Hereditary Angioedema Patient’s Preferences in Treatment

Описание: Courtney Olson, MD, Beth Israel Deaconess Medical Center, discusses results of a survey of patients with hereditary angioedema (HAE) and their preferences for an oral long-term prophylactic (LTP) treatment.

HAE is a rare disease characterized by recurrent episodes of severe swelling of the skin and mucous membranes. The frequency of attacks usually increases after puberty. Attacks most often affect 3 parts of the body: Skin, gastrointestinal tract, and upper airway. Attacks may involve one area of the body at a time, or they may involve a combination of areas. They always go away on their own but last from 2 to 4 days. While people with HAE have reported various triggers of attacks, emotional stress, physical stress, and dental procedures are the most commonly reported triggers. HAE may be caused by genetic changes in the C1NH gene (also called the SERPING1 gene) or in the F12 gene. In some cases, the cause is not yet known.

150 adult patients with HAE participated in an online survey. 99% of participants were taking either an acute or LTP HAE medication. The most important attributes of LTP treatments were effectiveness in preventing attacks and reduction in the severity of attacks. Additionally, patients place more importance on the route of administration and convenience than on dosing frequency. When equally efficacious, 54% prefer an oral daily therapy to a bi-weekly injection. 52% preferred an oral LTP over injections as infrequent as every three months.