BACKGROUND: Augmentation with exogenous alpha1-antitrypsin (alpha1-AT) is the only specific therapy for alpha1-AT deficiency. Uncertainty persists concerning its effectiveness. PURPOSE: To test the hypothesis that augmentation therapy in patients with alpha1-AT deficiency slows the decline in FEV1. STUDY SELECTION: Randomized and nonrandomized clinical studies with either parallel-group design or single cohort pre-post design were eligible if they compared augmentation therapy with a control regimen and if long-term (> 1 y) longitudinal FEV1 follow-up data were collected. DATA SYNTHESIS: FEV1 data from five trials with 1509 patients were combined by random effects meta-analysis. The decline in FEV1 was slower by 23% (absolute difference, 13.4 ml/year; CI, 1.5 to 25.3 ml/year) among all patients receiving augmentation therapy. This overall protective effect reflected predominantly the results in the subset of patients with baseline FEV1 30-65% of predicted. In that subset, augmentation was associated with a 26% reduction in rate of FEV1 decline (absolute difference, 17.9 ml/year; CI, 9.6 to 26.1 ml/year). Similar trends amongst patients with baseline FEV1 percent of predicted < 30% or > 65% were not statistically significant. CONCLUSIONS: This meta-analysis supports the conclusion that augmentation can slow lung function decline in patients with AAT deficiency Patients with moderate obstruction are most likely to benefit.
BACKGROUND: Augmentation with exogenous alpha1-antitrypsin (alpha1-AT) is the only specific therapy for alpha1-AT deficiency. Uncertainty persists concerning its effectiveness. PURPOSE: To test the hypothesis that augmentation therapy in patients with alpha1-AT deficiency slows the decline in FEV1. STUDY SELECTION: Randomized and nonrandomized clinical studies with either parallel-group design or single cohort pre-post design were eligible if they compared augmentation therapy with a control regimen and if long-term (> 1 y) longitudinal FEV1 follow-up data were collected. DATA SYNTHESIS: FEV1 data from five trials with 1509 patients were combined by random effects meta-analysis. The decline in FEV1 was slower by 23% (absolute difference, 13.4 ml/year; CI, 1.5 to 25.3 ml/year) among all patients receiving augmentation therapy. This overall protective effect reflected predominantly the results in the subset of patients with baseline FEV1 30-65% of predicted. In that subset, augmentation was associated with a 26% reduction in rate of FEV1 decline (absolute difference, 17.9 ml/year; CI, 9.6 to 26.1 ml/year). Similar trends amongst patients with baseline FEV1 percent of predicted < 30% or > 65% were not statistically significant. CONCLUSIONS: This meta-analysis supports the conclusion that augmentation can slow lung function decline in patients with AAT deficiencyPatients with moderate obstruction are most likely to benefit.
Authors: Darcy D Marciniuk; P Hernandez; M Balter; J Bourbeau; K R Chapman; G T Ford; J L Lauzon; F Maltais; D E O'Donnell; D Goodridge; C Strange; A J Cave; K Curren; S Muthuri Journal: Can Respir J Date: 2012 Mar-Apr Impact factor: 2.409
Authors: Theodore G Liou; Eric P Elkin; David J Pasta; Joan R Jacobs; Michael W Konstan; Wayne J Morgan; Jeffrey S Wagener Journal: J Cyst Fibros Date: 2010-05-14 Impact factor: 5.482
Authors: Robert A Sandhaus; Gerard Turino; Mark L Brantly; Michael Campos; Carroll E Cross; Kenneth Goodman; D Kyle Hogarth; Shandra L Knight; James M Stocks; James K Stoller; Charlie Strange; Jeffrey Teckman Journal: Chronic Obstr Pulm Dis Date: 2016-06-06
Authors: Adam Wanner; Stephen C Groft; J Russell Teagarden; Jeffrey Krischer; Barry R Davis; Christopher S Coffey; David H Hickam; Jeffrey Teckman; David R Nelson; Michael L McCaleb; Rohit Loomba; Charlie Strange; Robert A Sandhaus; Mark Brantly; Jonathan M Edelman; Albert Farrugia Journal: Chronic Obstr Pulm Dis Date: 2015-04-28