PhD Scientific Days 2025

Theoretical and Translational Medicine III.

Evolution of Hereditary Angioedema Prophylaxis in Hungary Over Four Decades

Name of the presenter

Horváth Hanga Réka

Institute/workplace of the presenter

Department of Internal Medicine and Haematology, Semmelweis University

Authors

Hanga Réka Horváth¹, Beáta Visy^1,2, Kinga Viktória Kőhalmi^1,3,4, Zsuzsanna Balla^1,5, Noémi Andrási^1,6, Ibolya Czaller^1,7, Zsuzsanna Zotter^1,8, Henriette Farkas¹

1: Hungarian Angioedema Centre of Reference and Excellence, Department of Internal Medicine and Haematology, Semmelweis University, Budapest, Hungary
2: Heim Pál National Institute of Paediatrics, Budapest, Hungary
3: Department of Rheumatology & Clinical Immunology, Semmelweis University, Budapest, Hungary
4: Department of Rheumatology, National Institute of Locomotor Diseases and Disabilities, Budapest, Hungary
5: HNO-Praxis Schaffhausen, Schaffhausen, Switzerland
6: Pediatric Center, Tűzoltó Street Department, Semmelweis University, Budapest, Hungary
7: Szt. Margit Outpatient Clinic – Pulmonology, Budapest, Hungary
8: Bristol Urological Institute, Bristol, United Kingdom

Text of the abstract

Introduction:Hereditary angioedema (HAE) causes a great burden for patients because of the unpredictability of HAE attacks. Therefore, one main goal of treatment is to prevent HAE attacks using long-term prophylaxis (LTP). In the past fifty years, many LTP options became available, from traditional non-specific [attenuated androgens (e.g. danazol), antifibrinolytics (e.g. tranexamic acid (TA))] to modern, more specific [intravenous (IV) or subcutaneous (SC) C1 inhibitor (C1INH) concentrate, lanadelumab, berotralstat] drugs.
Aims: To analyse the usage of different LTP options by Hungarian HAE patients and the efficacy and safety of the drugs used.
Methods: We extracted and analysed data on LTP usage, HAE attack rate, patient-reported side-effects, and laboratory parameters from the Hungarian HAE Registry between 1979 and 2023.
Results: Danazol LTP was introduced in Hungary in 1985 and TA in 1995. Although, before 2000, more than 50% of patients used danazol, this ratio gradually decreased to 13.6% in 2023, while the proportion of patients not receiving LTP gradually increased from approximately 40% to 72.7%. TA usage peaked at 13.9% in the early 2000s and has decreased to a single patient (0.6%) in 2023. Since 1999, twelve patients have received temporary off-label IV-C1INH prophylaxis. Clinical trials of modern LTP options have included eleven patients since 2014. Two patients received modern LTPs through post-trial access in 2023. In 2022, we introduced SC-C1INH to four patients and lanadelumab to eight.
Danazol and TA were effective in reducing the number of HAE attacks in 60% of patients. With the minimal effective dose used, there were no clinically significant changes in the investigated laboratory parameters. Patients on SC-C1INH and lanadelumab had fewer HAE attacks and a better quality of life compared to the periods before treatment. They did not experience any serious side-effects and no clinically significant changes in the laboratory parameters were observed.
Conclusion: In Hungary, the changes in LTP usage follow the global trends. Both modern and traditional LTP options proved safe and effective in our patient population when used with appropriate monitoring.
Funding: Supported by the EKÖP-2024-31 New National Excellence Program of the Ministry for Culture and Innovation from the source of the National Research, Development and Innovation Fund.