Craig, T;
Zuraw, B;
Longhurst, H;
Cicardi, M;
Bork, K;
Grattan, C;
Katelaris, C;
... Christiansen, SC; + view all
(2019)
Long-Term Outcomes with Subcutaneous C1-Inhibitor Replacement Therapy for Prevention of Hereditary Angioedema Attacks.
The Journal of Allergy and Clinical Immunology: In Practice
, 7
(6)
1793-1802.e2.
10.1016/j.jaip.2019.01.054.
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Abstract
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 Clinical Study for Optimal Management of Preventing Angioedema with Low-Volume Subcutaneous C1-Inhibitor Replacement Therapy (COMPACT). Objective To assess the long-term safety, occurrence of angioedema attacks, and use of rescue medication with C1-INH(SC). Methods Open-label, randomized, parallel-arm extension of COMPACT across 11 countries. Patients with frequent angioedema attacks, either study treatment-naive or who had completed COMPACT, were randomly assigned (1:1) to 40 IU/kg or 60 IU/kg C1-INH(SC) twice per week, with conditional uptitration to optimize prophylaxis (ClinicalTrials.gov registration no. NCT02316353). Results A total of 126 patients with a monthly attack rate of 4.3 in 3 months before entry in COMPACT were enrolled and treated for a mean of 1.5 years; 44 patients (34.9%) had more than 2 years of exposure. Mean steady-state C1-INH functional activity increased to 66.6% 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 annualized 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 more than 2 years, 19 (83%) were attack-free during months 25 to 30 of treatment. Conclusions 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.
| Type: | Article |
|---|---|
| Title: | Long-Term Outcomes with Subcutaneous C1-Inhibitor Replacement Therapy for Prevention of Hereditary Angioedema Attacks |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1016/j.jaip.2019.01.054 |
| Publisher version: | https://doi.org/10.1016/j.jaip.2019.01.054 |
| Language: | English |
| Additional information: | This is an open access article under the CC-BY NC- ND license (http://creativecommons.org/licenses/by-nc-nd/ 4.0/) |
| Keywords: | Science & Technology, Life Sciences & Biomedicine, Allergy, Immunology, C1-esterase inhibitor, HAEGARDA, Hereditary angioedema, Subcutaneous, Long-term, Prophylaxis, Safety, C1 INHIBITOR DEFICIENCY, PATHOPHYSIOLOGY, PROPHYLAXIS, MANAGEMENT, DIAGNOSIS, CONSENSUS |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10089106 |
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