Introduction: Intravenous plasma-purified alpha-1 antitrypsin (IV-AAT) has been shown to be biochemically effective in treating AAT deficiency (AATD) since the 1980s. The RAPID-OLE study demonstrated a beneficial effect of IV-AAT on preservation of lung parenchymal density, as measured by CT lung density, a marker of emphysema and disease progression.

Methods: Our study followed 615 severe AATD patients over a mean of 58 months (max of 286 months) across 3 countries with socialised healthcare - Ireland, Switzerland and Austria but with differing access to IV-AAT. Spirometry and diffusion capacity were measured at intervals over this timespan.

Results: Kaplan-Meier survival analysis over 25 years demonstrated a survival benefit from receiving IV-AAT compared to those not receiving IV-AAT (p = 0.00076) (figure 1). 
There was no appreciable difference in rate of decline of lung function between those receiving IV-AAT and those who did not.

Conclusion: Our three-country observational study demonstrates that the benefit of IV-AAT may not be appreciable when using lung function as a marker of disease progression in AATD and that IV-AAT has benefits beyond preserving lung function decline.

Figure 1: Kaplan-Meier survival analysis stratified by augmentation therapy status across all three countries.