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

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.

• Authors: Timothy J Craig, Robyn J Levy, Richard L Wasserman, Againdra K Bewtra, David Hurewitz, Krystyna Obtułowicz, Avner Reshef, Bruce Ritchie, Dumitru Moldovan, Todor Shirov, Vesna Grivcheva-Panovska, Peter C Kiessling, Heinz-Otto Keinecke, Jonathan A Bernstein
• Journal: The Journal of allergy and clinical immunology (J Allergy Clin Immunol) Vol. 124 Issue 4 Pg. 801-8 (Oct 2009) ISSN: 1097-6825 [Electronic] United States
• PMID: 19767078 (Publication Type: Clinical Trial, Phase II, Clinical Trial, Phase III, Journal Article, Multicenter Study, Randomized Controlled Trial)

Chemical References

• Complement C1 Inhibitor Protein
• Complement Inactivating Agents

Topics

• Acute Disease
• Adolescent
• Adult
• Aged
• Angioedemas, Hereditary (drug therapy)
• Child
• Complement C1 Inhibitor Protein (administration & dosage, adverse effects, therapeutic use)
• Complement Inactivating Agents (administration & dosage, adverse effects, therapeutic use)
• Double-Blind Method
• Female
• Humans
• Injections, Intravenous
• Kaplan-Meier Estimate
• Male
• Middle Aged
• Young Adult