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

Autor: Andrási N; Hungarian Angioedema Reference Center, 3(rd) Department of Internal Medicine, Semmelweis University, Hungary; 2(nd) Department of Pediatrics, Semmelweis University, Hungary., Veszeli N; Hungarian Angioedema Reference Center, 3(rd) Department of Internal Medicine, Semmelweis University, Hungary; MTA-SE Immunology & Hematology Research Group, Semmelweis University and Hungarian Academy of Sciences, Hungary., Holdonner Á; Hungarian Angioedema Reference Center, 3(rd) Department of Internal Medicine, Semmelweis University, Hungary., Temesszentandrási G; 3(rd) Department of Internal Medicine, Semmelweis University, Hungary., Kőhalmi KV; Hungarian Angioedema Reference Center, 3(rd) Department of Internal Medicine, Semmelweis University, Hungary; Hospital of Hospitaller Brothers of St. John of God, Department of Rheumatology, Hungary., Varga L; Hungarian Angioedema Reference Center, 3(rd) Department of Internal Medicine, Semmelweis University, Hungary., Farkas H; Hungarian Angioedema Reference Center, 3(rd) Department of Internal Medicine, Semmelweis University, Hungary. Electronic address: farkas.henriette@med.semmelweis-univ.hu.
Jazyk: angličtina
Zdroj: International immunopharmacology [Int Immunopharmacol] 2020 Mar; Vol. 80, pp. 106216. Date of Electronic Publication: 2020 Jan 24.
DOI: 10.1016/j.intimp.2020.106216
Abstrakt: 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.
Competing Interests: Declaration of Competing Interest N. Andrási, Á. Holdonner, Gy. Temesszentandrási have no conflict of interest, N. Veszeli has received travel grants from CSL Behring. K. V. Kőhalmi has received travel grants from CSL Behring and Shire. L. Varga has received travel grants from CSL Behring and Shire Human Genetic Therapies Inc. H. Farkas has received honoraria and travel grants from CSL Behring, Shire, Swedish Orphan Biovitrum, and Pharming; and/or served as a consultant for these companies and has participated in clinical trials/registries for BioCryst, CSL Behring, Pharming, and Shire.
(Copyright © 2020 The Authors. Published by Elsevier B.V. All rights reserved.)
Databáze: MEDLINE
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