What are the benefits of IV augmentation therapy for the treatment of alpha1-antitrypsin deficiency (AATD)?

Updated: Sep 11, 2020
  • Author: Dora E Izaguirre Anariba, MD, MPH; Chief Editor: John J Oppenheimer, MD  more...
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Answer

No controlled studies have proven that IV augmentation therapy improves survival or slows the rate of emphysema progression. Results from the NIH patient registry and a comparison of Danish and German registries have been published, and both suggest that augmentation therapy has beneficial effects. Although they were not controlled treatment trials, the similarity of the results suggests that the findings are significant.

The NIH report described an overall death rate 1.5 times higher for those who did not receive augmentation therapy and a rate of FEV1 decline (54 mL/y) in alpha1-antitrypsin-deficient individuals, about twice that of healthy nonsmokers but about 50% that of smokers (108 mL/y). While Prolastin augmentation therapy did not improve the average FEV1 decline (54 mL/y) in the group as a whole, participants with moderate airflow obstruction (FEV1 35-60% of predicted value) had a slower rate of decline (mean difference 27 mL/y).

These findings bolster the long-held belief that augmentation therapy provides clinical benefit. Studies of Aralast and Zemaira have shown equivalency with Prolastin in achieving and maintaining alpha1-antitrypsin serum levels and alveolar epithelial levels above the target level. No studies of Aralast or Zemaira have been performed to show effects on FEV1, rate of decline of FEV1, or survival.


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