PhD Scientific Days 2025
Budapest, 7-9 July 2025
Theoretical and Translational Medicine III.
Evolution of Hereditary Angioedema Prophylaxis in Hungary Over Four Decades
Előadó neve
Dr. Horváth Hanga Réka
Neptun code
C9JAKS
Előadó munkahelye
Department of Internal Medicine and Haematology, Semmelweis University
Előadó telefonszáma
+36209921414
Előadó e-mail címe
horvath.hanga@stud.semmelweis.hu
Az előadás címe
Evolution of Hereditary Angioedema Prophylaxis in Hungary Over Four Decades
Szerző(k) neve és munkahelye
Hanga Réka Horváth1, Beáta Visy1,2, Kinga Viktória Kőhalmi1,3,4, Zsuzsanna Balla1,5, Noémi Andrási1,6, Ibolya Czaller1,7, Zsuzsanna Zotter1,8, 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
Bemutatás módja
Szóbeli
Szekció
Theoretical and Translational Medicine III.
Language of the presentation
English
Preferred session
Theoretical and Translational Medicine
Összefoglaló szövege
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.
University
Semmelweis University
Supervisor
Prof. Dr. Henriette Farkas
Publication of my abstract
I give consent to the publication of my abstract on the website of the congress.
phd.section.field
in doctoral studies after complex exam (PhD)
Kind
Szabad
Status
elfogadva
Accepted presentation method
szóbeli
Előadás fájl jóváhagyás
jóváhagyta
Előadó
6960
Start
15:45
End
16:00