Treatment with augmentation therapy is effective in slowing the progression of emphysema in patients with alpha-1 antitrypsin deficiency (AATD), according to a new study presented at the 2013 American Thoracic Society International Conference in Philadelphia.
The study showed the efficacy of augmentation therapy in preventing the loss of lung tissue as measured by computed tomography (CT) scan lung density at full inspiration (TLC), which is a more sensitive measure of disease progression than conventional parameters. This is the first prospective study to demonstrate the efficacy and safety of augmentation therapy in a randomized, placebo-controlled trial using this parameter.
“Our experience of the last quarter-century has been that augmentation therapy is associated with better preserved lung function and reduced mortality, “ said lead author Kenneth R. Chapman, MD, director of the Asthma and Airway Centre of the University Health Network, in Toronto.
“This randomized, placebo-controlled trial using a sensitive measure of lung density adds the most rigorous evidence to date that augmentation therapy slows the progression of emphysema in patients with Alpha-1 antitrypsin deficiency. The effect of A1-PI seen in this trial was both clinically and statistically significant, finally confirming its benefit in preventing the loss of lung tissue in patients with this potentially debilitating disease.”
Read the news release on the study.
CSL Behring sponsored the RAPID trial, which randomly assigned 180 Alpha-1 patients to receive either the augmentation product Zemaira or a placebo for a two-year period. The trial was conducted at 28 sites in the United States, Europe, Canada, Australia and Russia.
The trial is the first well-powered randomised, placebo-controlled trial to use CT scan lung density as the primary outcome measure. Professor Rob Stockley explained at the recent Info Day in Bristol that CT scans are considered the most sensitive measure of emphysema detection and progression.