Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registry
Background: Hereditary angioedema (HAE) due to C1 esterase inhibitor deficiency (C1-INH-HAE) is characterized by recurrent swelling attacks. A European treatment registry was established to review the adverse event profile and efficacy of recombinant human C1 esterase inhibitor (rhC1-INH) for HAE at...
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2021-04-01
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doaj-6f5b107c5bc54f7aac383897a79cb3142021-05-08T04:22:20ZengElsevierWorld Allergy Organization Journal1939-45512021-04-01144100535Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registryAnna Valerieva0Maria T. Staevska1Vesna Grivcheva-Panovska2Milos Jesenak3Kinga Viktória Kőhalmi4Katarina Hrubiskova5Andrea Zanichelli6Luca Bellizzi7Anurag Relan8Roman Hakl9Henriette Farkas10Department of Allergology, Medical University of Sofia, Sofia, Bulgaria; Corresponding author.Department of Allergology, Medical University of Sofia, Sofia, BulgariaPHI University Clinic of Dermatology, School of Medicine, University Saints Cyril and Methodius, Skopje, MacedoniaUniversity Hospital in Martin, Comenius University in Bratislava, Jessenius Faculty of Medicine, Martin, SlovakiaHungarian Angioedema Center of Excellence and Reference, Department of Internal Medicine and Haematology, Semmelweis University, Budapest, Hungary; Hospital of Hospitaller Brothers of St. John of God, Budapest, Hungary; Department of Rheumatology and Clinical Immunology, Semmelweis University, Budapest, HungaryComenius University in Bratislava and University Hospital, Bratislava, SlovakiaASST Fatebenefratelli Sacco, Ospedale Luigi Sacco-University of Milan, Milan, ItalyPharming Technologies BV, Leiden, the NetherlandsPharming Healthcare Inc., Warren, NJ, USASt. Anne's University Hospital, Masaryk University, Brno, CzechiaHungarian Angioedema Center of Excellence and Reference, Department of Internal Medicine and Haematology, Semmelweis University, Budapest, HungaryBackground: Hereditary angioedema (HAE) due to C1 esterase inhibitor deficiency (C1-INH-HAE) is characterized by recurrent swelling attacks. A European treatment registry was established to review the adverse event profile and efficacy of recombinant human C1 esterase inhibitor (rhC1-INH) for HAE attacks. Methods: Individuals with C1-INH-HAE were enrolled following a decision to treat with rhC1-INH and provision of written informed consent. Medical history and baseline HAE information were collected at screening. Healthcare providers entered data on HAE attacks, response to treatment, and adverse events using a web-based questionnaire. Results: From July 1, 2011, through December 1, 2019, 71 patients with C1-INH-HAE (30 male/41 female; mean age, 47.3 years; age range, 19–78 years) in 9 countries reported 2356 attacks and were treated with rhC1-INH. Before registry entry, patients, including 20 (28.2%) who were on maintenance therapy/prophylaxis at registry enrollment, experienced a mean of 25 HAE attacks per year (median, 16 [range, 0–185]). Most treated HAE attacks were abdominal (46.1%), followed by peripheral (38.3%), oro-facial-pharyngeal (14.8%), urogenital (3.2%), and laryngeal (2.6%). The mean rhC1-INH dose was 3307 U (43.3 U/kg). Patients reported symptom improvement within 4 h for 97.8% of attacks (2305/2356) with rhC1-INH; most attacks (99.8%; 2351/2356) required only 1 dose. Five attacks were treated with a second dose (total rhC1-INH dose administered for attack, 4200 U). No hypersensitivity, thrombotic/thromboembolic events, or drug-related serious adverse events were reported. Conclusion: The rhC1-INH treatment registry provided real-world data on the treatment of 2356 HAE attacks that were consistent with clinical trial data of rhC1-INH in patients with C1-INH-HAE.http://www.sciencedirect.com/science/article/pii/S1939455121000296AngioedemaHereditaryComplement C1 inhibitor proteinRecombinant human C1 esterase inhibitorRegistryRuconest |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Anna Valerieva Maria T. Staevska Vesna Grivcheva-Panovska Milos Jesenak Kinga Viktória Kőhalmi Katarina Hrubiskova Andrea Zanichelli Luca Bellizzi Anurag Relan Roman Hakl Henriette Farkas |
spellingShingle |
Anna Valerieva Maria T. Staevska Vesna Grivcheva-Panovska Milos Jesenak Kinga Viktória Kőhalmi Katarina Hrubiskova Andrea Zanichelli Luca Bellizzi Anurag Relan Roman Hakl Henriette Farkas Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registry World Allergy Organization Journal Angioedema Hereditary Complement C1 inhibitor protein Recombinant human C1 esterase inhibitor Registry Ruconest |
author_facet |
Anna Valerieva Maria T. Staevska Vesna Grivcheva-Panovska Milos Jesenak Kinga Viktória Kőhalmi Katarina Hrubiskova Andrea Zanichelli Luca Bellizzi Anurag Relan Roman Hakl Henriette Farkas |
author_sort |
Anna Valerieva |
title |
Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registry |
title_short |
Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registry |
title_full |
Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registry |
title_fullStr |
Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registry |
title_full_unstemmed |
Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registry |
title_sort |
recombinant human c1 esterase inhibitor for hereditary angioedema attacks: a european registry |
publisher |
Elsevier |
series |
World Allergy Organization Journal |
issn |
1939-4551 |
publishDate |
2021-04-01 |
description |
Background: Hereditary angioedema (HAE) due to C1 esterase inhibitor deficiency (C1-INH-HAE) is characterized by recurrent swelling attacks. A European treatment registry was established to review the adverse event profile and efficacy of recombinant human C1 esterase inhibitor (rhC1-INH) for HAE attacks. Methods: Individuals with C1-INH-HAE were enrolled following a decision to treat with rhC1-INH and provision of written informed consent. Medical history and baseline HAE information were collected at screening. Healthcare providers entered data on HAE attacks, response to treatment, and adverse events using a web-based questionnaire. Results: From July 1, 2011, through December 1, 2019, 71 patients with C1-INH-HAE (30 male/41 female; mean age, 47.3 years; age range, 19–78 years) in 9 countries reported 2356 attacks and were treated with rhC1-INH. Before registry entry, patients, including 20 (28.2%) who were on maintenance therapy/prophylaxis at registry enrollment, experienced a mean of 25 HAE attacks per year (median, 16 [range, 0–185]). Most treated HAE attacks were abdominal (46.1%), followed by peripheral (38.3%), oro-facial-pharyngeal (14.8%), urogenital (3.2%), and laryngeal (2.6%). The mean rhC1-INH dose was 3307 U (43.3 U/kg). Patients reported symptom improvement within 4 h for 97.8% of attacks (2305/2356) with rhC1-INH; most attacks (99.8%; 2351/2356) required only 1 dose. Five attacks were treated with a second dose (total rhC1-INH dose administered for attack, 4200 U). No hypersensitivity, thrombotic/thromboembolic events, or drug-related serious adverse events were reported. Conclusion: The rhC1-INH treatment registry provided real-world data on the treatment of 2356 HAE attacks that were consistent with clinical trial data of rhC1-INH in patients with C1-INH-HAE. |
topic |
Angioedema Hereditary Complement C1 inhibitor protein Recombinant human C1 esterase inhibitor Registry Ruconest |
url |
http://www.sciencedirect.com/science/article/pii/S1939455121000296 |
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