Background: Acute treatment must be permanently accessible for every patient diagnosed with hereditary angioedema (HAE). In many cases this type of therapy does not provide/offer sufficient control of the disease, so long-term prophylaxis (LTP) is recommended. In the case of regular and prolonged/extended administration of drugs, the route of administration is essential. The aim of the investigation was to assess the control of HAE among patients in Romania receiving the available medications, while also examining potential correlations within the outcomes.
Material and methods: A phone call was made to all adult patients registered in the Romanian HAE Registry. Patients with confirmed diagnosis of HAE who had at least one angioedema attack in the last three months were asked to complete, online, the angioedema control test (AECT) for one- and three months respectively. AECT scores were calculated according to the authors’ instructions.
Results: A total of 121 patients were contacted. Of these, 83 complies with the eligibility criteria and 56 completed the questionnaires (response rate 67.4%), 18 (32.1%) men and 38 (67.9%) women. Acute, home administered treatment with Icatibant or pdC1-INH was available for every patient during the study time. Nine (14.5%) participants used LTP too, with pdC1-INH. These treatments ensured an adequate control of the disease in only 13 patients (21%) in case of the three-month AECT, of whom 2 used LTP. The one-month questionnaire showed a well-controlled disease in 14 patients (23%), from which only 1 was on prophylactic therapy.
Conclusion: In most Romanian HAE patients, the available drugs do not offer a proper control of the disease. Even though a first-line drug for LTP is available, its administration route by intravenous injections makes it inconvenient for many patients, highlighting the necessity for new, easy-to administer drugs for HAE patients from our country.