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Review
, 6 (1), 100-114

Intravenous Alpha-1 Antitrypsin Therapy for Alpha-1 Antitrypsin Deficiency: The Current State of the Evidence

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Review

Intravenous Alpha-1 Antitrypsin Therapy for Alpha-1 Antitrypsin Deficiency: The Current State of the Evidence

Mark L Brantly et al. Chronic Obstr Pulm Dis.

Abstract

Alpha-1 antitrypsin deficiency (AATD) is a largely monogenetic disorder associated with a high risk for the development of chronic obstructive pulmonary disease (COPD) and cirrhosis. Intravenous alpha-1 antitrypsin (AAT) therapy has been available for the treatment of individuals with AATD and COPD since the late 1980s. Initial Food and Drug Administration (FDA) approval was granted based on biochemical efficacy. Following its approval, the FDA, scientific community and third-party payers encouraged manufacturers of AAT therapy to determine its clinical efficacy. This task has proved challenging because AATD is a rare, orphan disorder comprised of individuals who are geographically dispersed and infrequently identified. In addition, robust clinical trial outcomes have been lacking until recently. This review provides an update on the evidence for the clinical efficacy of intravenous AAT therapy for patients with AATD-related emphysema.

Keywords: AAT therapy; alpha-1 antitrypsin deficiency; clinical efficacy; copd; disease modification; emphysema.

Conflict of interest statement

GaryThe authors have no conflicts of interest to declare. Medical writing assistance was provided by Steven Foster of Meridian HealthComms Ltd (Plumley, United Kingdom), funded by CSL Behring.

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Grant support

Medical writing assistance was funded by CSL Behring
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