Schilsky M L, Scheinberg I H, Sternlieb I
Department of Medicine, Albert Einstein College of Medicine, Bronx, New York 10461.
Hepatology. 1994 Mar;19(3):583-7. doi: 10.1002/hep.1840190307.
The objective of this study was to determine the indications for and results of liver transplantation in patients with Wilson's disease on the basis of results of a survey with retrospective review of data obtained on 55 transplants performed at centers in the United States and Europe. The study group comprised 32 females and 23 males, aged 8.5 to 51 yr, with features diagnostic of Wilson's disease. Indication for orthotopic liver transplantation included hepatic insufficiency (n = 32), wilsonian fulminant hepatitis (n = 21), intractable neurological Wilson's disease (n = 1) and gastrointestinal hemorrhage (n = 1). Forty-three patients have survived, at this writing, from 3 mo to 20 yr. Mean and median survival after orthotopic liver transplantation were 2.7 and 2.5 yr, respectively. Survival at 1 yr was 79%. Nonfatal complications occurred in five patients. Of the seven patients given transplants for hepatic insufficiency who manifested neurological and/or psychiatric manifestations at the time of orthotopic liver transplantation, four showed improvement of these symptoms. One patient given a transplant for intractable neurological disease improved but died of a vascular complication. Our data demonstrate that liver transplantation is life-saving but not without risk for patients with wilsonian fulminant hepatitis or chronic severe hepatic insufficiency unresponsive to medical therapy. Furthermore, neurological or psychiatric symptoms due to Wilson's disease may improve after liver transplantation; however, the role of this procedure in the management of patients with neurological Wilson's disease in the absence of hepatic insufficiency is still uncertain.
本研究的目的是，基于对美国和欧洲各中心开展的55例肝移植手术数据进行回顾性调查的结果，确定威尔逊病患者肝移植的指征及结果。研究组包括32名女性和23名男性，年龄在8.5至51岁之间，具有威尔逊病的诊断特征。原位肝移植的指征包括肝功能不全（n = 32）、威尔逊暴发性肝炎（n = 21）、难治性神经型威尔逊病（n = 1）和胃肠道出血（n = 1）。撰写本文时，43例患者已存活3个月至20年。原位肝移植后的平均生存期和中位生存期分别为2.7年和2.5年。1年生存率为79%。5例患者出现非致命性并发症。在7例因肝功能不全接受移植且在原位肝移植时出现神经和/或精神症状的患者中，4例症状有所改善。1例因难治性神经疾病接受移植的患者病情改善，但死于血管并发症。我们的数据表明肝移植可挽救生命，但对于威尔逊暴发性肝炎或对药物治疗无反应的慢性严重肝功能不全患者而言并非毫无风险。此外，威尔逊病所致的神经或精神症状在肝移植后可能改善；然而，该手术在无肝功能不全的神经型威尔逊病患者管理中的作用仍不确定。
