Abstract Number: PB0594
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: In recent years, several studies suggested that extended half-life FVIII products allowed a reduced infusion frequency and a lower clotting factor consumption (CFC), while improving compliance, outcomes and patient’s quality of life (QoL).
Aims: The objective of this study was to evaluate the impact, in prophylaxis optimization and consumption, of switching our patients from standard half-life (SHL) products to rFVIII-Fc.
Methods: We retrospectively evaluated, for 12 months before and after the switching, data on prophylaxis regimen, focusing on prophylactic infusion frequency, all and joint bleeding (ABR a jABR) and CFC. Reasons for switching were also analyzed.
Results: Since october 2018, 25 severe and 1 moderate hemophilia patients, mean age 25 years (range: 5–68), were switched to rFVIII-Fc. Reducing the burden of IV infusion and improving protection and adherence were considered the main reasons for switching. Two patients had an history of inhibitors, and 1 was submitted to a successful immune tolerance induction with rFVIII-Fc. Patients started prophylaxis with the previous dose and prophylaxis regimen was then adapted to individual pharmacokinetic (PK) profiles and clinical needs.
For adults and adolescents, the dosing interval lengthened for 87.5% of subjects. 7/26 (46%) patients were dosed every 3 days; 7/26 (27%) twice a week and 5/26 (19%) every 4 days. In 4 children < 12 years prophylaxis was possible ever 3 days or twice a week. Mean ABR and mean jABR were lower while on prophylaxis with rFVIII-Fc (3.54 vs 0.96 and 2.31 vs 0.58, respectively). Number of patients with zero bleeds was higher with rFVIII-Fc than with SHL (4/26 vs 16/26). After switching to rFVIII-Fc concentrates, the mean annual CFC dropped 25%.
Conclusions: We observed a clear reduction in weekly infusion frequency; a lower annual CFC but more important prophylaxis was efficacious across all ages and was associated with lower ABRs.
To cite this abstract in AMA style:
Campaniço S, Rodrigues F, Pereira A, Amorim J, Correia C, Parusnikova L, Catarino C. 2 Years ofPprophylaxis with rFVIII-Fc in Haemophilia in a Portuguese Haemophilia Center: Did Doctors and Patients See a Change? [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/2-years-ofpprophylaxis-with-rfviii-fc-in-haemophilia-in-a-portuguese-haemophilia-center-did-doctors-and-patients-see-a-change/. Accessed June 25, 2022.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/2-years-ofpprophylaxis-with-rfviii-fc-in-haemophilia-in-a-portuguese-haemophilia-center-did-doctors-and-patients-see-a-change/