Assessing the efficacy of Alpha₁-Proteinase inhibitor (human) augmentation therapy for Alpha₁-Antitrypsin deficiency - Related emphysema: Challenges and opportunities.

ABSTRACT: Clinical benefit of Alpha₁-Proteinase Inhibitor (Human) (A₁-PI) products for Alpha₁-antitrypsin deficiency (AATD) is uncertain, based on a systematic review of observational studies and randomized controlled trials (RCTs) in AATD of Alpha₁-Proteinase Inhibitor (Human) (A₁-PI) products. At the recommended dose, A₁-PI products raise its serum concentration but do not normalize levels. Observational studies suggest A₁-PI might modestly slow progression of airflow limitation in patients with intermediate airflow obstruction, a finding not confirmed by three placebo-controlled RCTs of limited power, which showed non-significant rates of forced expiratory volume in 1 s (FEV₁) change favoring placebo. These RC

SUBMITTER: Pierce LR 

PROVIDER: S-EPMC11190464 | biostudies-literature | 2024 Jun

REPOSITORIES: biostudies-literature