3 research outputs found
Assessment of inhibitory antibodies in patients with hereditary angioedema treated with plasma-derived C1 inhibitor
Background Limited data are available regarding C1 inhibitor (C1-INH) administration and anti–C1-INH antibodies.
Objective To assess the incidence of antibody formation during treatment with pasteurized, nanofiltered plasma-derived C1-INH (pnfC1-INH) in patients with hereditary angioedema with C1-INH deficiency (C1-INH-HAE) and the comparative efficacy of pnfC1-INH in patients with and without antibodies.
Methods In this multicenter, open-label study, patients with C1-INH-HAE (≥12 years of age) were given 20 IU/kg of pnfC1-INH per HAE attack that required treatment and followed up for 9 months. Blood samples were taken at baseline (day of first attack) and months 3, 6, and 9 and analyzed for inhibitory anti–C1-INH antibody (iC1-INH-Ab) and noninhibitory anti–C1-INH antibodies (niC1-INH-Abs).
Results The study included 46 patients (69.6% female; mean age, 38.9 years; all white) who received 221 on-site pnfC1-INH infusions; most patients received 6 or fewer infusions. No patient tested positive (titer ≥1:50) for iC1-INH-Ab at any time during the study. Thirteen patients (28.2%) had detectable niC1-INH-Abs in 1 or more samples. Nine patients (19.6%) had detectable niC1-INH-Abs at baseline; 3 of these had no detectable antibodies after baseline. Of 10 patients (21.7%) with 1 or more detectable result for niC1-INH-Abs after baseline, 6 had detectable niC1-INH-Abs at baseline. Mean times to symptom relief onset and complete symptom resolution per patient were similar for those with or without anti–niC1-INH-Abs.
Conclusion Administration of pnfC1-INH was not associated with iC1-INH-Ab formation in this population. Noninhibitory antibodies were detected in some patients but fluctuated during the study independently of pnfC1-INH administration and appeared to have no effect on pnfC1-INH efficacy
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