ArticleHaemophilia : the official journal of the World Federation of Hemophilia
Beyond Standard Half-Life: Real-world Pharmacokinetics of Efanesoctocog Alfa in a Single Centre.
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. Not yet cited in PubMed.
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Abstract
introductionEfanesoctocog alfa (EFA) is an ultra-extended half-life factor VIII (FVIII) developed to address limitations of conventional prophylaxis in haemophilia A. Although the XTEND trials reported low interindividual pharmacokinetic (PK) variability, real-world data remain important to better characterize PK profiles across patient subgroups.
aimTo report real-world PK data for EFA.
methodsIn this single-centre study, adults and children (>6 years) with severe haemophilia A at Strasbourg University Hospital switched from a prior FVIII product to EFA. A simplified PK assessment was performed once or twice per patient after a 50 IU/kg infusion. FVIII activity was measured pre-infusion and at 3, 24, 96 h, and 7 days post-infusion. Patients were reassessed at steady state after ≥6 weeks.
resultsIn adults, mean (range) half-life was 52 h (35-62). Mean incremental recovery at 3 h was 2.98 IU/dL per IU/kg (1.85-3.82). Mean FVIII trough levels were 17 IU/dL (5-27) at switch and 19 IU/dL (10-27) at steady state. Significant interindividual variability was observed. Half-life correlated positively with VWF:Ag levels (r = 0.63; p < 0.05) and body weight (r = 0.62; p < 0.05). In children, mean half-life was 53 h (48-59), mean recovery 2.05 IU/dL per IU/kg (1.86-2.26), and trough levels 13-14 IU/dL.
conclusionsPK profiles matched or exceeded those reported in the XTEND studies, with a slightly longer half-life and higher trough FVIII levels. Notable interindividual variability highlights the value of PK assessments, even for ultra-extended half-life FVIII, to optimize individualized patient management.
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