Evaluating the control of the disease on current treatments available for the Romanian hereditary angioedema patients to assess the need for innovative, easy-to-administer drugs

Noemi Anna Bara; Iris Nadasan; Valentin Nadasan; Diana Deleanu

doi:10.2478/amma-2024-0009

Noemi Anna Bara UMFST George Emil Palade Targu Mures
Iris Nadasan UMFST George Emil Palade Targu Mures
Valentin Nadasan UMFST George Emil Palade Targu Mures
Diana Deleanu UMFST George Emil Palade Targu Mures, UMF Iuliu Hatieganu Cluj Napoca, Institutul Regional de Gastroenterologie si Hepatologie Octavian Fodor Cluj Napoca

DOI: https://doi.org/10.2478/amma-2024-0009

Keywords: angioedema, hereditary angioedema, quality of life, on-demand, long-term prophylaxis

Abstract

Background: The availability of on-demand treatment is essential for all patients with a confirmed diagnosis of hereditary angioedema (HAE). When this type of medication does not provide an adequate control of the disease, long-term prophylaxis (LTP) should be considered. In case of regular and prolonged use of drugs, the route of administration is critical. The objective of this study was to evaluate the control of the disease on available treatments for Romanian HAE patients and to assess the need for innovative, easy-to-administer drugs.

Material and methods:

Adult patients from the Romanian HAE Registry were contacted by phone in March 2022. Those with confirmed diagnosis of HAE and more than one attack in the last three months were invited to complete the one- and three-months angioedema control test (AECT), online. AECT scores were computed according to the AECT authors’ instructions.

Results: Out of 121 patients, 68 met the eligibility criteria. The questionnaire was completed by 56 patients (response rate 82.4%), of which 18 (32.1%) were men and 38 (67.9%) women. On-demand treatment (Icatibant or pdC1-INH) was available at home to all respondents during the study period. LTP with intravenous pdC1-INH was used by 9 patients (14.5%). On these treatments, the three-months AECT revealed a well-controlled disease in only 13 patients (23.2%), of whom 2 used LTP. When the questions referred only to the last month, an adequate control was achieved in 14 patients (25.0%), only one of them being on LTP.

Conclusions: The on-demand therapy does not ensure adequate control of the disease in most Romanian HAE patients. Despite the availability of intravenous pdC1-INH as LTP, the route of administration makes it inconvenient for many patients, suggesting the need for easy-to-administer drugs for patients with HAE from Romania.