PB0556 - Evolution of the therapeutic arsenal used in Hemophilia A: experience in a Spanish Haemophilia Treatment Center

Background: The treatment of patients with hemophilia A (PWHA), based on the intravenous administration of factor concentrates, has presented great changes in recent years to solve some challenges: the short half-life of the so-called “standard half-life (SHL)” drugs, the intravenous administration and the development of inhibitors.
In recent years, new drugs have been developed and have appeared extended half-life recombinant factors (EHL), mimetic drugs, hemostasis rebalancing therapy and gene therapy.

Aims: To perform a comparative analysis between the therapies used in our center in PWHA over time.

Methods: Observational and retrospective analysis of therapeutic evolution in PWHA in a Haemophilia Treatment Center (HTC) between 2015-2023.

Results: A total of 79 PWHA undergoing prophylactic treatment; 24% are under 12 years-old.
The results in global population are: in 2015, 90% of patients received treatment with SHL; in period 2017-2019, patients receiving treatment with EHL began to increase (figure 1); in 2021, the number of patients treated with SHL and EHL was equal, with SHL being used in only 10% in 2023; in 2022 the number of patients treated with mimetic drugs rises (28%), and in 2023 it surpasses EHL therapy (44% vs 39%).

Results in population under 12 years-old: Start of using EHL in 2021 (21% of patients in 2023); use of emicizumab starts in 2021; currently it accounts for 63% of patients, compared to 15% treated with SHL (figure 2).

Regarding subcutaneous drugs (SD), in 2017 the first treatment (emicizumab) began. After this, the number of patients treated with some SD (mimetics or rebalancing therapy) has been increasing exponentially (currently 48%).

Conclusion(s): Since 2017, there has been a progressive change regarding the treatment of PWHA.
The appearance of EHL factors has meant, together with mimetic drugs, a change in the therapeutic paradigm.
In pediatric population, the use of emicizumab has become the predominant therapy in 2023.