Safety and efficacy of C1 esterase inhibitor for acute attacks in children with hereditary angioedema.

Abstract

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.

• Authors: William Lumry, Daniel Soteres, Richard Gower, Kraig W Jacobson, H Henry Li, Hongzi Chen, Jennifer Schranz
• Journal: Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology (Pediatr Allergy Immunol) Vol. 26 Issue 7 Pg. 674-80 (Nov 2015) ISSN: 1399-3038 [Electronic] England
• PMID: 26171584 (Publication Type: Clinical Trial, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
• Copyright: © 2015 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.

Chemical References

• Complement C1 Inhibitor Protein
• Complement Inactivating Agents

Topics

• Acute Disease
• Angioedemas, Hereditary (drug therapy)
• Child
• Child, Preschool
• Complement C1 Inhibitor Protein (therapeutic use)
• Complement Inactivating Agents (therapeutic use)
• Disease Progression
• Drug Administration Schedule
• Female
• Humans
• Male
• Treatment Outcome