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Thorax 1998;53:1007-1009; doi:10.1136/thx.53.12.1007
Copyright © 1998 BMJ Publishing Group Ltd & British Thoracic Society.
Thorax 1998;53:1007-1009 ( December )

Editorial

Augmentation therapy for severe alpha 1-antitrypsin deficiency: is the jury still out on a trial?

The first 150 words of the full text of this article appear below.

As with the administration of insulin for diabetes mellitus, the rationale for administering purified alpha 1-antiprotease in severe alpha 1-antitrypsin (alpha 1AT) deficiency---a treatment dubbed "augmentation therapy"---is to provide the substance whose deficiency is deemed responsible for the pulmonary sequelae of alpha 1AT deficiency. Indeed, even as intravenous augmentation therapy has been advocated for severely deficient individuals with established airflow obstruction by some official societies (but not all),1 2 and even as one commercial product has been licensed by the United States Federal Drug Administration with other intravenous and inhaled preparations currently under investigation, the efficacy of augmentation therapy continues to be actively debated. As with many controversies in medicine, this one centres on the nature and adequacy of available supportive evidence versus the gaps in current knowledge. In the wake of the recently published outcome data from the American National Heart, Lung, and Blood Institute (NHLBI) sponsored Registry of Patients with . . . [Full text of this article]


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This article has been cited by other articles:

  • Stoller, J K (2004). {alpha}1-Antitrypsin deficiency. Thorax 59: 92-93 [Full Text]  
  • Tremblay, G. M., Vachon, E., Larouche, C., Bourbonnais, Y. (2002). Inhibition of Human Neutrophil Elastase-Induced Acute Lung Injury in Hamsters by Recombinant Human Pre-elafin (Trappin-2). Chest 121: 582-588 [Abstract] [Full Text]  
  • Stoller, J. K., Brantly, M., Fleming, L. E., Bean, J. A., Walsh, J. (2000). Formation and Current Results of a Patient-Organized Registry for {alpha}1-Antitrypsin Deficiency. Chest 118: 843-848 [Abstract] [Full Text]  
  • Stockley, R A (2000). Alpha-1-antitrypsin deficiency: what next?. Thorax 55: 614-618 [Full Text]  

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