TY - JOUR T1 - Safety of bi-weekly infusion of A<sub>1</sub>-PI augmentation therapy in RAPID JF - European Respiratory Journal JO - Eur Respir J DO - 10.1183/13993003.congress-2015.PA999 VL - 46 IS - suppl 59 SP - PA999 AU - Niels Seersholm AU - Robert Sandhaus AU - Kenneth R. Chapman AU - Jonathan Burdon AU - Eeva Piitulainen AU - James Stocks AU - Michael Tortorici AU - Tanja Rosenberg AU - Oliver Vit AU - Martin Bexon AU - Jonathan Edelman AU - N. Gerard McElvaney Y1 - 2015/09/01 UR - http://erj.ersjournals.com/content/46/suppl_59/PA999.abstract N2 - BackgroundThe RAPID trial showed that infusion of alpha1-proteinase inhibitor (A1-PI; 60mg/kg/week) slows lung density decline in A1-PI-deficient patients (Chapman et al., Lancet, In Press).AimTo assess the safety of bi-weekly infusion of 120mg/kg A1-PI (Zemaira®, CSL Behring) given to cover 2-week periods when patients were unable to receive weekly dosing.MethodsSubjects were monitored for 7 days post biweekly 120mg/kg A1-PI infusion and the profile of adverse events (AEs) compared with placebo.ResultsIn total, 75 of 93 subjects (80.6%) received 333 bi-weekly infusion of 120mg/kg A1-PI (3.9% of 8,538 total infusions), and 70 of 87 subjects (80.5%) received 374 bi-weekly placebo infusions (5.2% of 7,192 total infusions). Predicted Cmax was 45.1µM after 120mg/kg and 28.5µM after 60mg/kg. The number of AEs after 120mg/kg A1-PI was comparable to placebo. Three serious AEs (bladder cancer; transurethral prostatectomy; chest pain) occurred with 120mg/kg A1-PI and none with placebo. None were considered related to treatment. A1-PI (N=75; I=333)Placebo (N=70; I=374)En (%)IAEREn (%)IAERNo. of AEs5926(34.7)0.1775731 (44.3)0.152No. of AEs within 24h129 (12.0)0.0361412 (17.1)0.037No. of related AEs64 (5.3)0.01833 (4.3)0.008N=subjects with biweekly infusions; I=biweekly infusions; E=events; n=subjects with events; IAER=infusion adjusted event rate (E/I)Safety of bi-weekly infusions in RAPIDConclusionsBi-weekly 120mg/kg A1-PI infusion was well tolerated, with an AE profile comparable to placebo. This short-term option enhances convenience for patients in the future. ER -