TY - JOUR
T1 - Efficacy of recombinant human C1 esterase inhibitor for the treatment of severe hereditary angioedema attacks
AU - Li, H. Henry
AU - Reshef, Avner
AU - Baker, James W.
AU - Harper, Joseph R.
AU - Relan, Anurag
N1 - Funding Information:
ogiesBV,Leiden,TheNetherlands autosomal dominant genetic disorder caused by mu- H. H. Li receives research grants from BioCryst, CSL Behring, Dyax, Pharming Tech- nologies, and Shire/ViroPharma; serves as a consultant for CSL Behring, Salix Pharma- ceuticals, and Shire/ViroPharma; and is a speaker for CSL Behring and Shire. A. Reshef receives research grants from CSL Behring, Pharming Technologies, Shire HGT, Staller- gens, and Teva Pharmaceuticals; and serves on the speakers’ bureaus for Pharming Technologies and Shire HGT, and on the advisory boards for CSL Behring and Shire HGT. J.W. Baker is a researcher for BioCryst, CSL Behring, Dyax, Pharming Technolo- gies, and Shire; serves as consultant for BioCryst; and serves on the speakers’ bureau for Shire. J.R. Harper is an employee of Pharming Healthcare Inc. and a former employee of Salix Pharmaceuticals. A. Relan is an employee of Pharming Healthcare Inc.
Publisher Copyright:
Copyright © 2017, OceanSide Publications, Inc., U.S.A.
PY - 2017/11/1
Y1 - 2017/11/1
N2 - Background: Severe attacks of hereditary angioedema (HAE) are debilitating and potentially life threatening, and can increase anxiety and the use of medical resources.Objective: This post hoc assessment evaluated recombinant human C1 esterase inhibitor (rhC1-INH) used to treat acute severe HAE attacks.Methods: In a double-blind, randomized-controlled trial (RCT), patients with an HAE attack (baseline visual analog scale score of ≥50 mm, with severe attacks defined as ≥75 mm) were randomly assigned to receive rhC1-INH (50 IU/kg for patients who weighed ≤84 kg; 4200 IU for patients who weighed ≤84 kg) or placebo. Also, in an open-label extension (OLE) study of rhC1-INH, oropharyngeal-laryngeal attacks were analyzed. Rescue therapy with rhC1-INH 50 IU/kg (≥4200 IU) was permitted after 4 hours or for life-threatening symptoms (in the RCT) or after 1 hour (in the OLE study). The primary end point measured the time to the beginning of symptom relief by using the Treatment Effects Questionnaire. Results: Of 75 adults in the RCT, 43 had severe attacks and received either rhC1-INH (n = 24) or placebo (n = 19). The median (95% confidence interval) time to the onset of symptom relief totaled 90.0 minutes (95% confidence interval, 47.0-120.0 minutes) versus 334.0 minutes (95% confidence interval, 105.0 to not calculable minutes; hazard ratio, 2.5; p = 0.02), for rhC1-INH and placebo, respectively. Open-label rhC1-INH rescue therapy was administered to 1 of 24 in the rhC1-INH group (4.2%) and 10 of 19 in the placebo group (52.6%). During the OLE study, the median onset of symptom relief with rhC1-INH for eight oropharyngeal-laryngeal HAE attacks was 69.0 minutes (95% confidence interval, 59.0-91.0 minutes). Conclusion: In the current study, rhC1-INH was efficacious in resolving severe HAE attacks, including oropharyngeallaryngeal attacks. The rhC1-INH rescue treatment rapidly improved symptoms for patients who received placebo and who experienced worsening or sustained symptoms.
AB - Background: Severe attacks of hereditary angioedema (HAE) are debilitating and potentially life threatening, and can increase anxiety and the use of medical resources.Objective: This post hoc assessment evaluated recombinant human C1 esterase inhibitor (rhC1-INH) used to treat acute severe HAE attacks.Methods: In a double-blind, randomized-controlled trial (RCT), patients with an HAE attack (baseline visual analog scale score of ≥50 mm, with severe attacks defined as ≥75 mm) were randomly assigned to receive rhC1-INH (50 IU/kg for patients who weighed ≤84 kg; 4200 IU for patients who weighed ≤84 kg) or placebo. Also, in an open-label extension (OLE) study of rhC1-INH, oropharyngeal-laryngeal attacks were analyzed. Rescue therapy with rhC1-INH 50 IU/kg (≥4200 IU) was permitted after 4 hours or for life-threatening symptoms (in the RCT) or after 1 hour (in the OLE study). The primary end point measured the time to the beginning of symptom relief by using the Treatment Effects Questionnaire. Results: Of 75 adults in the RCT, 43 had severe attacks and received either rhC1-INH (n = 24) or placebo (n = 19). The median (95% confidence interval) time to the onset of symptom relief totaled 90.0 minutes (95% confidence interval, 47.0-120.0 minutes) versus 334.0 minutes (95% confidence interval, 105.0 to not calculable minutes; hazard ratio, 2.5; p = 0.02), for rhC1-INH and placebo, respectively. Open-label rhC1-INH rescue therapy was administered to 1 of 24 in the rhC1-INH group (4.2%) and 10 of 19 in the placebo group (52.6%). During the OLE study, the median onset of symptom relief with rhC1-INH for eight oropharyngeal-laryngeal HAE attacks was 69.0 minutes (95% confidence interval, 59.0-91.0 minutes). Conclusion: In the current study, rhC1-INH was efficacious in resolving severe HAE attacks, including oropharyngeallaryngeal attacks. The rhC1-INH rescue treatment rapidly improved symptoms for patients who received placebo and who experienced worsening or sustained symptoms.
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U2 - 10.2500/aap.2017.38.4088
DO - 10.2500/aap.2017.38.4088
M3 - Article
C2 - 28874235
AN - SCOPUS:85034947908
SN - 1088-5412
VL - 38
SP - 456
EP - 461
JO - Allergy and Asthma Proceedings
JF - Allergy and Asthma Proceedings
IS - 6
ER -