Background: Recent additions to the hemophilia prophylaxis success story are extended half-life products (EHLs) and non-factor replacement therapies. As a consequence, prophylaxis now ambitiously aims at achieving zero bleeds in patients, instead of simply converting their phenotype from severe to moderate.

Aims: To evaluate progress made in achieving treatment goals over a 3 year period in the real world setting of a pediatric hemophilia center.

Methods: Records of severe hemophilia patients on prophylaxis were retrospectively studied with regards to age, product type, annualized bleeding rate (ABR) and joint ABR for years 2018, 2019 and 2020.

Results: In 2018, 21 patients with a mean age of 12.4 (5-18) years were evaluated, out of which 16 (76.2%) were on standard half-life products (SHLs), 2 (9.5%) on EHLs, whereas 3 (14.3%) switched  from SHL to EHL during the study year. Mean ABR was 4.5 (0-13) and mean AjBR 3.2 (0-11). In 2 (9.5%) patients zero bleeds were reported. 
In 2019, 22 patients with a mean age of 11.9 (3-18) years were evaluated, out of which 13 (59%) were on SHLs, 5 (22.7%) on EHLs, whereas 2 (9%) switched during study year. Emicizumab was administered to 2 (9%) patients. Mean ABR was 4 (0-10) and mean AjBR 3 (0-9). Zero bleeds were reported in 4 patients (18.3%).
In 2020, 26 patients with a mean age of 10.5 (1-18) years were evaluated. In total, 14(53.8%) were on SHLs, 8 (30.7%) on EHLs, 1 (3.84%) switched from SHL to EHL and 3 received emicizumab (11.5%). Mean ABR was 1.8 (0-8) and mean AjBR 1.1 (0-8). Zero bleeds were reported in 6 patients (23%).

Conclusions: Improvement in offered prophylaxis is attainable with the use of new therapies. However, the ultimate goal of zero bleeds is still difficult to achieve in the real world setting.

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/is-zero-bleeds-a-realistic-goal-for-hemophilia-patients-in-the-real-world-setting/