1. Continued benefit demonstrated with BAY 81-8973 prophylaxis in previously treated children with severe haemophilia A: Interim analysis from the LEOPOLD Kids extension study
- Author
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Victor S. Blanchette, Bryce A. Kerlin, Rolf Ljung, Nikki Church, Valentina Uscatescu, Sonata Saulytė Trakymienė, L. Rusen, Despina Tseneklidou-Stoeter, Horst Beckmann, and Gili Kenet
- Subjects
Pediatrics ,medicine.medical_specialty ,Haemophilia A ,Hemorrhage ,030204 cardiovascular system & hematology ,Haemophilia ,Hemophilia A ,clinical trial ,full-length factor VIII ,haemophilia A ,long-term observation ,prophylaxis ,recombinant proteins ,03 medical and health sciences ,0302 clinical medicine ,medicine ,Humans ,Adverse effect ,Child ,Factor VIII ,business.industry ,Hematology ,Bleed ,medicine.disease ,Interim analysis ,Clinical trial ,Treatment Outcome ,Quartile ,030220 oncology & carcinogenesis ,business ,Bay - Abstract
Introduction BAY 81-8973 (Kovaltry®), a recombinant factor VIII (rFVIII) product, was efficacious and well tolerated in paediatric previously treated patients (PTPs) with severe haemophilia A for ≥50 exposure days (EDs) in the LEOPOLD Kids study. Because long-term prophylaxis (≥100 EDs) can provide substantial patient benefits, FVIII products should demonstrate long-term safety and efficacy. Aim To demonstrate long-term (≥100 EDs) efficacy and safety of BAY 81-8973 in paediatric PTPs. Methods PTPs aged ≤12 years with severe haemophilia A without inhibitors could continue in the ongoing open-label extension study after completing ≥50 EDs in the LEOPOLD Kids main study. Patients received BAY 81-8973 for prophylaxis (25–50 IU/kg ≥2×/week), bleed treatment, and surgery. Bleeds were documented in electronic patient diaries. Inhibitor development was monitored every 6 months. Results At the August 2017 interim data cutoff, 46 patients (median [range] age at enrolment, 6.0 [1.0–11.0] years) had spent a median (range) of 602.5 (148–1069) EDs and 4.6 (1.0–5.9) years in the main plus extension studies. Median (quartile [Q]1; Q3) annualised bleeding rate for bleeds within 48 h after a prophylaxis infusion and total bleeds was 1.0 (0.2; 1.9) and 2.0 (0.4; 3.6), respectively. Most (>94%) bleeds were mild or moderate; 71.8% were treated with ≤1 infusion. BAY 81-8973 was also well tolerated with only one treatment-related adverse event (transient, low-titre inhibitor which did not require treatment adjustment). Conclusion BAY 81-8973 was efficacious for prophylaxis and treatment of bleeds during >4.5 years in paediatric PTPs with severe haemophilia A.
- Published
- 2019