TRIAL OF PENICILLAMINE IN ADVANCED PRIMARY BILIARY-CIRRHOSIS
TRIAL OF PENICILLAMINE IN ADVANCED PRIMARY BILIARY-CIRRHOSIS
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DOI：
10.1056/nejm198504183121602
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发表时间：
1985-01-01
影响因子：
158.5
通讯作者：
MCCALL, JT
中科院分区：
文献类型：
--
作者：
DICKSON, ER;FLEMING, TR;MCCALL, JT
组织学晚期原发性胆汁性肝硬化患者（227例）进入一项双盲、随机、对照试验，以确定青霉胺（1 g/天）是否有效; 111例患者接受药物治疗，116例患者接受安慰剂治疗。两组在临床、生化和组织学特征方面具有高度可比性。与安慰剂相比，青霉胺治疗没有导致生存率的总体改善。临床症状和系列肝脏实验室值反映了疾病的进展性质，两组相似。在连续活检标本的形态学特征方面，治疗组之间无实质性差异。主要副作用的发展导致22%的服用该药物的患者永久停用青霉胺。青霉胺对组织学晚期原发性胆汁性肝硬化患者无效。该试验在早期组织学疾病患者中继续进行，这些患者的预后较好，需要更长时间的随访。
Patients (227) with histologically advanced primary biliary cirrhosis entered a double-blind, randomized, controlled trial to determine whether penicillamine (1 g/day) was therapeutically effective; 111 patients received the drug, and 116 received placebo. The 2 groups were highly comparable at entry with regard to clinical, biochemical and histologic features. Penicillamine therapy did not result in an overall improvement in survival as compared with placebo. Clinical symptoms and serial hepatic laboratory values reflected the progressive nature of the disease and were similar in both groups. There were no substantial differences between treatment groups in the morphologic features of sequential biopsy specimens. The development of major side effects led to permanent discontinuation of penicillamine in 22% of the patients taking the drug. Penicillamine is not useful for patients with histologically advanced primary biliary cirrhosis. The trial is being continued in patients with early histologic disease whose better prognosis necessitates longer follow-up.