Department of Internal Medicine, University Hospital Gasthuisberg, University of Leuven, Leuven, Belgium.
Eur J Gastroenterol Hepatol. 2010 May;22(5):564-71. doi: 10.1097/MEG.0b013e3283353df8.
Detailed data on long-term effectiveness of various drug therapies in Wilson's disease (WD) are lacking. Therefore, we retrospectively reviewed our patient cohort treated with D-penicillamine.
This study reports on the clinical presentation, the diagnostic evaluation, and the disease course in 24 WD patients treated long-term (15+/-12 years, between 1969 and 2009) with D-penicillamine.
The overall survival in our cohort was 91.6%. Twenty-two of 24 patients had liver disease at presentation, 17 of 24 patients (71%) had cirrhosis, 11 of whom had complications of cirrhosis. Six of 11 of these patients showed hepatological improvement (five of six) or stabilization (one of six), three of 11 were transplanted, one of 11 died, one of 11 discontinued follow-up. In the six of 17 cirrhotic patients without complications, improvement (four of six) or stabilization (two of six) occurred. Of all other patients (seven of 24), five of seven showed improvement (three of five) or stabilization (two of five), hepatological deterioration occurred only in one patient due to poor therapy compliance and one of seven discontinued follow-up. Neuropsychiatric symptoms were present in 13 of 24 at presentation and resolved in one of 13, decreased in seven of 13, stabilized in four of 13 and worsened in one of 13 patients (due to poor compliance). In general, we observed a favorable hepatological and neurological evolution with D-penicillamine.
Despite the presence of liver disease or neuropsychiatric symptoms at baseline in all but one of the patients, we report beneficial results on liver and neurological disease after very long-term treatment with D-penicillamine, thereby adding to its reputation as 'first-line' therapy in WD.
Wilson 病（WD）各种药物治疗的长期疗效的详细数据缺乏。因此，我们回顾性地复习了用 D-青霉胺治疗的患者队列。
本研究报告了 24 例 WD 患者的临床表现、诊断评估和疾病过程，这些患者长期（1969 年至 2009 年之间 15+/-12 年）用 D-青霉胺治疗。
我们队列的总生存率为 91.6%。24 例患者中有 22 例在就诊时患有肝病，17 例（71%）有肝硬化，其中 11 例有肝硬化并发症。这 11 例患者中有 6 例（6/11）显示肝生化改善（5/6）或稳定（1/6），3 例（3/11）接受肝移植，1 例（1/11）死亡，1 例（1/11）失访。在 11 例无并发症的肝硬化患者中，6 例（6/17）改善（4/6）或稳定（2/6）。在其余的所有患者（24 例中的 7 例）中，5 例（5/7）显示改善（3/5）或稳定（2/5），1 例（1/7）肝生化恶化，因为治疗不依从，1 例（1/7）失访。13 例患者在就诊时存在神经精神症状，1 例（13/13）缓解，7 例（13/13）减轻，4 例（13/13）稳定，1 例（13/13）恶化（因治疗不依从）。总的来说，我们观察到用 D-青霉胺治疗后，患者的肝和神经疾病有良好的演变。
尽管除了 1 例患者以外，所有患者在基线时都存在肝病或神经精神症状，但我们报告了长期用 D-青霉胺治疗后肝和神经疾病的有益结果，从而增加了 D-青霉胺在 WD 中的“一线”治疗地位。
