 WD 患者的依从性较好（97%对 80%），但在神经 WD 患者中无差异（91%对 81%）。DPA 的药物不良反应更为常见（15%对 3%）。
DPA 和 ZS 对大多数 WD 患者有效。两种治疗方法均无明显优势。因此，ZS 可作为 DPA 的一线替代药物。
