Treatment of Wilson's disease with zinc. XVIII. Initial treatment of the hepatic decompensation presentation with trientine and zinc
Treatment of Wilson's disease with zinc. XVIII. Initial treatment of the hepatic decompensation presentation with trientine and zinc
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DOI：
10.1016/s0022-2143(03)00157-4
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发表时间：
2003-12-01
期刊：
影响因子：
--
通讯作者：
Brewer, GJ
中科院分区：
文献类型：
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作者：
Askari, FK;Greenson, J;Brewer, GJ
我们已经用曲恩汀和锌的组合治疗了9例因威尔逊病引起的肝功能失代偿的患者，通常至少4个月，然后过渡到锌维持治疗。所有这些患者都有低白蛋白血症，除了我之外，所有人都有高胆红素血症，7人有腹水。所有这些患者根据其初始Child-Turcoffe- Pugh（CTP）评分都是肝移植的候选人。移植候选人的最低列名标准是得分大于7。9例患者中有8例显示CTP评分为10或更高。另一种用于威尔逊病的评分系统是Nazer预后指数，其中评分超过6表示如果用青霉胺治疗，患者不可能在没有移植的情况下存活。我们的两名患者的Nazer评分高于6分。通过我们的药物治疗，所有9名患者的肝功能恢复正常，如CTP评分正常化至5所反映的。由于共存的神经系统疾病，我们的9例患者中的1例开始接受神经系统方案，并在曲恩汀/锌治疗2周后随机接受四硫代钼酸盐（TM）和锌。该患者的肝功能恢复比其他8例患者快得多，所有患者均接受曲恩汀/锌治疗，表明TM治疗提供了进一步的优势。总之，我们能够收治9名出现肝衰竭的患者-其中8名CTP评分表明可能需要肝移植，其中2名Nazer预后评分表明如果仅接受青霉胺治疗，他们不太可能生存-并对他们进行药物治疗，所有9名患者均康复。我们认为曲恩汀/锌联合治疗应作为Wilson病肝衰竭初始治疗的标准，因为其疗效与青霉胺相当或略上级青霉胺，而且副作用发生率低得多。此外，TM值得研究，以确定肝Wilson病的治疗是否可以进一步改善。
We have treated 9 patients who presented with hepatic decompensation resulting from Wilson's disease with a combination of trientine and zinc, generally for at least 4 months, followed by transition to zinc maintenance therapy. All of these patients had hypoalbuminemia, all but I had hyperbilirubinemia, and 7 had ascites. All of these patients would have been candidates for liver transplantation on the basis of their initial Child-Turcoffe- Pugh (CTP) scores. The minimal listing criteria for transplant candidates is a score greater than 7. Eight of the 9 patients had demonstrated a CTP score of 10 or higher. The other scoring system that has been used in Wilson's disease to determine need for transplantation is the prognostic index of Nazer, in which a score over 6 indicates that the patient is unlikely to survive without a transplant if treated with penicillamine. Two of our patients had Nazer scores higher than 6. With our medical therapy, all 9 of these patients have recovered normal liver function as reflected by normalization of their CTP scores to 5. Because of coexisting neurologic disease, I of our 9 patients was initiated on a neurologic protocol and by chance randomized to receive tetrathiomolybdate (TM) and zinc after 2 weeks of trientine/zinc treatment. This patient's liver function recovered much more rapidly than did that of the other 8 patients, all of whom were treated with trientine/zinc, suggesting that TM therapy offers a further advantage. In summary, we were able to take 9 patients who presented with liver failure -8 of whom had CTP scores indicating a potential need for liver transplantation and 2 of whom had Nazer prognostic scores indicating that they were not likely to survive if treated only with penicillamine - and treat them medically, with recovery in all 9. We believe the trientine/zinc combination therapy should be the standard for initial treatment of liver failure in Wilson's disease because its efficacy is equal or slightly superior to that of penicillamine and because it has a much lower incidence of side effects. Moreover, TM warrants study to determine whether therapy for hepatic Wilson's disease can be further improved.