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    Poster Networking

    Pediatrics

    Poster Networking Session

    PB0251 - The impact of switching from intravenous recombinant factor VIII concentrate to a non-factor concentrate on health-related quality of life in boys with hemophilia A

    Sunday, June 25, 2023
    18:30 – 19:30 ET
    Room: Exhibition
    Background: In Canada, treatment for hemophilia A has consisted of regular intravenous infusions of clotting factor VIII concentrates to prevent bleeds and maintain joint health. Novel non-factor therapies can be administered subcutaneously and less frequently. Health-related quality of life (HR-QoL) is critical in determining the impact of novel therapies. The Canadian Hemophilia Outcomes-Kids Life Assessment Tool (CHO-KLATv3.0) and Hemophilia Family Impact Tool (H-FIT) are validated tools that measure HR-QoL and the impact of hemophilia on persons with hemophilia (PWH) and caregivers.

    Aims: To compare HR-QoL of, and burden of caring for, PWH, pre/post-switch to emicizumab.

    Methods: Persons aged 0-18 with moderate/severe hemophilia A on prophylaxis switching to emicizumab and their caregivers completed questionnaires at product switch (baseline), 3 and 6-months post-switch. PWH aged 7-18 completed the self-report CHO-KLAT and caregivers completed the parent-report CHO-KLAT and H-FIT. Spearman’s correlations were calculated to compare self/parent-report CHO-KLAT scores. Wilcoxon signed ranks tests were used to compare CHO-KLAT and H-FIT scores pre/post-switch.

    Results: Caregivers (n=31) and PWH (n=23) completed the questionnaires. Median age of PWH was 11 years (4-18, 100% male), with most (96.8%) infusing FVIII concentrates >2 times/week pre-switch. Both questionnaires are scored 0-100, with 100 representing best HR-QoL and least family burden, respectively. There was a statistically significant correlation between child and caregiver CHO-KLAT at baseline (ρ = 0.64*, CI=.29-.84) and 3-months (ρ =0.63*, CI=.11-.88), indicating similar caregiver-perceived, and child, HR-QoL. CHO-KLAT and HFIT scores improved from pre- to 3-months post-switch but did not achieve statistical significance. From 3-months to 6-months, post-switch scores deteriorated slightly.

    Conclusion(s): Patients reported highest CHO-KLAT scores 3-months post-switch. These findings are supported by HR-QoL literature where a ‘leveling out’ occurs after adjusting to treatment changes. Further studies are needed to expand on these preliminary results and may be relevant for improving hemophilia care.