Findings from CSL Behring’s RAPID study have demonstrated the effectiveness of augmentation therapy in slowing emphysema due to Alpha-1.

CSL Behring, a global biopharmaceutical company sponsored the study in which 180 severely deficient Alpha-1 patients were randomly assigned to receive either the augmentation product Zemaira or a placebo over a two year period followed by a two year ‘open-label’ period in which all participants were offered augmentation therapy. CT scan lung density was measured at baseline, three months, one and two years. Secondary endpoints included spirometry, changes in exercise capacity and the rate of pulmonary exacerbations over two years.

Zemaira is a highly purified alpha-1 protein derived from human plasma which is used to raise therapeutic levels of the alpha-1 protein in those with Alpha-1 (augmentation therapy). Prior to the RAPID study its efficacy in augmentation therapy on the frequency of pulmonary exacerbations and the progression of emphysema had not been established in clinical trials.

According to findings of the RAPID (Randomized, Placebo-controlled Trial of Augmentation Therapy in Alpha-1 Proteinase Inhibitor Deficiency) study, which was published by The Lancet, patients with Alpha-1 treated with Alpha₁-Proteinase Inhibitor (Zemaira) therapy exhibited a lower annual rate of lung density decline compared to those who were given the placebo, when measured using chest computed tomography (CT), at full inspiration. Secondary outcome variables and adverse events were not significantly different between groups.

The Lancet article stated that an interim analysis of data from the two-year extension trial suggested that early treatment with augmentation therapy showed persistent efficacy in patients with Alpha-1 and emphysema. In addition, when patients who had been receiving a placebo in the original two-year trial switched to treatment with augmentation therapy, their lung density decline (which was more rapid than the treated group in the main study) slowed to the same rate as the treatment group. “These findings should encourage early introduction of augmentation therapy to patients with emphysema due to Alpha-1 and should also stimulate further research into optimum dosing.”.

Lead author Kenneth Chapman, MD, director of the Asthma and Airway Centre of the University Health Network in Toronto, Canada, called the trial “a landmark study validating almost two decades of focus on the lung-density endpoint as the most sensitive way to track lung tissue decline and the seven-year collaboration of an international team of investigators. It is 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 (Zemaira) 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’.

To read more on this study see here