OBJECTIVES: In Slovakia there is long-term unavailability of hard data on healthcare management and treatment of hereditary angioedema (HAE), which can be used for preparation of cost-effectiveness analysis and budget impact analysis. Therefore, a descriptive cross-sectional database study analyzing the prophylactic treatment of patients with HAE was conducted.

METHODS: A descriptive cross-sectional database study was conducted using the registry of patients with HAE. Individual unstructured and unique data on patients were summarized in a database form. The analysis of data was conducted to summarize the proportion of different types of prophylactic treatments and identify their effect on the course of HAE.

RESULTS: The study analyzed data from 128 patients, which was a representative cohort considering the prevalence of the disease in Slovakia. Out of this patients’ cohort, 57.81% were treated with prophylactic treatment, of which 93.24% were treated with danazol, lanadelumab and berotralstat. Prophylactic treatment with danazol was well tolerated in 46.67% of patients, any adverse events were reported in 11.11%. Tolerance of prophylactic treatment with lanadelumab was good in all treated patients, no adverse events were reported. Prophylactic treatment with berotralstat was well tolerated in 50.00% of patients, adverse events were reported in 25.00%. Treatment was rated as excellent in 82.22% of patients treated with danazol, in 100% patients treated with lanadelumab and in 87.60% of patients treated with berotralstat. A reduction of seizure frequency by more than 90% was predominantly observed in patients treated with lanadelumab (93.33%) compared to patients treated with berotralstat (50.00%). In patients treated with danazol, a reduction of seizure frequency by more than 90% was observed in 20.00% of patients.

CONCLUSIONS: The study has compared the prophylactic treatment of patients based on a descriptive cross-sectional database study in which the seizure frequencies and selected characteristics of patients with HAE were determined in Slovakia.