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Recombinant human C1 esterase inhibitor treatment for hereditary angioedema attacks in children.
Pediatr Allergy Immunol 2019; 30(5):562-568PA

Abstract

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.

Authors+Show Affiliations

Barzilai University Hospital, Ashkelon, Israel.Medical University Skopje, Skopje, North Macedonia.Technion Faculty of Medicine, Bnai Zion Medical Center, Haifa, Israel.The Tel Aviv Medical Center, Tel Aviv, Israel.Pediatric Hospital, Krakow, Poland.MediQuest Clinical Research, Sangeorgiu de Mures, Romania.Semmelweis University, Budapest, Hungary.Institute of Immunology and Allergology, Pilsen, Czech Republic.Portland Clinical Research, Portland, Oregon, USA.Pharming Healthcare Inc., Bridgewater, New Jersey, USA.Pharming Group NV, Leiden, The Netherlands.Charité Universitätsmedizin Berlin, Berlin, Germany.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

30993784

Citation

Reshef, Avner, et al. "Recombinant Human C1 Esterase Inhibitor Treatment for Hereditary Angioedema Attacks in Children." Pediatric Allergy and Immunology : Official Publication of the European Society of Pediatric Allergy and Immunology, vol. 30, no. 5, 2019, pp. 562-568.
Reshef A, Grivcheva-Panovska V, Kessel A, et al. Recombinant human C1 esterase inhibitor treatment for hereditary angioedema attacks in children. Pediatr Allergy Immunol. 2019;30(5):562-568.
Reshef, A., Grivcheva-Panovska, V., Kessel, A., Kivity, S., Klimaszewska-Rembiasz, M., Moldovan, D., ... Magerl, M. (2019). Recombinant human C1 esterase inhibitor treatment for hereditary angioedema attacks in children. Pediatric Allergy and Immunology : Official Publication of the European Society of Pediatric Allergy and Immunology, 30(5), pp. 562-568. doi:10.1111/pai.13065.
Reshef A, et al. Recombinant Human C1 Esterase Inhibitor Treatment for Hereditary Angioedema Attacks in Children. Pediatr Allergy Immunol. 2019;30(5):562-568. PubMed PMID: 30993784.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Recombinant human C1 esterase inhibitor treatment for hereditary angioedema attacks in children. AU - Reshef,Avner, AU - Grivcheva-Panovska,Vesna, AU - Kessel,Aharon, AU - Kivity,Shmuel, AU - Klimaszewska-Rembiasz,Maria, AU - Moldovan,Dumitru, AU - Farkas,Henriette, AU - Gutova,Vaclava, AU - Fritz,Stephen, AU - Relan,Anurag, AU - Giannetti,Bruno, AU - Magerl,Markus, Y1 - 2019/05/29/ PY - 2018/12/04/received PY - 2019/04/02/revised PY - 2019/04/04/accepted PY - 2019/4/18/pubmed PY - 2019/4/18/medline PY - 2019/4/18/entrez KW - angioedema KW - child KW - complement C1 inactivator proteins KW - complement C1s KW - hereditary KW - hereditary angioedema type I and type II KW - recombinant proteins SP - 562 EP - 568 JF - Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology JO - Pediatr Allergy Immunol VL - 30 IS - 5 N2 - 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. SN - 1399-3038 UR - https://www.unboundmedicine.com/medline/citation/30993784/Recombinant_human_C1_esterase_inhibitor_treatment_for_hereditary_angioedema_attacks_in_children L2 - https://doi.org/10.1111/pai.13065 DB - PRIME DP - Unbound Medicine ER -