CONGENITAL DEFICIENCY OF ALPHA1-ANTITRYPSIN REGISTRY

ALPHA1-抗胰蛋白酶登记先天性缺陷

• 批准号: 6311213
• 负责人: RALPH Glenn O'BRIEN
• 金额: $ 5万
• 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 2000-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6311213
• 关键词: alpha 1 antitrypsin deficiency; computer data analysis; emphysema; metabolism disorder chemotherapy; patient /disease registry; protease inhibitor; respiratory function

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.

本登记研究的主要目的是描述 严重先天性α 1- 抗胰蛋白酶缺乏症，无论患者是否正在接受 长期替代治疗。 严重的先天性α 1-抗胰蛋白酶与早期 肺气肿的发病，通常在生命的第三个十年。 一 纠正这一缺陷的方法是用 α 1-抗胰蛋白酶（称为α 1-蛋白酶（A1 Pi）） 其纯化形式的抑制剂）。 目前没有数据库 可用于估计临床获益程度，如果 A1 π 肺气肿的缓慢进展和缺乏 足够的控制组使得难以评估 蛋白酶抑制剂通过对照临床试验。 一 登记册是收集长期影响数据的一种替代办法， A1 Pi长期替代治疗对肺功能减退率影响 功能 登记册还将包括不属于 接受替代疗法，以获得更好的 了解与下列疾病相关的肺功能下降率 先天性缺乏α 1-抗胰蛋白酶

项目成果

Clinical features of individuals with PI*SZ phenotype of alpha 1-antitrypsin deficiency. alpha 1-Antitrypsin Deficiency Registry Study Group.

α1-抗胰蛋白酶缺乏症 PI*SZ 表型个体的临床特征。

• DOI: 10.1164/ajrccm.154.6.8970361
• 发表时间: 1996
• 期刊: American journal of respiratory and critical care medicine
• 影响因子: 24.7
• 作者: Turino,GM;Barker,AF;Brantly,ML;Cohen,AB;Connelly,RP;Crystal,RG;Eden,E;Schluchter,MD;Stoller,JK
• 通讯作者: Stoller,JK

Mortality in individuals with severe deficiency of alpha1-antitrypsin: findings from the National Heart, Lung, and Blood Institute Registry.

严重缺乏 α1-抗胰蛋白酶的个体的死亡率：国家心肺血液研究所登记处的调查结果。

• DOI: 10.1378/chest.127.4.1196
• 发表时间: 2005
• 期刊: Chest
• 影响因子: 9.6
• 作者: Stoller,JamesK;TomashefskiJr,Joseph;Crystal,RonaldG;Arroliga,Alejandro;Strange,Charlie;Killian,DermotN;Schluchter,MarkD;Wiedemann,HerbertP
• 通讯作者: Wiedemann,HerbertP

Quality control of spirometry testing in the registry for patients with severe alpha1-antitrypsin deficiency. alpha1-Antitrypsin Deficiency Registry Study Group.

严重 α1-抗胰蛋白酶缺乏症患者登记处肺活量测定的质量控制。

• DOI: 10.1378/chest.111.4.899
• 发表时间: 1997
• 期刊: Chest
• 影响因子: 9.6
• 作者: Stoller,JK;Buist,AS;Burrows,B;Crystal,RG;Fallat,RJ;McCarthy,K;Schluchter,MD;Soskel,NT;Zhang,R
• 通讯作者: Zhang,R

Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin. The Alpha-1-Antitrypsin Deficiency Registry Study Group.

α1-抗胰蛋白酶严重缺乏的个体的生存率和 FEV1 下降。

• DOI: 10.1164/ajrccm.158.1.9712017
• 发表时间: 1998
• 期刊: American journal of respiratory and critical care medicine
• 影响因子: 24.7