ArticleThrombosis journal2026
Transition to Emicizumab from standard-dose FVIII prophylaxis in young children with hemophilia A: a real-world study.
Article in Thrombosis journal, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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Who cites it
1 citing paper in PubMed.
- Plasma emicizumab concentrations and bleeding rates in children and adults with severe hemophilia A.Research and practice in thrombosis and haemostasis · 2026Observational
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Authors and funding
2 authors.
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No grant is acknowledged in the PubMed record.
Abstract
backgroundReal-world evidence on transition from standard-dose FVIII to Emicizumab prophylaxis in young children with hemophilia A remains limited, particularly in developing countries.
methodsWe performed a single-center, retrospective, self-controlled study in boys with severe hemophilia A without inhibitors. Outcomes during 12 months of standard-dose factor VIII (FVIII) prophylaxis were compared with outcomes during 12 months after transition to Emicizumab prophylaxis (in this real-world cohort, most patients received reduced-dose maintenance regimens). Annualized bleeding rates (overall, treated, joint, and spontaneous) and zero-bleeding proportions were assessed. Direct annual prophylaxis drug costs were calculated using locally applicable drug prices and converted to USD.
resultsThirty-one boys were included with a median age of 4.5 years. During FVIII prophylaxis, the median FVIII dose was 32.3 IU/kg/infusion with a median 3.1 infusions/week; median trough FVIII was 3.9 IU/dL. After transition, mean Emicizumab exposure was 4.2 mg/kg/month (range 3.0-6.0) with a mean dosing interval of 14 days. Overall ABR decreased from 2.48 to 0.32, treated ABR from 1.64 to 0.29, joint ABR from 0.83 to 0.13, and spontaneous ABR from 0.26 to 0 (all P < 0.05). The proportion of patients with zero bleeds increased from 35% to 81% (P < 0.001), and zero treated bleeds from 52% to 69% (P < 0.05). Mean annual prophylaxis drug costs were lower with Emicizumab than with FVIII (34,417 vs. 47,168 USD; P < 0.001).
conclusionsIn this selected pediatric cohort of children who transitioned from standard-dose FVIII to Emicizumab and remained on treatment for at least 12 months, lower observed bleeding rates and lower direct prophylaxis drug costs were seen during the Emicizumab period. These findings should be interpreted descriptively because the retrospective design and eligibility criteria introduced substantial selection bias.
trial registrationNot applicable.
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