PMID- 22500781
OWN - NLM
STAT- MEDLINE
DCOM- 20120906
LR  - 20131106
IS  - 1744-7682 (Electronic)
IS  - 1471-2598 (Linking)
VI  - 12
IP  - 6
DP  - 2012 Jun
TI  - A review of augmentation therapy for alpha-1 antitrypsin deficiency.
PG  - 685-700
LID - 10.1517/14712598.2012.676638 [doi]
AB  - 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.
FAU - Mohanka, Manish
AU  - Mohanka M
AD  - Respiratory Institute, Cleveland Clinic, 9500 Euclid Avenue, A90, Cleveland, OH 
      44195, USA.
FAU - Khemasuwan, Danai
AU  - Khemasuwan D
FAU - Stoller, James K
AU  - Stoller JK
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Review
DEP - 20120414
PL  - England
TA  - Expert Opin Biol Ther
JT  - Expert opinion on biological therapy
JID - 101125414
RN  - 0 (Protease Inhibitors)
RN  - 0 (Recombinant Proteins)
RN  - 0 (SERPINA1 protein, human)
RN  - 0 (alpha 1-Antitrypsin)
RN  - EC 3.4.21.37 (Leukocyte Elastase)
SB  - IM
MH  - Animals
MH  - Cost-Benefit Analysis
MH  - Drug Costs
MH  - *Enzyme Replacement Therapy/adverse effects/economics
MH  - Genetic Therapy
MH  - Humans
MH  - Infusions, Intravenous
MH  - Leukocyte Elastase/antagonists & inhibitors/metabolism
MH  - Protease Inhibitors/therapeutic use
MH  - Recombinant Proteins/administration & dosage
MH  - Treatment Outcome
MH  - alpha 1-Antitrypsin/*administration & dosage/adverse effects/economics/genetics
MH  - alpha 1-Antitrypsin Deficiency/economics/enzymology/genetics/*therapy
EDAT- 2012/04/17 06:00
MHDA- 2012/09/07 06:00
CRDT- 2012/04/17 06:00
PHST- 2012/04/17 06:00 [entrez]
PHST- 2012/04/17 06:00 [pubmed]
PHST- 2012/09/07 06:00 [medline]
AID - 10.1517/14712598.2012.676638 [doi]
PST - ppublish
SO  - Expert Opin Biol Ther. 2012 Jun;12(6):685-700. doi: 10.1517/14712598.2012.676638. 
      Epub 2012 Apr 14.