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Human plasma-derived nanofiltered C1 esterase inhibitor (C1 INH-nf) is used to treat acute angioedema attacks in patients with hereditary angioedema (HAE), but data regarding the use in children are sparse.
METHODS: Patients 2 to <12 years of age, body weight ≥10 kg, with a diagnosis of HAE type I or II were recruited for a multicenter open-label trial. Patients were recruited into 2 weight categories (10-25 kg, >25 kg). Each weight category included 2 dosing levels: C1 INH-nf (500 units [U], 1000 U) and C1 INH-nf (1000 U, 1500 U) respectively. Patients experiencing an angioedema attack were given a single intravenous dose. Primary efficacy endpoint was the onset of unequivocal relief of the defining symptom within 4 hours following initiation of C1 INH-nf treatment.
RESULTS: Nine children were treated: 3 (10-25 kg) received 500 U; 3 (>25 kg) received 1000 U; and 3 (>25 kg) received 1500 U. The lower weight/higher dose category (10-25 kg, 1000 U) was not successfully enrolled. All patients completed the study. Most angioedema attacks (n = 5) were abdominal. All patients met the primary endpoint; median time to unequivocal symptom relief was 0.5 (range: 0.25-2.5) hours. Doses of C1 INH-nf ranged from 20.8-51.9 U/kg.
CONCLUSIONS: Treatment of a single angioedema attack with C1 INH-nf doses of 500 U (in patients 10-25 kg), 1000 U, and 1500 U (in patients >25 kg) were well tolerated. Doses of C1 INH-nf <1000 U may be appropriate in some pediatric patients. This article is protected by copyright. All rights reserved.
Authors: Lumry W, Soteres D, Gower R, Jacobson KW, Li HH, Chen H, Schranz J
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