ArticleHaemophilia : the official journal of the World Federation of Hemophilia
Integration of Efanesoctocog Alfa in Clinical Practice for Children, Adolescents, and Young Adults With Severe Haemophilia A.
Article in Haemophilia : the official journal of the World Federation of Hemophilia. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
1 citing paper in PubMed.
- Comprehensive Assessment of the Transition to Efanesoctocog Alfa Prophylaxis in a Paediatric Haemophilia A Cohort.Haemophilia : the official journal of the World Federation of HemophiliaObservational
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Authors and funding
4 authors.
Funding
Abstract
introductionEfanesoctocog 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).
aimThis retrospective, single-centre study evaluated the management and outcomes of pediatric clinic patients who initiated efanesoctocog alfa for haemophilia A bleed treatment or prevention.
methodsClinical characteristics, pharmacokinetic data, hemostatic outcomes, and non-standardised patient and caregiver feedback were collected by medical record review.
resultsOverall, 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.
conclusionIndividuals 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.
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