Abstract Number: PB1158
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Novel Biotherapeutics in Hemophilia
Background: In the last years, several extended half-life (EHL) products became available to prophylaxis in persons with Hemophilia A (pwHA) which increased real-world experience. EHL products might lead to higher trough levels without increasing infusion frequency or lead to reduce infusion frequency while maintaining through levels.
Aims: To evaluate outcomes after switching 35 pwHA from standard half-life (SHL) to EHL products from three Portuguese Haemophilia Centers, comparing bleeding rates (BR) and amount of FVIII used, in the same period of time before and after starting EHL.
Methods: Since august 2018, 35 males, mean age 23 yrs (range 2-66), all but one with severe HA, switched from different SHL products to rFVIIIFc. The mean period with ELH was 10.6 months (range 4-16) with 51% of patients with >12 months of observation. Comparisons were performed for BR, joint bleed rates (JBR) and amounts of clotting factor used, before and after switching. Half-lives (HL) of the SHL and EHL products (pharmacokinetics with WAPPS-Hemo platform) were also evaluated in twelve patients.
Results: 50% of patients (17) reduced injection frequency as it was aimed, two started prophylaxis with rFVIIIFc and in the others it was intended higher trough levels. The median BR was 2 (mean 3.9, range 0 – 22) with SHL products and 0 (mean 1.6; 0 – 15) with rFVIIIFc. The median JBR was 1 (mean 2.6; 0 – 13) with SHL products and 0 (mean 1.3; 0 – 14) with rFVIIIFc. The amount of FVIII decreased 18.7%: mean 177900 units/patient with SHL and 144714 units/patient with rFVIIIFc. The mean HL with EHL products (14.9 hrs, range: 8.75-22.5) increased 18.8% comparing with the mean HL with SHL products (12.1 hrs, range: 7.5-14.8).
Conclusions: Our data confirmed lower BRs and reduced injection frequency with increased HL and simultaneous reduction of the amount of FVIII administered.
To cite this abstract in AMA style:
Morais S, Campaniço S, Coutinho M, Rodrigues F, Calheiros M, Pereira M, Pereira A, Guz M, Cruz E, Oliveira C. Optimizing Outcomes in Hemophilia A Prophylaxis Using Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc): Results from Three Portuguese Haemophilia Centers [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/optimizing-outcomes-in-hemophilia-a-prophylaxis-using-recombinant-factor-viii-fc-fusion-protein-rfviiifc-results-from-three-portuguese-haemophilia-centers/. Accessed October 1, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/optimizing-outcomes-in-hemophilia-a-prophylaxis-using-recombinant-factor-viii-fc-fusion-protein-rfviiifc-results-from-three-portuguese-haemophilia-centers/