Abstract Number: PB0484
Meeting: ISTH 2021 Congress
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.
To cite this abstract in AMA style:Adramerina A, Teli A, Symeonidis S, Nianiu A, Economou M. Is Zero Bleeds a Realistic Goal for Hemophilia Patients in the Real World Setting? [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 1). https://abstracts.isth.org/abstract/is-zero-bleeds-a-realistic-goal-for-hemophilia-patients-in-the-real-world-setting/. Accessed September 16, 2021.
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