BAY 81-8973 demonstrates long-term safety and efficacy in children with Severe haemophilia A: results from the LEOPOLD kids extension study /

Rolf Ljung; Anthony K. C Chan; Sanjay P Ahuja; Maria Elisa Mancuso; Jose Francisco Cabre Marquez; Florian Volk; Victor Blanchette; Bryce A Kerlin; Sonata Šaulytė Trakymienė; Heidi Glosli; Gili Kenet

doi:10.1111/ejh.14362

Title	BAY 81-8973 demonstrates long-term safety and efficacy in children with Severe haemophilia A: results from the LEOPOLD kids extension study /
Authors	Ljung, Rolf ; Chan, Anthony K. C ; Ahuja, Sanjay P ; Mancuso, Maria Elisa ; Marquez, Jose Francisco Cabre ; Volk, Florian ; Blanchette, Victor ; Kerlin, Bryce A ; Šaulytė Trakymienė, Sonata ; Glosli, Heidi ; Kenet, Gili
DOI	10.1111/ejh.14362
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Is Part of	European journal of haematology.. John Wiley and Sons Inc. 2025, vol. 114, iss. 3, p. 556-565.. ISSN 0902-4441. eISSN 1600-0609
Keywords [eng]	clinical trial ; factor VIII ; haemophilia A ; long-term ; prophylaxis
Abstract [eng]	Objectives: To report the long-term safety and efficacy of BAY 81-8973 in the LEOPOLD Kids extension phase. Methods: Patients received BAY 81-8973 (25–50 IU/kg) at least twice weekly. The primary endpoint was safety, assessed in all patients who entered the extension phase (n = 82). Efficacy endpoints were assessed in patients without high-titre inhibitors/immune tolerance induction (n = 67). Results: Children (n = 82) received BAY 81-8973 for a median of 3.1 years per patient and a median of 405 exposure days per patient. Long-term BAY 81-8973 treatment was well tolerated, with no cases of de novo inhibitor development in the extension phase. Annualised bleeding rates (ABRs) within 48 h of prophylaxis were low for all bleeds (median [IQR], 0.7 [0–1.9]; mean, 1.4 [SD, 2.1]) and for joint bleeds (median [IQR], 0 [0–0.7]; mean, 0.5 [SD, 1.1]) (n = 67). Twenty-one of 67 patients (31.3%) had zero bleeds within 48 h of prophylaxis; the treatment response was ‘good’/‘excellent’ in 87.9% of bleeds, and most bleeds resolved with ≤ 2 BAY 81-8973 infusions (83.5%). Conclusion: Long-term BAY 81-8973 treatment is well tolerated and maintains low ABRs for all bleeds and joint bleeds in children with severe haemophilia A. Trial Registration: ClinicalTrials.gov identifier: NCT01311648.
Published	John Wiley and Sons Inc
Type	Journal article
Language	English
Publication date	2025
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„BAY 81-8973 demonstrates long-term safety and efficacy in children with Severe haemophilia A: results from the LEOPOLD kids extension study /“