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

Result code in IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00159816%3A_____%2F21%3A00074717" target="_blank" >RIV/00159816:_____/21:00074717 - isvavai.cz</a>

Alternative codes found

RIV/00216224:14110/21:00121728

Result on the web

<a href="https://www.sciencedirect.com/science/article/pii/S1939455121000296?via%3Dihub" target="_blank" >https://www.sciencedirect.com/science/article/pii/S1939455121000296?via%3Dihub</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1016/j.waojou.2021.100535" target="_blank" >10.1016/j.waojou.2021.100535</a>

Result language

angličtina

Original language name

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

Original language description

Background: Hereditary angioedema (HAE) due to C1 esterase inhibitor deficiency (C1-INHHAE) 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.

Czech name

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Czech description

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Type

J_imp - Article in a specialist periodical, which is included in the Web of Science database

CEP classification

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OECD FORD branch

30102 - Immunology

Project

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Continuities

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Publication year

2021

Confidentiality

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Name of the periodical

World Allergy Organization Journal

ISSN

1939-4551

e-ISSN

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Volume of the periodical

14

Issue of the periodical within the volume

4

Country of publishing house

GB - UNITED KINGDOM

Number of pages

8

Pages from-to

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UT code for WoS article

000647796400004

EID of the result in the Scopus database

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