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Long-Term Outcomes with Subcutaneous C1-Inhibitor Replacement Therapy for Prevention of Hereditary Angioedema Attacks
T. Craig, B. Zuraw, H. Longhurst, M. Cicardi, K. Bork, C. Grattan, C. Katelaris, G. Sussman, PK. Keith, W. Yang, J. Hébert, J. Hanzlikova, P. Staubach-Renz, I. Martinez-Saguer, M. Magerl, E. Aygören-Pürsün, H. Farkas, A. Reshef, S. Kivity, S....
Language English Country United States
Document type Clinical Trial, Phase III, Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't
- MeSH
- Child MeSH
- Adult MeSH
- Angioedemas, Hereditary prevention & control MeSH
- Complement C1 Inhibitor Protein administration & dosage MeSH
- Injections, Subcutaneous MeSH
- Middle Aged MeSH
- Humans MeSH
- Adolescent MeSH
- Young Adult MeSH
- Aged MeSH
- Treatment Outcome MeSH
- Check Tag
- Child MeSH
- Adult MeSH
- Middle Aged MeSH
- Humans MeSH
- Adolescent MeSH
- Young Adult MeSH
- Male MeSH
- Aged MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Clinical Trial, Phase III MeSH
- Multicenter Study MeSH
- Research Support, Non-U.S. Gov't MeSH
- Randomized Controlled Trial MeSH
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.
AARA Research Center Dallas Texas
Allergy and Asthma Clinical Research Walnut Creek Calif
Allergy Asthma and Immunology Center Tulsa Okla
Barzilai University Medical Center Allergy Immunology and Angioedema Ashkelon Israel
Bernstein Clinical Research Center LLC Cincinnati Ohio
Clinical Research Center of Alabama Alabama Allergy and Asthma Birmingham Ala
Clinical Research Centre Hautklinik und Poliklinik der Universitätsklinik Mainz Mainz Germany
Clinique Specialisée en Allergie de la Capitale Québec QC Canada
CSL Behring LLC King of Prussia Pa
Department of Dermatology and Allergy Charité Universitätsmedizin Berlin Berlin Germany
Department of Dermatology Johannes Gutenberg University Mainz Germany
Department of Internal Medicine Virginia Commonwealth University Richmond Va
Department of Medicine Immunology and Allergy Campbelltown Hospital Campbelltown NSW Australia
Department of Medicine University of California San Diego and San Diego VA Healthcare La Jolla Calif
Dr Donald Levy MD Orange Calif
Fakultni nemocnice Plzen Ustav Imunologie a Alergologie Plzen Czech Republic
Gordon Sussman Clinical Research Inc Toronto ON Canada
Hospital Universitario La Fe Servicio de Alergia Bulevar del Sur Valencia Spain
HZRM Haemophilia Center Rhein Main Moerfelden Walldorf Germany
Immunology Department Barts Health NHS Trust The Royal London Hospital London United Kingdom
Institute for Asthma and Allergy Chevy Chase Md
McMaster University Medical Center Site Hamilton ON Canada
Medical Research of Arizona Scottsdale Ariz
Ottawa Allergy Research Corp Ottawa ON Canada
Servicio de Alergia Hospital La Paz Institute for Health Research U754 Madrid Spain
Spitalul Clinic Municipal Cluj Cluj Napoca Romania
St John's Institute of Dermatology Tower Wing Guy's Hospital London United Kingdom
Tel Aviv Sourasky Medical Center Allergy and Immunology Unit Tel Aviv Israel
References provided by Crossref.org
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- $a Craig, Timothy $u Department of Medicine, Pediatrics and Graduate Studies, Penn State University Hershey Medical Center, Hershey, Pa. Electronic address: tcraig@psu.edu.
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- $a 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.
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