Abstract Number: PB1063
Meeting: ISTH 2020 Congress
Background: To prevent bleeding in severe hemophilia A (defined as factor VIII (FVIII) activity < 1%), regular replacement therapy with FVIII concentrates is required. Until recently only products with standard half-life (SHL) were available for prophylaxis. The therapeutic landscape has expanded with the availability of extended-half life products (EHL), which have been developed to reduce the burden of prophylaxis.
Aims: To compare prophylaxis regimen, clinical outcomes, factor utilization and costs in patients with severe haemophilia A, switched from prior prophylaxis with a SHL to efmoroctocog-alfa, a recombinant FVIII-Fc-fusion protein with extended half-life, the first approved EHL used in clinical practice.
Methods: We performed a retrospective cohort study of persons with severe haemophilia A, who were switched from regular prophylaxis with SHL to prophylaxis with EHL (i.e. rFVIII-Fc) in Austria. All persons at participating sites who were switched until December 2018 and who had been treated with the EHL for at least 3 months were eligible. Data was collected from hemophilia-specific patient diaries and medical records. Furthermore, costs with EHL-prophylaxis were compared to prior prophylaxis regimen.
Results: Twenty male persons with severe haemophilia A (median (range) age and weight: 34 (9-75) years and 72.5 (27.5-109) kg) with evaluable data were included in this analysis. The two most common reasons for switching to rFVIII-Fc were high rates of bleeding with SHL and need of less frequent dosing/injections. Switch to rFVIII-Fc resulted in a significantly decreased annualized bleeding rate (ABR) and number of infusions. Factor utilization and costs were comparable to the prior prophylaxis with SHL, a slight increase in costs was not statistically significant. Detailed results are shown in Figure 1.
Conclusions: Switch to EHL (rFVIII-Fc) was associated with an improved clinical outcome, reflected by reduction of the ABR, and less frequent infusions, without a significantly higher factor usage or a significant cost increase.
To cite this abstract in AMA style:Ay C, Feistritzer C, Rettl J, Schuster G, Vavrovsky A, Pabinger I. Bleeding Outcomes, Factor Utilization and Cost Comparisons after Switching to an Extended Half-life Product for Prophylaxis in Haemophilia A in Austria [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/bleeding-outcomes-factor-utilization-and-cost-comparisons-after-switching-to-an-extended-half-life-product-for-prophylaxis-in-haemophilia-a-in-austria/. Accessed May 17, 2021.
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