Haemophilia : the official journal of the World Federation of Hemophilia · 2026
Real-World Effectiveness and Safety of Concizumab Prophylaxis in Hemophilia: Results From a National French Cohort.
СтатьяНаблюдательное исследованиеМногоцентровое исследованиеНазвание и аннотация: Перевод в очереди
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Galeotti Caroline, Rauch Antoine, Gatti Helene, Desage Stephanie, Butelet Alexandre, Biron-Andreani Christine, Susen Sophie, d'Oiron Roseline, Chanteau Guillaume, Proulle Valerie, Dargaud Yesim
Аннотация
BACKGROUND: Inhibition of tissue factor pathway inhibitor represents an advanced rebalancing strategy for hemophilia, applicable across hemophilia A and B irrespective of inhibitor status. Although concizumab has demonstrated efficacy in clinical trials, real-world data remain very limited.
OBJECTIVES: To evaluate the real-world effectiveness and safety of concizumab prophylaxis in an unselected national cohort of patients treated in routine clinical practice in France.
METHODS: This cross-sectional observational study included all patients receiving concizumab through compassionate-use or Early Access Programs across six French hemophilia centers. Retrospective data were collected from treatment initiation to a predefined cutoff (1, December 2025). Outcomes included bleeding rates, target joint status, surgical management, laboratory monitoring practices, adherence, and adverse events.
RESULTS: Eleven patients were included, ten with hemophilia B with inhibitors and one with hemophilia B without inhibitors. Mean exposure to concizumab was 22.8 months. Compared with the pre-treatment period, concizumab prophylaxis was associated with a marked reduction in bleeding, including complete absence of bleeding in four patients and resolution of target joints in nine of ten patients. Nine surgical procedures, including two major surgeries, were performed under concizumab prophylaxis with concomitant rFVIIa without thrombotic complications. No thrombotic events or injection-site pain were reported. None of the patients discontinued concizumab prophylaxis.
CONCLUSIONS: In this nationwide real-world cohort, concizumab prophylaxis was effective, well tolerated, and manageable in routine practice, including during surgical procedures. These data support its use as a valuable prophylactic option in carefully selected patients with hemophilia B and inhibitors, while underscoring the importance of individualized monitoring and thrombotic risk assessment.
OBJECTIVES: To evaluate the real-world effectiveness and safety of concizumab prophylaxis in an unselected national cohort of patients treated in routine clinical practice in France.
METHODS: This cross-sectional observational study included all patients receiving concizumab through compassionate-use or Early Access Programs across six French hemophilia centers. Retrospective data were collected from treatment initiation to a predefined cutoff (1, December 2025). Outcomes included bleeding rates, target joint status, surgical management, laboratory monitoring practices, adherence, and adverse events.
RESULTS: Eleven patients were included, ten with hemophilia B with inhibitors and one with hemophilia B without inhibitors. Mean exposure to concizumab was 22.8 months. Compared with the pre-treatment period, concizumab prophylaxis was associated with a marked reduction in bleeding, including complete absence of bleeding in four patients and resolution of target joints in nine of ten patients. Nine surgical procedures, including two major surgeries, were performed under concizumab prophylaxis with concomitant rFVIIa without thrombotic complications. No thrombotic events or injection-site pain were reported. None of the patients discontinued concizumab prophylaxis.
CONCLUSIONS: In this nationwide real-world cohort, concizumab prophylaxis was effective, well tolerated, and manageable in routine practice, including during surgical procedures. These data support its use as a valuable prophylactic option in carefully selected patients with hemophilia B and inhibitors, while underscoring the importance of individualized monitoring and thrombotic risk assessment.