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 language | English (US) |
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Pages (from-to) | 1727-1737 |
Number of pages | 11 |
Journal | Patient Preference and Adherence |
Volume | 10 |
DOIs | |
State | Published - Sep 7 2016 |
Externally published | Yes |
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