Premium
Safety and efficacy of C1 esterase inhibitor for acute attacks in children with hereditary angioedema
Author(s) -
Lumry William,
Soteres Daniel,
Gower Richard,
Jacobson Kraig W.,
Li H. Henry,
Chen Hongzi,
Schranz Jennifer
Publication year - 2015
Publication title -
pediatric allergy and immunology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.269
H-Index - 89
eISSN - 1399-3038
pISSN - 0905-6157
DOI - 10.1111/pai.12444
Subject(s) - hereditary angioedema , medicine , angioedema , dosing , c1 inhibitor , gastroenterology , clinical endpoint , clinical trial , dermatology
Background 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 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 end‐point was the onset of unequivocal relief of the defining symptom within 4 h 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 end‐point; median time to unequivocal symptom relief was 0.5 (range: 0.25–2.5) h. Doses of C1 INH ‐nf ranged from 20.8 to 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.