Background Hereditary angioedema (HAE) type I is a rare genetic disorder, autosomal dominant, caused by a deficiency of the enzyme C1 inhibitor (C1INH). It is characterised by recurrent and unpredictable episodes of oedema primarily affecting the extremities, face, throat and intestinal wall and may jeopardise the patient's life if not treated properly. Available treatments for acute attacks are icatibant acetate (IA) given subcutaneously (sc), fresh plasma and C1INH esterase (CE) (a foreign substance – likely to result in antibodies), both given intravenously.
Purpose To describe our experience in the treatment of acute episodes of HAE in two patients (siblings) from our health area.
Materials and methods Retrospective observational study. Period: January 2008 – September 2011. Data source: medical history, Sinfhos® software. Data: age and sex, prophylactic treatment, number of crises and accident and emergency (A&E) visits, acute treatment, efficiency, safety and cost.
Results Two siblings (male and female, 33 and 31 years old respectively) with no prophylactic treatment, had poorly-controlled symptoms and poor tolerance. Between them, the two came to the A&E 19 times, presenting attacks with laryngeal oedema. 11 were treated with CE; in 3 cases a second dose was needed and in another one 4 doses. IA was already available in the hospital; the last 4 outbreaks were treated with a single dose of this agent with complete resolution of symptoms. There were no adverse effects with either drug.
Conclusions In all episodes treated with IA, the outbreak was resolved with a single dose. The SC route of administration of the IA allows self-administration in a crisis if laryngeal oedema is not involved. The haematological safety profile, storage at room temperature and that it isn't a foreign substance represent additional advantages of IA against haematological derivatives. The average price of treatment with CE was € 1060.78 and with IA € 1692.29.
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