Does alpha1-antitrypsin augmentation therapy slow the annual decline in FEV1 in patients with severe hereditary alpha1-antitrypsin deficiency? Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL) alpha1-AT study group

摘要: Patients with severe hereditary alpha1-antitrypsin deficiency (alpha1-ATD) face a high risk of developing emphysema at young age. Intravenous augmentation therapy purified human (alpha1-AT) is now available. However, controlled trial to show its efficacy has never been carried out. The aim this study was compare the decline in forced expiratory volume one second (deltaFEV1) between Danish patients who had received and German treated weekly infusion alpha1-AT. From files alpha1-ATD register, 97 exsmokers, PiZ phenotype for whom results least two lung function measurements an interval 1 yr were available, identified. group infusions alpha1-AT, 60 mg x kg(-1) body weight, 198 biannual deltaFEV1 compared treatment groups by random effects modelling. significantly lower than untreated group, annual declines 53 mL yr(-1) (95% confidence CI) 48-58 yr(-1)) 75 CI 63-87 yr(-1)), respectively (p=0.02). differed respect gender initial FEV1% predicted. Gender did not have any influence on deltaFEV1. Stratification pred showed significant effect only 31-65%, reduced 21 yr(-1). This nonrandomized suggests that moderately may slow second.