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

J 2021

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

VALERIEVA, A., M. T. STAEVSKA, V. GRIVCHEVA-PANOVSKA, M. JESENAK, K. V. KOHALMI et. al.

Základní údaje

Originální název

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

Autoři

VALERIEVA, A. (garant), M. T. STAEVSKA, V. GRIVCHEVA-PANOVSKA, M. JESENAK, K. V. KOHALMI, K. HRUBISKOVA, A. ZANICHELLI, L. BELLIZZI, A. RELAN, Roman HAKL (203 Česká republika, domácí) a H. FARKAS

Vydání

WORLD ALLERGY ORGANIZATION JOURNAL, AMSTERDAM, ELSEVIER, 2021, 1939-4551

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30102 Immunology

Stát vydavatele

Nizozemské království

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 5.516

Kód RIV

RIV/00216224:14110/21:00121728

Organizační jednotka

Lékařská fakulta

DOI

http://dx.doi.org/10.1016/j.waojou.2021.100535

UT WoS

000647796400004

Klíčová slova anglicky

Angioedema; Hereditary; Complement C1 inhibitor protein; Recombinant human C1 esterase inhibitor; Registry; Ruconest

Štítky

14110114, rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 8. 6. 2021 10:59, Mgr. Tereza Miškechová

Anotace

V originále

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 & ndash;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 & ndash;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.

Citovat

VALERIEVA, A., M. T. STAEVSKA, V. GRIVCHEVA-PANOVSKA, M. JESENAK, K. V. KOHALMI, K. HRUBISKOVA, A. ZANICHELLI, L. BELLIZZI, A. RELAN, Roman HAKL a H. FARKAS. Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registry. WORLD ALLERGY ORGANIZATION JOURNAL. AMSTERDAM: ELSEVIER, 2021, roč. 14, č. 4, s. 1-8. ISSN 1939-4551. Dostupné z: https://dx.doi.org/10.1016/j.waojou.2021.100535.

@article{1774646,
   author = {Valerieva, A. and Staevska, M. T. and GrivchevaandPanovska, V. and Jesenak, M. and Kohalmi, K. V. and Hrubiskova, K. and Zanichelli, A. and Bellizzi, L. and Relan, A. and Hakl, Roman and Farkas, H.},
   article_location = {AMSTERDAM},
   article_number = {4},
   doi = {http://dx.doi.org/10.1016/j.waojou.2021.100535},
   keywords = {Angioedema; Hereditary; Complement C1 inhibitor protein; Recombinant human C1 esterase inhibitor; Registry; Ruconest},
   language = {eng},
   issn = {1939-4551},
   journal = {WORLD ALLERGY ORGANIZATION JOURNAL},
   title = {Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registry},
   url = {https://www.worldallergyorganizationjournal.org/article/S1939-4551(21)00029-6/pdf},
   volume = {14},
   year = {2021}
}

TY  - JOUR
ID  - 1774646
AU  - Valerieva, A. - Staevska, M. T. - Grivcheva-Panovska, V. - Jesenak, M. - Kohalmi, K. V. - Hrubiskova, K. - Zanichelli, A. - Bellizzi, L. - Relan, A. - Hakl, Roman - Farkas, H.
PY  - 2021
TI  - Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registry
JF  - WORLD ALLERGY ORGANIZATION JOURNAL
VL  - 14
IS  - 4
SP  - 1-8
EP  - 1-8
PB  - ELSEVIER
SN  - 19394551
KW  - Angioedema
KW  - Hereditary
KW  - Complement C1 inhibitor protein
KW  - Recombinant human C1 esterase inhibitor
KW  - Registry
KW  - Ruconest
UR  - https://www.worldallergyorganizationjournal.org/article/S1939-4551(21)00029-6/pdf
N2  - 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 & ndash;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 & ndash;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.
ER  -

VALERIEVA, A., M. T. STAEVSKA, V. GRIVCHEVA-PANOVSKA, M. JESENAK, K. V. KOHALMI, K. HRUBISKOVA, A. ZANICHELLI, L. BELLIZZI, A. RELAN, Roman HAKL a H. FARKAS. Recombinant human C1 esterase inhibitor for hereditary angioedema attacks: A European registry. \textit{WORLD ALLERGY ORGANIZATION JOURNAL}. AMSTERDAM: ELSEVIER, 2021, roč.~14, č.~4, s.~1-8. ISSN~1939-4551. Dostupné z: https://dx.doi.org/10.1016/j.waojou.2021.100535.

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