A national survey of four decades of hereditary angioedema prophylaxis: Efficacy and safety of old and new drugs.

Background: Hereditary angioedema (HAE) imposes a significant burden on patients due to the unpredictability of attacks. Long-term prophylaxis (LTP) aims to prevent these episodes and improve quality of life. Over the past five decades, LTP options have evolved from non-specific treatments (fresh frozen plasma, attenuated androgens, antifibrinolytics) to more targeted therapies (intravenous or subcutaneous C1 inhibitor (C1INH), lanadelumab, berotralstat).

Objective: To assess the use, efficacy, and safety of various LTP therapies among Hungarian HAE patients.

Methods: Data from the Hungarian HAE Registry (1979-2023) were analysed, including LTP usage, attack rates, side effects, and laboratory parameters.

Results: Danazol and tranexamic acid use increased over the first 30 years but has declined in the last decade. Conversely, the use of modern LTP therapies and the proportion of patients relying solely on on-demand medication have significantly increased over the past decade. Danazol and tranexamic acid reduced attack rates in 60 % of patients without significant changes in the laboratory parameters at the lowest effective doses. Furthermore, 100 % of patients on subcutaneous C1INH, lanadelumab, and berotralstat experienced fewer attacks and improved quality of life compared to pre-treatment periods, with no serious side effects or significant laboratory abnormalities.

Conclusions: Modern prophylaxis usage is increasing among Hungarian HAE-C1INH patients, following the global trends. Both modern and traditional LTP options proved safe and effective in our patient population when used with appropriate monitoring.