Efficacy of human C1 esterase inhibitor concentrate compared with placebo in acute hereditary angioedema attacks

Background Hereditary angioedema caused by C1 esterase inhibitor deficiency is a rare disorder. Objective To compare the efficacy of pasteurized C1 esterase inhibitor concentrate (Berinert, CSL Behring) at intravenous doses of 10 or 20 U/kg body weight with placebo in the treatment of single, acute...

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Published in:Journal of allergy and clinical immunology 2009-10, Vol.124 (4), p.801-808
Main Authors: Craig, Timothy J., MD, Levy, Robyn J., MD, Wasserman, Richard L., PhD, MD, Bewtra, Againdra K., MD, Hurewitz, David, MD, Obtułowicz, Krystyna, MD, Reshef, Avner, MD, Ritchie, Bruce, MD, Moldovan, Dumitru, MD, Shirov, Todor, MD, Grivcheva-Panovska, Vesna, MD, Kiessling, Peter C., PhD, Keinecke, Heinz-Otto, MS, Bernstein, Jonathan A., MD
Format: Article
Language:English
Subjects:
Abdomen
Acute Disease
Adolescent
Adult
Aged
Allergic diseases
Allergy and Immunology
Angioedema
Angioedemas, Hereditary - drug therapy
Biological and medical sciences
Body weight
C1 inhibitor
C1 inhibitor deficiency
C1-INH
Child
Clinical trials
Complement C1 Inhibitor Protein - administration & dosage
Complement C1 Inhibitor Protein - adverse effects
Complement C1 Inhibitor Protein - therapeutic use
Complement Inactivating Agents - administration & dosage
Complement Inactivating Agents - adverse effects
Complement Inactivating Agents - therapeutic use
Double-Blind Method
Drug dosages
Drug therapy
esterase
Female
Fundamental and applied biological sciences. Psychology
Fundamental immunology
HAE
hereditary angioedema
Human immunodeficiency virus
Humans
Immunopathology
Injections, Intravenous
Intravenous administration
Kaplan-Meier Estimate
Male
Medical sciences
Middle Aged
Pain
Parvovirus B19
Plasma
Sarcoidosis. Granulomatous diseases of unproved etiology. Connective tissue diseases. Elastic tissue diseases. Vasculitis
Seroconversion
Skin allergic diseases. Stinging insect allergies
Vomiting
Young Adult
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Summary:Background Hereditary angioedema caused by C1 esterase inhibitor deficiency is a rare disorder. Objective To compare the efficacy of pasteurized C1 esterase inhibitor concentrate (Berinert, CSL Behring) at intravenous doses of 10 or 20 U/kg body weight with placebo in the treatment of single, acute abdominal or facial attacks in patients with hereditary angioedema. Methods This was a randomized, double-blind, placebo-controlled study in 125 patients with type I or II hereditary angioedema. The primary outcome was time from start of treatment to onset of symptom relief. Secondary outcomes were time to complete resolution, proportion of patients with worsened intensity of angioedema symptoms between 2 and 4hours after treatment, and number of vomiting episodes within 4 hours. Results Median time to onset of relief was significantly shorter with C1 esterase inhibitor concentrate at a dose of 20 U/kg than with placebo (0.5 vs 1.5 hours; P = .0025), whereas with 10 U/kg, the time to onset of relief was only slightly shorter than with placebo (1.2 vs 1.5 hours; P = .2731). Compared with placebo, the reduction in time to onset of relief was greatest for severe attacks (0.5 vs 13.5 hours). The secondary outcomes consistently supported the efficacy of the 20 U/kg dose. C1 esterase inhibitor concentrate was safe and well tolerated. No seroconversions were observed for HIV, hepatitis virus, or human B19 virus. Conclusion C1 esterase inhibitor concentrate given intravenously at a dose of 20 U/kg is an effective and safe treatment for acute abdominal and facial attacks in patients with hereditary angioedema, with a rapid onset of relief.
ISSN:0091-6749
1097-6825
DOI:10.1016/j.jaci.2009.07.017