Intravenous augmentation treatment and lung density in severe α1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled tria -

Intravenous augmentation treatment and lung density in severe α1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled tria
Information type: Randomised controlled trials
Source: Lancet Public Health
Specialities: Respiratory disorders
Summary
2 year RCT (n=180 with α1 antitrypsin deficiency) found CT-measured lung density (LD) at total lung capacity alone provides evidence that α1 proteinase inhibitor augmentation therapy slows progression of emphysema (annual rate LD loss: −1.45 vs. -2.19 g/L /year placebo).
UKMi comment
A commentary notes that one unanswered question is when to initiate α1 antitrypsin augmentation treatment. The study enrolled patients with mild to moderate airflow obstruction. The author suggests that with evidence of clinical efficacy now shown, use of augmentation treatment earlier than at present, before onset of decline in lung function, should be explored. He adds that the challenge remains of a means to augment α1 antitrypsin concentrations in patients with α1 antitrypsin deficiency without the need for intravenous infusions every week, and to do so in a less costly manner (present treatment costs US$ 100,000 per individual per year).
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