C1 Esterase Inhibitor Effective in Hereditary Angioedema

Single 20 U/kg dose safe, complete symptom resolution at any body location in 15.5 hrs

THURSDAY, Nov. 3 (HealthDay News) -- A single dose of 20 U/kg C1 esterase inhibitor (C1-INH) concentrate is safe and effective for treatment of successive hereditary angioedema (HAE) attacks at any body location, according to a study published in the December issue of Allergy.

Timothy J. Craig, D.O., from Penn State University College of Medicine in Hershey, and colleagues investigated the safety and efficacy of long-term treatment with 20 U/kg C1-INH for 1,085 HAE attacks at any body location in 57 patients (aged 10 to 53 years). Efficacy outcomes were analyzed on a per-patient and per-attack basis, and included patient reported time to onset of symptom relief, and time to complete resolution of all symptoms. Safety was assessed through adverse events, vital signs, viral safety, and anti-C1-INH antibodies. The participants were followed up for a median duration of 24 months.

The investigators found that the median time to onset of symptom relief in the per-patient analysis was 0.46 hours, and was similar for all types of attacks (0.39 to 0.48 hours). The median time to complete resolution of symptoms was 15.5 hours. Efficacy outcomes were not clinically affected by relevant demographic factors, type of HAE, intensity of attacks, time to treatment, use of androgens, and presence of anti-C1-INH antibodies. No patient had treatment-related safety concerns or inhibitory anti-C1-INH antibodies.

"C1-INH concentrate at a single dose of 20 U/kg body weight is safe and provides reliable efficacy for the long-term treatment of successive HAE attacks at any body location," the authors write.

Several authors disclosed financial relationships with pharmaceutical and biotherapeutics companies, including CSL Behring, which funded the study.

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