Abstract Number: PB0564
Meeting: ISTH 2021 Congress
Background: Phase III pivotal studies have proven the safety and efficacy of extended half-life agents. However, studies under everyday clinical practice conditions in national health care environments are still limited, although they can offer valuable information.
Aims: Aim of this study is to collect data on outcomes of severe and moderate haemophilia A patients, who switched from prophylactic or on demand treatment with standard half-life factor VIII to rFVIIIFc.
Methods: This is a multicenter observational study, in all 5 haemophilia centres in Greece. Until interim data lock point, 59 patients were enrolled and included in the current analysis. 45 patients had already completed the 12 months of follow-up after switch.
Results: Mean(range) age of patients was 31(6-56) years and 50(84.7%) had severe haemophilia A. For patients on prophylaxis before and after the switch (n=42), there was statistically significant decrease in meanABR from 3.1 to 1.5(p= 0.002), while the percentage of patients with zero bleeds increased from 35.7% to 62.2%., Of total bleeds in patients started rFVIIIFc, 56.8% were spontaneous vs 76.6% before switch. Decrease in ABR was more profound in patients (n=8) transferred from on demand to prophylaxis (mean8.0 before switch to 0.7 after, p=0,047). Significant decrease in ABR was also noted in the subgroup of patients (n=21) on secondary prophylaxis (4.4 before switch to 2.0 after, p=0,008), compared to the subgroup (n=20) on primary prophylaxis(2.2 before to 0.9 after, p=0,153). Joint health improved after switch (mean HJHS score: 14.9 before to 11.7 after, p=0,008),as was thelevel of pain experienced by the patients (mean VAS score: 4.9 before to 3.5 after, p<0.001). Nine adverse events, none of them related to rFVIIIFc and no inhibitors were observed.
Conclusions: These interim data from the first multicenter study concerning people with haemophilia A in Greece, are encouraging for the safety and effectiveness of newer drugs with improved pharmacokinetics.
To cite this abstract in AMA style:Katsarou O, Kouramba A, Nomikou E, Kampelos G, Vakalopoulou S, Moka H, Economou M, Gourtsa V, Michalopoulou E, Pergantou H. Real World Data on Haemophilia A Patients Switching to rFVIIIFc Treatment in Greece. Interim Results from the Multicenter Observational Study TOOL [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 1). https://abstracts.isth.org/abstract/real-world-data-on-haemophilia-a-patients-switching-to-rfviiifc-treatment-in-greece-interim-results-from-the-multicenter-observational-study-tool/. Accessed September 23, 2021.
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