PB0534 - Improved well-being in people with hemophilia A and inhibitor in emicizumab prophylaxis

Background: Bleeding and treatment particularities directly impact the well-being of people with hemophilia A (PwHA). Prophylaxis with emicizumab presents a reduction in bleeding, with less invasive treatment regimens and a lower frequency of administration. Despite this, little is known about the impact of emicizumab prophylaxis on the well-being of PwHA.

Aims: We aimed to evaluate well-being in PwHA on emicizumab prophylaxis.

Methods: PwHA on emicizumab prophylaxis for at least 1 year were invited at several Brazilian Hemophilia Treatment Centers. Clinical and treatment data before (12 months) and during (12 months) emicizumab prophylaxis were collected. Bleeds were classified according to their cause as spontaneous, post-traumatic, and unknown, and only counted if they received any treatment for hemostasis. Well-being was self-assessed using the Hemophilia Well-Being Index (HWBI). Higher scores correspond to better well-being.

Results: Of the total of 8 PwHA included, 6/8 (75%) had severe hemophilia A. All (8/8; 100%) failed immune tolerance induction and had inhibitors, receiving prophylaxis with bypassing agents. The median age at emicizumab prophylaxis start was 17 years (range 6-71). The total number of treated bleeds reduced from 20 (10 post-traumatic) to 2 (1 spontaneous and 1 post-traumatic), before and during emicizumab prophylaxis, respectively. The median (range) annual bleeding rate reduced from 3 (0-5) to 0 (0-1) and the number of PwHA with zero bleed increased from 2/8 (25%) to 6/8 (75%), before and during emicizumab prophylaxis, respectively. The median (range) of the HWBI total score increased from 19 (7-28) before to 27 (19-32) in the first year of emicizumab prophylaxis.

Conclusion(s): In this small PwHA and inhibitor population, well-being improved after 1 year of emicizumab prophylaxis.