Recombinant Human-C1 Inhibitor Is Effective and Safe for Repeat Hereditary Angioedema Attacks

H. Henry Li, Dumitru Moldovan, Jonathan A. Bernstein, Avner Reshef, Gregor Porebski, Marcin Stobiecki, James Baker, Robyn Levy, Anurag Relan, Marc Riedl

Research output: Contribution to journalArticle

Abstract

Background: Hereditary angioedema (HAE) caused by a deficiency in functional C1 esterase inhibitor (C1INH) is characterized by recurrent episodes of cutaneous and/or mucosal/submucosal tissue swelling affecting multiple anatomic locations. Previous studies demonstrated efficacy of recombinant human C1INH (rhC1INH) for acute HAE attacks. Objective: This study evaluated the efficacy and safety of rhC1INH (50 IU/kg) for the treatment of multiple HAE attacks in an open-label extension study. Methods: Time to onset of symptom relief and time to minimal symptoms were assessed using a Treatment Effect Questionnaire (TEQ), a visual analog scale, and a 6-point ordinal scale Investigator Score. Results: Forty-four patients received rhC1INH, and a single dose was administered for 215 of 224 (96%) attacks. Median time to beginning of symptom relief based on TEQ for the first 5 attacks was 75.0 (95% CI, 69-89) minutes, ranging from 62.5 (95% CI, 48-90) to 134.0 (95% CI, 32-119) minutes. Median time to minimal symptoms using TEQ for the first 3 attacks was 303.0 (95% CI, 211-367) minutes. rhC1INH was well tolerated. There were no discontinuations due to adverse events. No thrombotic or anaphylactic events were reported, and repeat rhC1INH treatments were not associated with neutralizing anti-C1INH antibodies. Conclusions: A single 50-IU/kg dose rhC1INH was effective for improving symptoms of an HAE attack with sustained efficacy for treatment of subsequent attacks. rhC1INH had a positive safety profile throughout the study. This study supports repeated use of rhC1INH over time in patients with HAE attacks.

Original languageEnglish (US)
Pages (from-to)417-423
Number of pages7
JournalJournal of Allergy and Clinical Immunology: In Practice
Volume3
Issue number3
DOIs
StatePublished - 2015

Keywords

  • Hereditary angioedema
  • Recombinant human C1 esterase inhibitor
  • Repeat attacks

ASJC Scopus subject areas

  • Immunology and Allergy

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    Li, H. H., Moldovan, D., Bernstein, J. A., Reshef, A., Porebski, G., Stobiecki, M., Baker, J., Levy, R., Relan, A., & Riedl, M. (2015). Recombinant Human-C1 Inhibitor Is Effective and Safe for Repeat Hereditary Angioedema Attacks. Journal of Allergy and Clinical Immunology: In Practice, 3(3), 417-423. https://doi.org/10.1016/j.jaip.2014.12.013