Prevention of Attacks of Hereditary Angioedema (HAE) with Subcutaneous C1-Esterase Inhibitor (C1-INH [SC]) by Anatomical Location: Results From the Phase III COMPACT Study (NCT01912456)

Rationale Subcutaneous C1-INH (HAEGARDA®, CSL Behring, Marburg) at 60 IU/kg was recently approved by the US FDA as routine prophylaxis to prevent hereditary angioedema (HAE) attacks in adolescents and adults, based on the COMPACT study. Methods In the COMPACT study, 45 patients self-administered C1-INH (SC) 60 IU/kg body weight or placebo twice weekly in a double-blind, crossover manner over two 16-week treatment periods.