Augmentation therapy

While there is no cure for alpha-1-antitrypsin (A1AT) deficiency (also known as alpha-1), there is treatment available.* By raising A1AT levels in the blood and lungs, augmentation therapy helps keep neutrophil elastase in check in alpha-1 patients. Augmentation therapy consists of intravenous infusions of A1AT. The goal of treatment is to elevate levels of A1AT in the plasma and lung interstitium and correct the deficiency state.1

  • Augmentation therapy has been demonstrated to raise levels of A1AT in the blood and lungs above the threshold needed to protect lung tissue
  • The efficacy of augmentation therapy has been demonstrated in both randomized, controlled studies2,3 as well as in an observational study with a multicenter registry of 1,129 patients.4 These efficacy benefits include:
    • Significantly increased 5-year survival for AATD patients on augmentation therapy versus not on augmentation therapy (observational registry study)4
    • Significant reduction in the rate of emphysema progression, measured as lung tissue density measured by CT densitometry (randomized controlled studies)2,3
    • Significant reduction in the rate of FEV1 decline among patients with mean FEV1 of 35-49% (observational registry study)4
  • ATS/ERS guidelines recommend intravenous augmentation therapy for patients with emphysema attributable to A1AT deficiency5

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  1. Stoller JK, Aboussouan LS. Alpha-1 antitrypsin deficiency. Lancet. 2005;365(9478):2225-2236
  2. Stockley RA, et al. Therapeutic efficacy of alpha-1 antitrypsin augmentation therapy on the loss of lung tissue: an integrated analysis of 2 randomised clinical trials using computed tomography densitometry. Respir Res. 2010; 11:136
  3. Chapman KR, et al. Intravenous augmentation treatment and lung density in severe α1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled trial. Lancet. 2015; 386: 360-68
  4. Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin.The Alpha-1-Antitrypsin Deficiency Registry Study Group. Am J Respir Crit Care Med. 1998;158:49-59
  5. American Thoracic Society/European Respiratory Society statement: standards for the diagnosis and management of individuals with alpha-1antitrypsin deficiency. Am J Respir Crit Care Med. 2003;168:818-900

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