Quantitative disease progression model of alpha-1 proteinase inhibitor therapy on computed tomography lung density in patients with alpha-1 antitrypsin deficiency

摘要

AimsEarly-onset emphysema attributed to -1 antitrypsin deficiency (AATD) is frequently overlooked and undertreated. RAPID-RCT/RAPID-OLE, the largest clinical trials of purified human -1 proteinase inhibitor (A(1)-PI; 60mgkg(-1)week(-1)) therapy completed to date, demonstrated for the first time that A(1)-PI is clinically effective in slowing lung tissue loss in AATD. A posthoc pharmacometric analysis was undertaken to further explore dose, exposure and response.