Intravenous augmentation treatment and lung density

Intravenous augmentation treatment and lung density in severe alpha1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled trial
Kenneth R Chapman, Jonathan G W Burdon, Eeva Piitulainen, Robert A Sandhaus, Niels Seersholm, James M Stocks, Berend C Stoel, Liping Huang, Zhenling Yao, Jonathan M Edelman, Noel G McElvaney, on behalf of the RAPID Trial Study Group*

Summary

Background

The efficacy of ?1 proteinase inhibitor (A1PI) augmentation treatment for ?1 antitrypsin deficiency has
not been substantiated by a randomised, placebo-controlled trial. CT-measured lung density is a more sensitive
measure of disease progression in ?1 antitrypsin deficiency emphysema than spirometry is, so we aimed to assess the
efficacy of augmentation treatment with this measure.
Methods The RAPID study was a multicentre, double-blind, randomised, parallel-group, placebo-controlled trial of
A1PI treatment in patients with ?1 antitrypsin deficiency. We recruited eligible non-smokers (aged 18–65 years) in
28 international study centres in 13 countries if they had severe ?1 antitrypsin deficiency (serum concentration with a forced expiratory volume in 1 s of 35–70% (predicted). We excluded patients if they had undergone, or were on
the waiting list to undergo, lung transplantation, lobectomy, or lung volume-reduction surgery, or had selective IgA
deficiency. We randomly assigned patients (1:1; done by Accovion) using a computerised pseudorandom number
generator (block size of four) with centre stratification to receive A1PI intravenously 60 mg/kg per week or placebo for
24 months. All patients and study investigators (including those assessing outcomes) were unaware of treatment
allocation throughout the study. Primary endpoints were CT lung density at total lung capacity (TLC) and functional
residual capacity (FRC) combined, and the two separately, at 0, 3, 12, 21, and 24 months, analysed by modified intention
to treat (patients needed at least one evaluable lung density measurement). This study is registered with
ClinicalTrials.gov, number NCT00261833. A 2-year open-label extension study was also completed (NCT00670007).

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