Individual approach to long-term therapy in patients with hereditary angioedema (HAE-C1-INH): A case series.

Abstract

Hereditary angioedema with deficiency of C1-Inhibitor (HAE-C1-INH) is associated with recurrent angioedema. Clinically these manifest as skin swellings, abdominal pain and/or potentially life-threatening laryngeal edema attacks. Symptoms however show wide inter- and intraindividual variations in frequency and intensity. HAE-C1-INH may lead to substantial secondary morbidity, impaired quality of life and significant burden of disease. We review three HAE cases with poorly controlled disease from different age groups who responded well to long term prophylaxis (LTP) with different approaches. The patients presented with a high frequency of HAE-attacks before starting prophylactic treatment. Both, burden of disease and patient preference were considered in the joint decision for initiating long term prophylaxis. The rate of HAE attacks in the three months prior to the current LTP in these patients were: 1,33 per month despite previous prophylaxis with a different medication (Case 1), 2,67 (Case 2) and 4,33 attacks per month (Case 3). In all three cases, LTP resulted in a substantial improvement of HAE symptoms with no or almost no attacks during the observation period (33, 15 and 6 months) which is reflected in improved disease control as per the angioedema control test (AECT). These cases demonstrate the ability of LTP to reach disease control by preventing or minimizing attacks in hereditary angioedema patients. In these three patients, adequate symptom control was achieved with three different types of LTP which were chosen according to patients´preference and which differ in mechanism of action, as well as mode and timing of administration.