Biochemical Efficacy and Safety of Monthly Augmentation Therapy for α1-Antitrypsin Deficiency

R. C. Hubbard, S. Sellers, D. Czerski, L. Stephens, Ronald Crystal

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The hereditary disorder α1-antitrypsin (α1AT) deficiency results in the development of emphysema due to a diminished anti—neutrophil elastase screen of the lower respiratory tract. Specific therapy for this disorder is available in the form of weekly intravenous infusions of human plasma α1AT, which effectively reconstitute the anti-elastase screen of the lung in these individuals. In an attempt to reduce the frequency of therapy we evaluated the ability of monthly infusions of α1AT to provide equivalent lower respiratory tract protection against neutrophil elastase. Intravenous infusion of 250 mg/kg of α1AT at 28-day intervals to nine individuals with α1AT deficiency and emphysema was carried out for 12 months. Serum α1AT levels exceeded the protective threshold for an average of 25 days after each dose of α1AT was administered. Furthermore, the postinfusion level of α1AT in the nadir lung epithelial lining fluid was fivefold greater than the preinfusion level, and the anti—neutrophil elastase capacity of the nadir epithelial lining fluid also was elevated significantly, nearly threefold above the preinfusion level. These results indicate that monthly administration of human α1AT is fully capable of adequately augmenting serum and lung α1AT levels and anti-elastase capacity and is therefore a rational alternative to weekly therapy.

Original languageEnglish
Pages (from-to)1259-1264
Number of pages6
JournalJAMA: The Journal of the American Medical Association
Issue number9
Publication statusPublished - 2 Sep 1988
Externally publishedYes


ASJC Scopus subject areas

  • Medicine(all)

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