A review of augmentation therapy for alpha-1 antitrypsin deficiency

Manish Mohanka, Danai Khemasuwan, James K. Stoller

Research output: Contribution to journalArticle

26 Scopus citations

Abstract

Introduction: Alpha-1 antitrypsin deficiency (AATD) is a relatively common, but under-recognized condition which manifests commonly with liver cirrhosis and emphysema. Specific therapy for lung-affected individuals with AATD is augmentation therapy, which consists of intravenous infusion of purified human plasma-derived alpha-1 antitrypsin (AAT). Augmentation therapy was first approved by the United States Food and Drug Administration (FDA) in 1987 for emphysema associated with severe AATD and today, six augmentation therapy preparations, all of which derive from pooled human plasma, have received FDA approval. Areas covered: This paper reviews augmentation therapy for AATD, including the various available commercial preparations, their processing and biochemical differences, evidence regarding biochemical and clinical efficacy, patterns of clinical use, adverse effect profiles, cost-effectiveness and potential uses in conditions other than emphysema associated with AATD. Novel and emerging strategies for treating AATD are briefly discussed next, including alternative dosing and administration strategies, recombinant preparations, small molecule inhibitors of neutrophil elastase and of AAT polymerization, autophagy-enhancing drugs and gene therapy approaches. Expert opinion: We conclude with a discussion of our approach to managing patients with AATD and use of augmentation therapy.

Original languageEnglish (US)
Pages (from-to)685-700
Number of pages16
JournalExpert Opinion on Biological Therapy
Volume12
Issue number6
DOIs
Publication statusPublished - Jun 2012

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Keywords

  • Alpha-1 antitrypsin
  • Augmentation therapy
  • Cirrhosis
  • Emphysema
  • Gene therapy
  • Panniculitis
  • Protease inhibitor
  • Pulmonary function test

ASJC Scopus subject areas

  • Pharmacology
  • Clinical Biochemistry
  • Drug Discovery

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