Analysis of Prophylactic Treatment of Patients With Hereditary Angioedema in the Slovak Republic in 2022: Results of the Descriptive Cross-Sectional Database Study

Speaker(s)

Ondrusova M1, Jesenak M2, Fajbikova A1, Garaiova J1, Švaro M1, Suchansky M3
1Pharm-In, spol. s r. o., Bratislava, BL, Slovakia, 2University Hospital Martin, Martin, Martin, Slovakia, 3Pharm-In, spol. s r. o., Bratislava, Slovakia

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.

Code

CO28

Topic

Clinical Outcomes

Topic Subcategory

Comparative Effectiveness or Efficacy

Disease

Drugs, Rare & Orphan Diseases