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    Hemophilia and Rare Bleeding Disorders

    Publication Only

    PO11 - Impact of simoctocog alfa prophylaxis on bleeding and joint health in Spanish patients with severe haemophilia A

    Wednesday, June 28, 2023
    13:00 – 14:00 ET
    Room: 10
    Background: Patients with severe haemophilia A are at high risk of joint bleeds, leading to joint damage and development of arthropathy. Prophylaxis with factor VIII can reduce bleeds and protect joint health.

    Aims: Monitor joint health in patients with severe haemophilia A on simoctocog alfa (Nuwiq®) prophylaxis in clinical practice.

    Methods: Data were analysed for 11 consecutive patients who received simoctocog alfa prophylaxis at La Paz University Hospital between 2018 and Jan 2023. Bleeding episodes were recorded during prophylaxis. Joint health was assessed at last examination using the Hemophilia Joint Health Score (HJHS) and Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) score.

    Results: Patients had a median (range) age of 29 (4–48) years. Five were previously untreated patients (PUPs; all < 7 years of age), six were previously treated patients (PTPs; aged 29–48 years). Median dose of simoctocog alfa was 43 (35–55) IU/kg. Five PTPs and one PUP were dosed ≤2x/week. All patients adhered to prophylaxis. Since Jan 2022, 2 PUPs and 1 PTP experienced 5 bleeding events (including 2 joint bleeds), of which 3 were spontaneous. One non-spontaneous bleeding event was treated. Median (range) annualised bleeding rates (ABRs) were 0 (0–3) for all and 0 (0–2) for joint bleeds. HJHS and HEAD-US scores were available for 4 PTPs and 4 PUPs. Median (range) HJHS scores were 30 (13–35) for PTPs and 0 in all PUPs. All four PTPs had evidence on HEAD-US of damage in at least one joint. Synovitis was observed in 3 PTPs. No PUPs had indications of joint damage. Physical activity data were available for 8 patients (4 reported moderate activity; 4 none). No inhibitors developed in any patient.

    Conclusion(s): Patients in our centre who received simoctocog alfa prophylaxis had low total and joint ABRs. Joint health will be monitored over time.