Abstract Number: PB0463
Meeting: ISTH 2021 Congress
Background: People with severe hemophilia B (HB) continue to experience bleeds and joint morbidity, despite therapeutic advancement and proliferation of prophylactic factor IX (FIX) therapy across Europe.
Aims: The objective of this research is to examine real-world outcomes of severe hemophilia B across Europe, among people receiving FIX prophylaxis.
Methods: We report 12 months’ outcomes using CHESS II (‘Cost of Hemophilia in Europe: a Socioeconomic Survey II’), a retrospective burden-of-illness study conducted in 2019-2020. People with non-severe HB, FIX inhibitors, or treated on-demand were excluded from the analysis. Results are presented as mean (standard deviation) or N (%).
Results: Of 132 people with severe HB in CHESS II, 75 subjects met the eligibility criteria and were included in the analysis. The mean age (years) and weight (kg) were 36.17 (15.97) and 77.27 (11.07). Respectively, 42 and 33 subjects received standard and extended half-life (SHL and EHL) FIX. Many experienced joint health issues, while the mean annualized bleeding rate was 2.40 (2.14).
The mean annual total medical cost per patient was €235,723 (€154,953), of which FIX therapy accounted for 98% of total cost. Mean annual FIX consumption was 245,175 IU (144,903) and the overall cost of FIX was €232,328 (€153,979). Average SHL FIX consumption and cost were 286,969 IU (153,211) at €186,528 (€116,403), while average EHL FIX consumption and cost were 191,680 IU (115,180) and €290,620 (€176,665), respectively. Mean EQ-5D-5L score was 0.67 (0.21), annual non-medical costs were €1,997, and indirect costs were €8,973 (among a subset of subjects who completed the patient-reported outcome survey: N=40).
Conclusions: Despite recent treatment advancements, this analysis of CHESS II shows that burden and unmet needs remain among people with severe HB treated with FIX prophylaxis in Europe.
To cite this abstract in AMA style:Burke T, Asghar S, O'Hara J, Chuang M, Sawyer E, Li N. Real-world Outcomes in People with Severe Hemophilia B Receiving FIX Prophylaxis across Europe: A CHESS II Analysis [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/real-world-outcomes-in-people-with-severe-hemophilia-b-receiving-fix-prophylaxis-across-europe-a-chess-ii-analysis/. Accessed May 19, 2022.
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