Observational studyHaemophilia : the official journal of the World Federation of Hemophilia
Comprehensive Assessment of the Transition to Efanesoctocog Alfa Prophylaxis in a Paediatric Haemophilia A Cohort.
Observational study 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. Not yet cited in PubMed.
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Abstract
backgroundEfanesoctocog alfa is a novel recombinant factor VIII (FVIII) with an ultra-extended half-life and stable pharmacokinetics, approved for prophylaxis in children with haemophilia A (HA).
objectiveTo describe the experience of transitioning paediatric patients with HA to efanesoctocog alfa using a comprehensive assessment.
methodsProspective observational study in a paediatric haemophilia unit. Patients <18 years without inhibitors were transitioned from prior prophylaxis to efanesoctocog alfa. Treatment regimen, adherence, annual bleeding rate (ABR), pharmacokinetics, joint health, sports, quality-of-life, treatment burden, treatment satisfaction/preference were compared at baseline and during follow-up between efanesoctocog alfa and previous treatment.
resultsEighteen patients (mean age 9.42 years) were included. After 365 ± 84.08 days, adherence improved and no inhibitors were detected. A significant increase in half-life was observed (from 13.57 ± 3.6 to 57.13 ± 6.1 h) which led to a lower ABR score in the series (0.39-0.05) and an improvement of joint health after 6 months (n = 15 patients), due to a relevant synovitis reduction (20% reduction in elbows; 13.3% in ankles) despite patients were moderately-highly physically active. Quality-of-life perception improved after 3 months (EQ-VAS increased: 90.72 ± 10.31 to 95.25 ± 5.3; p = 0.03) as well as treatment burden (HEMO-TEM global score in 12-18 year-old patients: 16.08 ± 11.43 to 11.72 ± 14.8). Patients/caregivers expressed a strong preference for efanesoctocog alfa.
conclusionTransitioning to efanesoctocog alfa was safe and effective in this paediatric cohort. Despite limited follow-up, data suggest this therapy offers meaningful clinical and psychosocial benefits.
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