TY - JOUR
T1 - Recombinant human C1 esterase inhibitor for acute hereditary angioedema attacks with upper airway involvement
AU - Riedl, Marc A.
AU - Li, H. Henry
AU - Cicardi, Marco
AU - Harper, Joseph R.
AU - Relan, Anurag
N1 - Funding Information:
Immunology,DO NOT COPYBarcelona, Spain, June 6–10, 2015 Technical editorial and medical writing assistance was provided by Mary Beth Mon- crief, Ph.D., and Jillian Gee, Ph.D., Synchrony Medical Communications, L.L.C., West Chester, PA, under the direction of the authors. Funding for this support was provided by Salix Pharmaceuticals, Bridgewater, New Jersey, and Pharming Health Care, Inc., Bridgewater, New Jersey This study was supported by Pharming Technologies BV, Leiden, the Netherlands, Pharming Healthcare, Inc., Bridgewater, New Jersey, and Salix Pharmaceuticals, Bridgewater, New Jersey M.A. Riedl receives research grants from BioCryst, CSL Behring, Dyax, Ionis, Pharm- ing Technologies, and Shire; serves as a consultant for Adverum Biotechnologies, Alnylam, Arrowhead, BioCryst, CSL Behring, Dyax, Global Blood Therapeutics, Ionis, KalVista Pharmaceuticals, Salix Pharmaceuticals, and Shire; and serves on the speak- ers’ bureaus for CSL Behring, Dyax, Salix, and Shire. H.H. Li receives research grants from BioCryst, CSL Behring, Dyax, Pharming Technologies, and Shire/ViroPharma; serves as a consultant for CSL Behring, Salix Pharmaceuticals, and Shire/ViroPharma;
Funding Information:
and serves as a speaker for CSL Behring and Shire. M. Cicardi reports receiving a research grant from Shire and serving as a speaker or consultant for Alnylam, BioCryst, CSL Behring, Pharming, Shire, and Sobi. J. Harper is an employee of Pharming Healthcare Inc., and a former employee of Salix Pharmaceuticals. A. Relan is an employee of Pharming Healthcare Inc Address correspondence to Marc A. Riedl, M.D., M.S., Division of Rheumatology, Allergy and Immunology, Department of Medicine University of California—San Diego, 8899 University Center Lane, Ste 230, San Diego, CA 92122 E-mail address: [email protected] Published online September 13, 2017 Copyright © 2017, OceanSide Publications, Inc., U.S.A.
Publisher Copyright:
Copyright © 2017, OceanSide Publications, Inc., U.S.A.
Copyright:
Copyright 2018 Elsevier B.V., All rights reserved.
PY - 2017/11/1
Y1 - 2017/11/1
N2 - Background: Recombinant human C1 esterase inhibitor (rhC1-INH) is approved for treatment of hereditary angioedema (HAE) in adolescents and adults. HAE attacks that involve the upper airway can be life threatening, and data on the administration of rhC1-INH for these types of attacks are currently limited. Objective: To evaluate the efficacy and safety of rhC1-INH for treatment of acute HAE attacks with upper airway involvement. Methods: A pooled analysis of data from three clinical trials with open-label extensions examined rhC1-INH for treatment of acute HAE attacks with upper airway involvement. Patients with functional plasma C1 esterase inhibitor <50% of normal who had experienced an acute upper airway HAE attack and received rhC1-INH were identified retrospectively based on severity of breathing or swallowing symptoms. The primary end point was the time to beginning of relief (time at which the overall visual analog scale score [0-100 mm] decreased from baseline by ≥20 mm for two consecutive time points [persistence]). Results: Of 683 acute HAE attacks treated with rhC1-INH, data for 45 attacks with upper airway involvement were included. The median time to the beginning of symptom relief was 67 minutes (95% confidence interval, 60-120 minutes) and did not differ by attack number or by baseline breathing or swallowing symptom severity. Most attacks (91.1%) achieved the beginning of relief within 4 hours of rhC1-INH treatment. All attacks resolved without the need for any additional medication, and no patients required intubation or tracheostomy. Treatment with rhC1-INH was well tolerated, with no adverse events reported in more than one patient (except HAE reported as an adverse event [n = 2]). Conclusion: This pooled analysis of clinical trial data supports the efficacy of rhC1-INH for treatment of acute HAE attacks with upper airway involvement.
AB - Background: Recombinant human C1 esterase inhibitor (rhC1-INH) is approved for treatment of hereditary angioedema (HAE) in adolescents and adults. HAE attacks that involve the upper airway can be life threatening, and data on the administration of rhC1-INH for these types of attacks are currently limited. Objective: To evaluate the efficacy and safety of rhC1-INH for treatment of acute HAE attacks with upper airway involvement. Methods: A pooled analysis of data from three clinical trials with open-label extensions examined rhC1-INH for treatment of acute HAE attacks with upper airway involvement. Patients with functional plasma C1 esterase inhibitor <50% of normal who had experienced an acute upper airway HAE attack and received rhC1-INH were identified retrospectively based on severity of breathing or swallowing symptoms. The primary end point was the time to beginning of relief (time at which the overall visual analog scale score [0-100 mm] decreased from baseline by ≥20 mm for two consecutive time points [persistence]). Results: Of 683 acute HAE attacks treated with rhC1-INH, data for 45 attacks with upper airway involvement were included. The median time to the beginning of symptom relief was 67 minutes (95% confidence interval, 60-120 minutes) and did not differ by attack number or by baseline breathing or swallowing symptom severity. Most attacks (91.1%) achieved the beginning of relief within 4 hours of rhC1-INH treatment. All attacks resolved without the need for any additional medication, and no patients required intubation or tracheostomy. Treatment with rhC1-INH was well tolerated, with no adverse events reported in more than one patient (except HAE reported as an adverse event [n = 2]). Conclusion: This pooled analysis of clinical trial data supports the efficacy of rhC1-INH for treatment of acute HAE attacks with upper airway involvement.
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U2 - 10.2500/aap.2017.38.4090
DO - 10.2500/aap.2017.38.4090
M3 - Article
C2 - 28903805
AN - SCOPUS:85035066462
SN - 1088-5412
VL - 38
SP - 462
EP - 466
JO - Allergy and Asthma Proceedings
JF - Allergy and Asthma Proceedings
IS - 6
ER -