1 research outputs found
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 abdominal or facial attacks in patients
with hereditary angioedema.
Methods: This was a randomized, double-blind, placebocontrolled 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 5.0025), 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 5 .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