REGISTRY-CONGENITAL DEFICIENCY OF ALPHA1-ANTITRYPSIN

登记-先天性α1-抗胰蛋白酶缺陷

• 批准号: 2317405
• 负责人: GEORGE W WILLIAMS
• 金额: $ 8万
• 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/2317405
• 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.

这一登记处的主要目标将是确定 重度先天性甲型肝炎的临床和实验室病程 抗胰蛋白酶缺乏症，无论患者是否正在接受治疗 长期替代疗法。 严重的先天性α1-抗胰蛋白酶与早期 肺气肿的发作，通常在生命的第三个十年。一 纠正这一缺陷的方法是通过替换 α1-抗胰蛋白酶(又称α1-蛋白酶(A1-PI)) 纯化形式的抑制剂)。目前没有数据库 可用于估计临床受益的程度，如果有 A1 PI。肺气肿的缓慢发展和缺乏 足够的控制组使评估 蛋白水解酶抑制剂通过临床对照试验。一个 登记处是收集关于长期-经济影响的数据的替代方案 A1PI定期替代治疗对肺衰退率的影响 功能。登记处还将包括未登记的个人 接受替代疗法，以获得更好的 对肺功能下降率的了解与以下方面有关 先天性甲型抗胰蛋白酶缺乏症。