Recombinant human C1 esterase inhibitor treatment for hereditary angioedema attacks in children

Avner Reshef, Vesna Grivcheva-Panovska, Aharon Kessel, Shmuel Kivity, Maria Klimaszewska-Rembiasz, Dumitru Moldovan, Henriette Farkas, Vaclava Gutova, Stephen Fritz, Anurag Relan, Bruno Giannetti, Markus Magerl

Research output: Article

3 Citations (Scopus)


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 anatomic 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: Recombinant human C1-INH was efficacious, safe, and well tolerated in children. Data support use of the same dosing regimen for HAE attacks in children (50 IU/kg; up to 4200 IU, followed by an additional dose, if needed) as is currently recommended for adolescents and adults.

Original languageEnglish
Pages (from-to)562-568
Number of pages7
JournalPediatric Allergy and Immunology
Issue number5
Publication statusPublished - aug. 2019

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Immunology and Allergy
  • Immunology

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    Reshef, A., Grivcheva-Panovska, V., Kessel, A., Kivity, S., Klimaszewska-Rembiasz, M., Moldovan, D., Farkas, H., Gutova, V., Fritz, S., Relan, A., Giannetti, B., & Magerl, M. (2019). Recombinant human C1 esterase inhibitor treatment for hereditary angioedema attacks in children. Pediatric Allergy and Immunology, 30(5), 562-568.