Self-administered C1 esterase inhibitor concentrates for the management of hereditary angioedema: Usability and patient acceptance

Research output: Contribution to journalReview articlepeer-review

Abstract

Hereditary angioedema (HAE) is a rare genetic disease characterized by episodic subcutaneous or submucosal swelling. The primary cause for the most common form of HAE is a deficiency in functional C1 esterase inhibitor (C1-INH). The swelling caused by HAE can be painful, disfiguring, and life-threatening. It reduces daily function and compromises the quality of life of affected individuals and their caregivers. Among different treatment strategies, replacement with C1-INH concentrates is employed for on-demand treatment of acute attacks and long-term prophylaxis. Three human plasma-derived C1-INH preparations are approved for HAE treatment in the US, the European Union, or both regions: Cinryze®, Berinert®, and Cetor®; however, only Cinryze is approved for long-term prophylaxis. Postmarketing studies have shown that home therapy (self-administered or administered by a caregiver) is a convenient and safe option preferred by many HAE patients. In this review, we summarize the role of self-administered plasma-derived C1-INH concentrate therapy with Cinryze at home in the prophylaxis of HAE.

Original languageEnglish (US)
Pages (from-to)1727-1737
Number of pages11
JournalPatient Preference and Adherence
Volume10
DOIs
StatePublished - Sep 7 2016
Externally publishedYes

Keywords

  • C1-INH concentrate
  • Disease management
  • First line
  • Hereditary angioedema
  • Prophylaxis
  • Self-administration

ASJC Scopus subject areas

  • Medicine (miscellaneous)
  • Social Sciences (miscellaneous)
  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
  • Health Policy

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