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

A. Reshef, V. Grivcheva-Panovska, A. Kessel, S. Kivity, M. Klimaszewska-Rembiasz, D. Moldovan, H. Farkas, V. Gutova, S. Fritz, A. Relan, B. Giannetti, M. Magerl,

. 2019 ; 30 (5) : 562-568. [pub] 20190529

Language English Country Great Britain

Document type Clinical Trial, Phase II, Journal Article, Research Support, Non-U.S. Gov't

Persistent link   https://www.medvik.cz/link/bmc20023798

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.

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$a Reshef, Avner $u Barzilai University Hospital, Ashkelon, Israel.
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$a 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.
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$a Grivcheva-Panovska, Vesna $u Medical University Skopje, Skopje, North Macedonia.
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$a Kessel, Aharon $u Technion Faculty of Medicine, Bnai Zion Medical Center, Haifa, Israel.
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$a Kivity, Shmuel $u The Tel Aviv Medical Center, Tel Aviv, Israel.
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$a Klimaszewska-Rembiasz, Maria $u Pediatric Hospital, Krakow, Poland.
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$a Moldovan, Dumitru $u MediQuest Clinical Research, Sangeorgiu de Mures, Romania.
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$a Farkas, Henriette $u Semmelweis University, Budapest, Hungary.
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$a Fritz, Stephen $u Portland Clinical Research, Portland, Oregon, USA.
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$a Relan, Anurag $u Pharming Healthcare Inc., Bridgewater, New Jersey, USA.
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$a Giannetti, Bruno $u Pharming Group NV, Leiden, The Netherlands.
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$a Magerl, Markus $u Charité Universitätsmedizin Berlin, Berlin, Germany.
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