TY  - JOUR
A1  - Craig, Timothy
A1  - Zuraw, Bruce
A1  - Longhurst, Hilary
A1  - Cicardi, Marco
A1  - Bork, Konrad
A1  - Grattan, Clive
A1  - Katelaris, Constance
A1  - Sussman, Gordon
A1  - Keith, Paul K.
A1  - Yang, William
A1  - Hébert, Jacques
A1  - Hanzlikova, Jana
A1  - Staubach-Renz, Petra
A1  - Martinez Saguer, Inmaculada
A1  - Magerl, Markus
A1  - Aygören-Pürsün, Emel
A1  - Farkas, Henriette
A1  - Reshef, Avner
A1  - Kivity, Shmuel
A1  - Neri, Sergio
A1  - Crișan, Ioana
A1  - Caballero, Teresa
A1  - Baeza, María Luisa
A1  - Hernandez, Maria Dolores
A1  - Li, Henry
A1  - Lumry, William
A1  - Bernstein, Jonathan A.
A1  - Hussain, Iftikar
A1  - Anderson, John
A1  - Schwartz, Lawrence B.
A1  - Jacobs, Joshua
A1  - Manning, Michael
A1  - Levy, Donald
A1  - Riedl, Marc A.
A1  - Christiansen, Sandra
A1  - Feuersenger, Henrike
A1  - Pragst, Ingo
A1  - Mycroft, Sarah
A1  - Pawaskar, Dipti
A1  - Jacobs, Iris
T1  - Long-term outcomes with subcutaneous C1-inhibitor replacement therapy for prevention of hereditary angioedema attacks
T2  - The journal of allergy and clinical immunology. In practice
N2  - 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. ClinicalTrials.gov registration: NCT02316353.
Results: 126 patients with a monthly attack rate of 4.3 in 3 months prior to entry in the COMPACT program were enrolled and treated for a mean of 1·5 years; 44 patients (34·9%) had >2 years exposure. Median steady-state C1-INH functional activity increased to a maximum of 73.0% with 60 IU/kg. Incidence of adverse events was low and similar in both dose groups (11·3 and 8·5 events per patient-year for 40 IU/kg and 60 IU/kg, respectively). For 40 IU/kg and 60 IU/kg, median annualised attack rates were 1·3 and 1·0, respectively and median rescue medication use was 0.2 and 0.0 times per year, respectively. Of 23 patients receiving 60 IU/kg for >2 years, 19 (83%) were attack-free during months 25–30 of treatment.
Conclusion: In patients with frequent HAE attacks, long-term replacement therapy with C1-INH(SC) is safe and exhibits a substantial and sustained prophylactic effect, with the vast majority of patients becoming free from debilitating disease symptoms.
KW  - C1-esterase inhibitor protein
KW  - HAEGARDA
KW  - Hereditary angioedema
KW  - Subcutaneous
KW  - Long-term
KW  - Prophylaxis
KW  - Safety
Y1  - 2019
UR  - http://publikationen.ub.uni-frankfurt.de/frontdoor/index/index/docId/49199
UR  - https://nbn-resolving.org/urn:nbn:de:hebis:30:3-491997
SN  - 2213-2201
SN  - 2213-2198
N1  - © 2019 The Authors. Published by Elsevier Inc. on behalf of the American Academy of Allergy, Asthma & Immunology. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/). (J Allergy Clin Immunol Pract 2019;7:1793-802)
VL  - 7
IS  - 6
SP  - 1793
EP  - 1802.e2
PB  - Elsevier
CY  - Amsterdam [u. a.]
ER  -