Recombinant Human C1 Esterase Inhibitor Treatment for Hereditary Angioedema Attacks in Children.

Authors:
Avner Reshef
Avner Reshef
University of Calgary
Canada
Vesna Grivcheva-Panovska
Vesna Grivcheva-Panovska
Penn State University College of Medicine
United States
Aharon Kessel
Aharon Kessel
Division of Allergy and Clinical Immunology
Israel
Shmuel Kivity
Shmuel Kivity
Tel Aviv University
Israel
Maria Klimaszewska-Rembiasz
Maria Klimaszewska-Rembiasz
Jagiellonian University Medical College
Poland
Dumitru Moldovan
Dumitru Moldovan
University of Calgary
Canada
Henriette Farkas
Henriette Farkas
Semmelweis University
Hungary

Pediatr Allergy Immunol 2019 Apr 16. Epub 2019 Apr 16.

Charité Universitätsmedizin Berlin, Berlin, Germany.

Background: Attacks of hereditary angioedema (HAE) due to C1 esterase inhibitor deficiency (C1-INH-HAE) usually begin during childhood or adolescence. However, limited data are available regarding indications and modalities of treatment of children. This study evaluated recombinant human C1-INH (rhC1-INH) for HAE attacks in children.

Methods: This open-label, phase 2 study included children aged 2-13 years with C1-INH-HAE. Eligible HAE attacks were treated intravenously with rhC1-INH 50 IU/kg body weight (maximum, 4200 IU). The primary end point was time to beginning of symptom relief (TOSR; ≥20 mm decrease from baseline in visual analog scale [VAS] score, persisting for two consecutive assessments); secondary end point was time to minimal symptoms (TTMS; <20 mm VAS score for all anatomical locations).

Results: Twenty children (aged 5-14 years; 73 HAE attacks) were treated with rhC1-INH. Seventy (95.9%) of the attacks were treated with a single dose of rhC1-INH. Seven (35.0%) children were treated for four or more attacks. Overall median TOSR was 60.0 minutes (95% confidence interval [CI], 60.0-65.0); data were consistent across attacks. Median TTMS was 122.5 minutes (95% CI, 120.0-126.0); data were consistent across attacks. No children withdrew from the study due to adverse events. No treatment-related serious adverse events or hypersensitivity reactions were reported; no neutralizing antibodies were detected.

Conclusions: rhC1-INH was efficacious, safe, and well tolerated in children. Data support use of same dosing regimen for HAE attacks in children (50 IU/kg; up to 4200 IU, followed by an additional dose, if needed) as currently recommended for adolescents and adults. This article is protected by copyright. All rights reserved.

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Source
http://dx.doi.org/10.1111/pai.13065DOI Listing
April 2019
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