REGISTRY-CONGENITAL DEFICIENCY OF ALPHA1-ANTITRYPSIN

登记-先天性α1-抗胰蛋白酶缺陷

• 批准号: 2317402
• 负责人: GEORGE W WILLIAMS
• 金额: $ 34.92万
• 依托单位: CLEVELAND CLINIC FOUNDATION
• 依托单位国家: 美国
• 财政年份: 1988
• 资助国家: 美国
• 起止时间: 1988-09-30 至 1994-09-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/2317402
• 关键词: REGISTRY; CONGENITAL; DEFICIENCY; ALPHA1; ANTITRYPSIN

The primary objective of this registry will be to characterize the clinical and laboratory course of severe congential alpha1- antitrypsin deficiency whether or not the patient is undergoing long-term replacement therapy. Severe congenital alpha1-antitrypsin is associated with the early onset of emphysema, usually by the third decade of life. One approach to correct this deficiency is through replacement with alpha1-antitrypsin (referred to as alpha1-proteinase (A1 Pi) inhibitor in its purified form). There currently is no data base available for estimating the degree of clinical benefit if any of A1 Pi. The slow progression of emphysema and lack of an adequate control group have made it difficult to evaluate the proteinase inhibitor through a controlled clinical trial. A registry is an alternative to collect data on the effect of long- term replacement therapy with A1 Pi on rate of decline of lung function. The registry will also include individuals who are not receiving the replacement therapy in order to obtain a better knowledge of the rate of decline of lung function associated with the congenital deficiency for alpha1-antitrypsin.

该注册表的主要目标是描述 严重先天性α1-的临床和实验室过程 抗胰蛋白酶缺乏症，无论患者是否正在接受 长期替代疗法。 严重的先天性 α1-抗胰蛋白酶与早期 肺气肿通常在三十岁时发病。 一 纠正这一缺陷的方法是通过替换 α1-抗胰蛋白酶（简称α1-蛋白酶（A1 Pi） 纯化形式的抑制剂）。 目前没有数据库 可用于估计临床获益程度（如果有） A1 皮。 肺气肿进展缓慢且缺乏 足够的对照组使得评估 通过对照临床试验的蛋白酶抑制剂。 一个 注册表是收集有关长期影响的数据的替代方法 A1 Pi 定期替代疗法对肺功能衰退率的影响 功能。 登记处还将包括不属于登记范围的个人 接受替代治疗以获得更好的效果 了解与以下因素相关的肺功能下降速度 先天性α1-抗胰蛋白酶缺乏症。