Editorial
Augmentation therapy for severe
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
1-antiprotease
in severe
1-antitrypsin (
1AT)
deficiency
a treatment dubbed "augmentation therapy"
is to
provide the substance whose deficiency is deemed responsible for the
pulmonary sequelae of
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
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