Timothy Craig, Bruce Zuraw, Hilary Longhurst, Marco Cicardi, Konrad Bork, Clive Grattan, Constance Katelaris, Gordon Sussman, Paul K Keith, William Yang, Jacques Hébert, Jana Hanzlikova, Petra Staubach-Renz, Inmaculada Martinez-Saguer, Markus Magerl, Emel Aygören-Pürsün, Henriette Farkas, Avner Reshef, Shmuel Kivity, Sergio Neri, Ioana Crisan, Teresa Caballero, Maria L Baeza, Maria Dolores Hernandez, Henry Li, William Lumry, Jonathan A Bernstein, Iftikar Hussain, John Anderson, Lawrence B Schwartz, Joshua Jacobs, Michael Manning, Donald Levy, Marc Riedl, Sandra Christiansen, Henrike Feuersenger, Ingo Pragst, Sarah Mycroft, Dipti Pawaskar, Iris Jacobs
BACKGROUND: For the prevention of attacks of hereditary angioedema (HAE), the efficacy and safety of subcutaneous human C1-esterase inhibitor (C1-INH[SC]; HAEGARDA® , CSL Behring) was established in the 16-week COMPACT trial. OBJECTIVE: To assess the long-term safety, occurrence of angioedema attacks, and use of rescue medication with C1-INH(SC). METHODS: Open-label, randomised, parallel-arm extension of COMPACT across 11 countries. Patients with frequent angioedema attacks, either study treatment-naïve or who had completed the COMPACT trial, were randomly assigned (1:1) to 40 IU/kg or 60 IU/kg C1-INH(SC) twice per week, with conditional up-titration to optimise prophylaxis...
February 14, 2019: Journal of Allergy and Clinical Immunology in Practice