Publications

Publications, Books, Book Chapters and Reviews by Prof. Marcus Maurer, MD

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Evaluation of avoralstat, an oral kallikrein inhibitor, in a phase 3 hereditary angioedema prophylaxis trial: the OPuS-2 study

Filename 326. Riedl et al., Eval. avoralstat OPUS2, Allergy 2018.pdf
Filesize 627 KB
Version o.326
Date added June 13, 2020
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Category Original Work
Tags C1-inhibitor, Hereditary Angioedema, oral kallikrein inhibitor, Prophylaxis
Authors Riedl, M., Farkas, H., Bernstein, J. A., Bouillet, L., Sheridan, W. P., and Maurer, M.
Citation Riedl, M., Farkas, H., Bernstein, J. A., Bouillet, L., Sheridan, W. P., and Maurer, M.: Evaluation of avoralstat, an oral kallikrein inhibitor, in a phase 3 hereditary angioedema prophylaxis trial: the OPuS-2 study. Allergy 2018: 73; 1871-1880.
Corresponding authors Riedl, M.
DocNum O.326
DocType PDF
Edition; Page 73; 1871-1880
IF 6.77
Publisher Allergy
ReleaseDate 2018

Background: Effective inhibition of plasma kallikrein may have significant benefits for patients with hereditary angioedema due to deficiency of C1 inhibitor (C1-INH- HAE) by reducing the frequency of angioedema attacks. Avoralstat is a small mole- cule inhibitor of plasma kallikrein. This study (OPuS-2) evaluated the efficacy and safety of prophylactic avoralstat 300 or 500 mg compared with placebo.

Methods: OPuS-2 was a Phase 3, multicenter, randomized, double-blind, placebo- controlled, parallel-group study. Subjects were administered avoralstat 300 mg, avo- ralstat 500 mg, or placebo orally 3 times per day for 12 weeks. The primary efficacy endpoint was the angioedema attack rate based on adjudicator-confirmed attacks.

Results: A total of 110 subjects were randomized and dosed. The least squares (LS) mean attack rates per week were 0.589, 0.675, and 0.593 for subjects receiving avoralstat 500 mg, avoralstat 300 mg, and placebo, respectively. Overall, 1 subject in each of the avoralstat groups and no subjects in the placebo group were attack- free during the 84-day treatment period. The LS mean duration of all confirmed attacks was 25.4, 29.4, and 31.4 hours for the avoralstat 500 mg, avoralstat 300 mg, and placebo groups, respectively. Using the Angioedema Quality of Life Questionnaire (AE-QoL), improved QoL was observed for the avoralstat 500 mg group compared with placebo. Avoralstat was generally safe and well tolerated.

Conclusions: Although this study did not demonstrate efficacy of avoralstat in pre- venting angioedema attacks in C1-INH-HAE, it provided evidence of shortened angioedema episodes and improved QoL in the avoralstat 500 mg treatment group compared with placebo.

(Last update: 02.2021)

Number of publications (original work and reviews) in peer-reviewed journals: 601
Number of original publications in peer-reviewed journals: 432
Number of reviews in peer-reviewed journals: 169
Cumulative IF of publications (original work & reviews) in peer-reviewed journals: 3326,21
Cumulative IF for original publications in peer-reviewed journals: 2659,47
Cumulative IF for reviews in peer-reviewed journals: 666,74
Citations, Hirsch index: (view on Web of Science) 24104