Abstract
Background
RAPID (NCT00261833) showed that augmentation therapy with alpha-1 proteinase inhibitor (A1-PI Z, Zemaira®, CSL Behring) slows lung density decline in A1-PI-deficient patients.
Aim
To assess clinical benefit over 48 months in subjects who completed RAPID and have data from ≥2 CT scans from the extension study to date (NCT00670007).
Methods
In a randomized, controlled design, RAPID subjects received A1-PI Z or placebo for 24 months, after which non-US subjects entered the 24-month extension study and all received A1-PI Z. Changes in adjusted lung density P15 at total lung capacity were assessed by CT.
Results
Rate of lung density decline was significantly reduced with A1-PI Z vs. placebo and was similar (1–1.5 g/L/y) among A1-PI Z subjects in the blinded and open-label phases. In the ongoing extension study, the delayed-start cohort (placebo–A1-PI Z) had a reduced rate of decline very similar to the early-start cohort.
Conclusions
A1-PI Z showed clinical benefit over 4 years and at different intervention points (early or delayed). Early treatment may reduce overall progression of emphysema in A1-PI-deficient patients.
- © 2014 ERS