Haemophilia : the official journal of the World Federation of Hemophilia · 2025
Integration of Efanesoctocog Alfa in Clinical Practice for Children, Adolescents, and Young Adults With Severe Haemophilia A.
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Jiang Debbie, Johnston Natalie, Echelman Daniel, Croteau Stacy E
Аннотация
INTRODUCTION: Efanesoctocog alfa is a novel, "ultra-extended half-life" FVIII concentrate for bleed treatment and prevention in haemophilia A. Clinical trials excluded individuals with active or prior FVIII inhibitors, those on emicizumab, and previously untreated patients (PUPs).
AIM: This retrospective, single-centre study evaluated the management and outcomes of pediatric clinic patients who initiated efanesoctocog alfa for haemophilia A bleed treatment or prevention.
METHODS: Clinical characteristics, pharmacokinetic data, hemostatic outcomes, and non-standardised patient and caregiver feedback were collected by medical record review.
RESULTS: Overall, 24 patients initiated efanesoctocog alfa, including seven who switched from emicizumab, five who previously achieved immune tolerance induction for FVIII inhibitors, and one PUP, who was successfully treated with a single infusion for his initial joint bleed. Mean FVIII activity on day 6 was 13.4 ± 6.1 IU/dL by one-stage assay for patients who switched from FVIII concentrate prophylaxis. Five switched from emicizumab without a washout, preventing the use of a one-stage assay, and had a mean chromogenic FVIII activity of 23.8 ± 5.2 IU/dL on day 6. Compared with prior prophylaxis, efanesoctocog alfa was associated with a lower mean treated annualised bleed rate (1.3 ± 1.5 vs 0.1 ± 0.3, p = 0.001) and annualised emergency department bleed evaluations (0.8 ± 1.4 vs 0.1 ± 0.4, p = 0.04). No recurrence of FVIII inhibitors occurred over a median follow-up of 22.6 months (range: 6.4-25.4). Patients/caregivers reported improvements in musculoskeletal discomfort, perception of hemostatic coverage, and confidence to pursue physical activity.
CONCLUSION: Individuals with clinically severe haemophilia A, including subgroups previously excluded from clinical trials, successfully transitioned to efanesoctocog alfa with excellent hemostatic outcomes and no inhibitor recurrence.
AIM: This retrospective, single-centre study evaluated the management and outcomes of pediatric clinic patients who initiated efanesoctocog alfa for haemophilia A bleed treatment or prevention.
METHODS: Clinical characteristics, pharmacokinetic data, hemostatic outcomes, and non-standardised patient and caregiver feedback were collected by medical record review.
RESULTS: Overall, 24 patients initiated efanesoctocog alfa, including seven who switched from emicizumab, five who previously achieved immune tolerance induction for FVIII inhibitors, and one PUP, who was successfully treated with a single infusion for his initial joint bleed. Mean FVIII activity on day 6 was 13.4 ± 6.1 IU/dL by one-stage assay for patients who switched from FVIII concentrate prophylaxis. Five switched from emicizumab without a washout, preventing the use of a one-stage assay, and had a mean chromogenic FVIII activity of 23.8 ± 5.2 IU/dL on day 6. Compared with prior prophylaxis, efanesoctocog alfa was associated with a lower mean treated annualised bleed rate (1.3 ± 1.5 vs 0.1 ± 0.3, p = 0.001) and annualised emergency department bleed evaluations (0.8 ± 1.4 vs 0.1 ± 0.4, p = 0.04). No recurrence of FVIII inhibitors occurred over a median follow-up of 22.6 months (range: 6.4-25.4). Patients/caregivers reported improvements in musculoskeletal discomfort, perception of hemostatic coverage, and confidence to pursue physical activity.
CONCLUSION: Individuals with clinically severe haemophilia A, including subgroups previously excluded from clinical trials, successfully transitioned to efanesoctocog alfa with excellent hemostatic outcomes and no inhibitor recurrence.