PMID- 27564674
OWN - NLM
STAT- MEDLINE
DCOM- 20180105
LR  - 20180924
IS  - 2325-6621 (Electronic)
IS  - 2325-6621 (Linking)
VI  - 13 Suppl 4
DP  - 2016 Aug
TI  - Randomized, Placebo-Controlled Trials in Alpha-1 Antitrypsin Deficiency.
PG  - S370-3
LID - 10.1513/AnnalsATS.201510-684KV [doi]
AB  - Alpha-1 antitrypsin deficiency (AATD) is a condition caused by the inheritance of 
      two mutated SERPINA1 gene alleles. Individuals with AATD are at increased risk of 
      injury to the liver and lungs. The pulmonary manifestations include precocious 
      onset of pulmonary emphysema and bronchiectasis. For nearly three decades, 
      treatment has been available to individuals with emphysema caused by AATD, but 
      this therapy-augmentation of plasma and tissue alpha-1 antitrypsin levels by 
      intravenous administration of human plasma-derived protein-was approved by 
      regulatory authorities based on its biochemical efficacy. This therapy appears to 
      slow the progression of emphysema in patients with AATD. The medical, patient, 
      and regulatory communities have sought assurance that this expensive therapy 
      provides measurable clinical benefit. Documenting such benefit has been difficult 
      because of the slow progression of the underlying lung disease in AATD, the 
      rarity of this genetic condition, and the lack of direct quantitative 
      measurements of emphysema progression. Over the past decade, quantitative 
      computed tomography (CT) densitometry of the lungs has been found to correlate 
      with severity and progression of emphysema. The recent publication of a 
      well-powered, masked, placebo-controlled study using CT densitometry to evaluate 
      the effectiveness of augmentation therapy at slowing the progression of emphysema 
      has provided some assurance of the clinical efficacy of this therapy.
FAU - Sandhaus, Robert A
AU  - Sandhaus RA
AUID- ORCID: 0000-0002-7937-2485
AD  - Division of Pulmonary, Critical Care Medicine, and Sleep, National Jewish Health, 
      Denver, Colorado.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Review
PL  - United States
TA  - Ann Am Thorac Soc
JT  - Annals of the American Thoracic Society
JID - 101600811
RN  - 0 (Serine Proteinase Inhibitors)
RN  - 0 (alpha 1-Antitrypsin)
SB  - IM
MH  - Absorptiometry, Photon
MH  - Humans
MH  - Pulmonary Emphysema/diagnostic imaging/etiology/*therapy
MH  - Randomized Controlled Trials as Topic
MH  - Serine Proteinase Inhibitors/*therapeutic use
MH  - Tomography, X-Ray Computed
MH  - Treatment Outcome
MH  - alpha 1-Antitrypsin/*therapeutic use
MH  - alpha 1-Antitrypsin Deficiency/complications/diagnostic imaging/*therapy
OTO - NOTNLM
OT  - alpha-1 antitrypsin deficiency
OT  - augmentation therapy
EDAT- 2016/08/27 06:00
MHDA- 2018/01/06 06:00
CRDT- 2016/08/27 06:00
PHST- 2016/08/27 06:00 [entrez]
PHST- 2016/08/27 06:00 [pubmed]
PHST- 2018/01/06 06:00 [medline]
AID - 10.1513/AnnalsATS.201510-684KV [doi]
PST - ppublish
SO  - Ann Am Thorac Soc. 2016 Aug;13 Suppl 4:S370-3. doi: 
      10.1513/AnnalsATS.201510-684KV.