Literature DB >> 31982824

Evaluation of the efficacy and safety of home treatment with the recombinant human C1-inhibitor in hereditary angioedema resulting from C1-inhibitor deficiency.

Noémi Andrási1, Nóra Veszeli2, Ágnes Holdonner3, György Temesszentandrási4, Kinga Viktória Kőhalmi5, Lilian Varga3, Henriette Farkas6.   

Abstract

OBJECTIVE: Conestat alpha, a C1-inhibitor produced by recombinant technology (rhC1-INH) is an acute treatment for edematous attacks occurring in hereditary angioedema (HAE) with C1-inhibitor deficiency (C1-INH-HAE). Our study evaluated the efficacy and safety of rhC1-INH administered during HAE attacks, and for short-term prophylaxis (STP). MATERIALS &
METHOD: Our prospective study analyzed the course of 544 HAE attacks experienced by the 21 C1-INH-HAE patients treated, as well as the outcome of 97 instances of STP implemented with rhC1-INH. Using a purpose-designed questionnaire, the patients recorded relevant, treatment-related information.
RESULTS: Time to the administration of rhC1-INH was 90.0 min (median) after the onset of HAE attacks. The symptoms started to improve as early as 60 min after the injection of rhC1-INH, and the attack resolved 730.0 min after treatment. The interval between the onset of the HAE attack and the administration of rhC1-INH correlated with time until the onset of improvement (R = 0.2053 p < 0.0001), and with time to the complete resolution of symptoms (R = 0.2805, p < 0.0001). Nine patients received STP with rhC1-INH in 97 instances. STP successfully prevented the HAE attack within 72 h of the event on 93/97 occasions. No local and serious systemic adverse events/effects were observed.
CONCLUSIONS: Treatment with rhC1-INH is effective and safe both for acute management, and for STP. Following the onset of an HAE attack, early administration of rhC1-INH may reduce time to the improvement and to the complete resolution of symptoms. Repeated administration of rhC1-INH does not impair its efficacy.
Copyright © 2020 The Authors. Published by Elsevier B.V. All rights reserved.

Entities:  

Keywords:  Efficacy; Hereditary angioedema; Recombinant human C1 inhibitor acute treatment; Safety; Short term prophylaxis

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Year:  2020        PMID: 31982824     DOI: 10.1016/j.intimp.2020.106216

Source DB:  PubMed          Journal:  Int Immunopharmacol        ISSN: 1567-5769            Impact factor:   4.932


  1 in total

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

Authors:  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
Journal:  World Allergy Organ J       Date:  2021-04-22       Impact factor: 4.084

  1 in total

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