Current options for prophylactic treatment of hereditary angioedema in the United States: patient-based considerations

Abstract

Hereditary angioedema (HAE) results from mutations in the C1-esterase inhibitor (C1 INH) gene that decrease production of C1 INH or render it dysfunctional. HAE is characterized by recurrent, unpredictable, bradykinin-mediated edema of the extremities, face, genitalia, trunk, gastrointestinal tract, or upper airway. Attacks causing laryngeal edema can be fatal. Patients with HAE need medications for acute attacks; some also require prophylaxis. Management requires consideration of the patient's disease burden and effect on the patient's quality of life. This review examines an individualized approach to identifying HAE patients who may benefit from prophylaxis. A literature search was performed for HAE and prophylaxis. HAE guidelines, case reports, safety studies, and randomized, controlled clinical prophylaxis trials were selected. Authors provided cases demonstrating individualized prophylaxis. U.S. Food and Drug Administration-approved options for prophylaxis of HAE include attenuated androgens and nanofiltered C1 INH (C1 INH-nf). In other countries, pasteurized C1 INH and purified C1 INH are also available. Alternative therapies include fresh frozen plasma for preprocedural prophylaxis and antifibrinolytics for long-term prophylaxis. Attenuated androgens reduce attack frequency in many patients. Adverse effects include weight gain, virilization, increased hair growth, hypercholesterolemia, depression, and liver adenomas. C1 INH-nf reduces frequency of attacks and is well tolerated. Each patient with HAE has unique needs, based on the nature and frequency of past attacks, proximity to a medical center, occupation, and the patient's wishes. These factors should be used to create a patient-centered approach to management of HAE.