PMID- 30775428
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20200929
IS  - 2372-952X (Print)
IS  - 2372-952X (Electronic)
IS  - 2372-952X (Linking)
VI  - 6
IP  - 1
DP  - 2018 Nov 28
TI  - Intravenous Alpha-1 Antitrypsin Therapy for Alpha-1 Antitrypsin Deficiency: The 
      Current State of the Evidence.
PG  - 100-114
LID - 10.15326/jcopdf.6.1.2017.0185 [doi]
AB  - 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.
FAU - Brantly, Mark L
AU  - Brantly ML
AD  - Division of Pulmonary, Critical Care and Sleep Medicine, University of Florida, 
      Gainesville.
FAU - Lascano, Jorge E
AU  - Lascano JE
AD  - Division of Pulmonary, Critical Care and Sleep Medicine, University of Florida, 
      Gainesville.
FAU - Shahmohammadi, Abbas
AU  - Shahmohammadi A
AD  - Division of Pulmonary, Critical Care and Sleep Medicine, University of Florida, 
      Gainesville.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20181128
PL  - United States
TA  - Chronic Obstr Pulm Dis
JT  - Chronic obstructive pulmonary diseases (Miami, Fla.)
JID - 101635411
PMC - PMC6373587
OTO - NOTNLM
OT  - AAT therapy
OT  - alpha-1 antitrypsin deficiency
OT  - clinical efficacy
OT  - copd
OT  - disease modification
OT  - emphysema
COIS- 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.
EDAT- 2019/02/19 06:00
MHDA- 2019/02/19 06:01
PMCR- 2018/11/28
CRDT- 2019/02/19 06:00
PHST- 2019/02/19 06:00 [entrez]
PHST- 2019/02/19 06:00 [pubmed]
PHST- 2019/02/19 06:01 [medline]
PHST- 2018/11/28 00:00 [pmc-release]
AID - 10.15326/jcopdf.6.1.2017.0185 [doi]
PST - epublish
SO  - Chronic Obstr Pulm Dis. 2018 Nov 28;6(1):100-114. doi: 
      10.15326/jcopdf.6.1.2017.0185.