Abstract
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.
| Original language | English |
|---|---|
| Pages (from-to) | 801-808 |
| Number of pages | 8 |
| Journal | Journal of Allergy and Clinical Immunology |
| Volume | 124 |
| Issue number | 4 |
| DOIs | |
| State | Published - Oct 2009 |
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All Science Journal Classification (ASJC) codes
- Immunology and Allergy
- Immunology
- Medicine(all)
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Efficacy of human C1 esterase inhibitor concentrate compared with placebo in acute hereditary angioedema attacks. / Craig, Timothy J.; Levy, Robyn J.; Wasserman, Richard L.; Bewtra, Againdra K.; Hurewitz, David; Obtułowicz, Krystyna; Reshef, Avner; Ritchie, Bruce; Moldovan, Dumitru; Shirov, Todor; Grivcheva-Panovska, Vesna; Kiessling, Peter C.; Keinecke, Heinz Otto; Bernstein, Jonathan A.
In: Journal of Allergy and Clinical Immunology, Vol. 124, No. 4, 10.2009, p. 801-808.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Efficacy of human C1 esterase inhibitor concentrate compared with placebo in acute hereditary angioedema attacks
AU - Craig, Timothy J.
AU - Levy, Robyn J.
AU - Wasserman, Richard L.
AU - Bewtra, Againdra K.
AU - Hurewitz, David
AU - Obtułowicz, Krystyna
AU - Reshef, Avner
AU - Ritchie, Bruce
AU - Moldovan, Dumitru
AU - Shirov, Todor
AU - Grivcheva-Panovska, Vesna
AU - Kiessling, Peter C.
AU - Keinecke, Heinz Otto
AU - Bernstein, Jonathan A.
PY - 2009/10
Y1 - 2009/10
N2 - 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.
AB - 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.
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UR - http://www.scopus.com/inward/citedby.url?scp=70449727672&partnerID=8YFLogxK
U2 - 10.1016/j.jaci.2009.07.017
DO - 10.1016/j.jaci.2009.07.017
M3 - Article
VL - 124
SP - 801
EP - 808
JO - Journal of Allergy and Clinical Immunology
JF - Journal of Allergy and Clinical Immunology
SN - 0091-6749
IS - 4
ER -