Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registry.

Autor:	Valerieva A; Department of Allergology, Medical University of Sofia, Sofia, Bulgaria., Staevska MT; Department of Allergology, Medical University of Sofia, Sofia, Bulgaria., Grivcheva-Panovska V; PHI University Clinic of Dermatology, School of Medicine, University Saints Cyril and Methodius, Skopje, Macedonia., Jesenak M; University Hospital in Martin, Comenius University in Bratislava, Jessenius Faculty of Medicine, Martin, Slovakia., Kőhalmi KV; Hungarian 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, Hungary., Hrubiskova K; Comenius University in Bratislava and University Hospital, Bratislava, Slovakia., Zanichelli A; ASST Fatebenefratelli Sacco, Ospedale Luigi Sacco-University of Milan, Milan, Italy., Bellizzi L; Pharming Technologies BV, Leiden, the Netherlands., Relan A; Pharming Healthcare Inc., Warren, NJ, USA., Hakl R; St. Anne's University Hospital, Masaryk University, Brno, Czechia., Farkas H; Hungarian Angioedema Center of Excellence and Reference, Department of Internal Medicine and Haematology, Semmelweis University, Budapest, Hungary.
Jazyk:	angličtina
Zdroj:	The World Allergy Organization journal [World Allergy Organ J] 2021 Apr 22; Vol. 14 (4), pp. 100535. Date of Electronic Publication: 2021 Apr 22 (Print Publication: 2021).
DOI:	10.1016/j.waojou.2021.100535
Abstrakt:	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. Competing Interests: A Valerieva reports serving as a consultant for Pharming Group NV; and receiving symposium sponsorship from Takeda/Shire, Sobi, and CSL Behring. MT Staevska reports receiving consultancy/speaker honoraria from Pharming Group NV and Sobi. V Grivcheva-Panovska reports serving as principal investigator for clinical trials sponsored by Pharming Group NV. M Jesenak reports receiving consultancy/speaker honoraria from CSL Behring, Shire, Sobi, and Takeda Pharmaceutical Co. Ltd.; and serving as a principal investigator for clinical trials sponsored by BioCryst Pharmaceuticals, Inc. and Pharming Group NV. K Viktória Kőhalmi reports receiving consultancy/speaker honoraria from CSL Behring and Shire. K Hrubiskova reports serving as co-investigator for clinical trial sponsored by Pharming Group NV and receiving consultancy honoraria from Takeda Pharmaceutical Co. Ltd. A Zanichelli reports serving as a consultant for CSL Behring and Shire HGT. L Bellizzi is an employee of Pharming Group NV. A Relan is an employee of Pharming Healthcare Inc. R Hakl reports receiving consultancy/speaker honoraria from CSL Behring, Shire, and Takeda Pharmaceutical Co. Ltd.; and serving as a principal investigator for clinical trials sponsored by BioCryst Pharmaceuticals, Pharming Group NV, and KalVista Pharmaceuticals. H Farkas reports receiving research grants from CSL Behring, Pharming Group NV, and Shire/Takeda; consultancy/speaker fees and honoraria from BioCryst Pharmaceuticals, Inc., CSL Behring, Pharming, and Shire HGT/Takeda; serving as an advisor for these companies, and as a principal investigator for clinical trials/registries for BioCryst, CSL Behring, KalVista, Pharming, and Shire/Takeda. (© 2021 The Authors.)
Databáze:	MEDLINE
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