RAPID
Randomized, Placebo-Controlled Trial of A1-PI Augmentation in Severe AATD
Patient / Population Intervention / Exposition Comparaison Critère de jugement
In 180 patients with severe (PiZZ) α-1-antitrypsin deficiency and emphysema, intravenous A1-PI augmentation 60 mg/kg weekly slowed annual loss of CT lung density compared with placebo.
N
180
Schéma
Multicentre RCT, 24 months
Critère
Annual CT lung-density decline at total lung capacity
Pertinence
3
Résultat-1.45 vs -2.19 g/L/year (difference 0.74; p=0.03); FEV1 changes similar.
Chapman KR, et al. Lancet. 2015;386(9991):360-368. 10.1016/S0140-6736(15)60860-1
Discussion et critique
First randomised proof that IV augmentation slows emphysema progression on densitometry; FEV1 endpoint remains unproven.