@article{913486f3086444a8b6afd5c317c77ece,
title = "Switching from Sucrose-Formulated rFVIII to Octocog Alfa (BAY 81-8973) Prophylaxis Improves Bleed Outcomes in the LEOPOLD Clinical Trials",
abstract = "Introduction: Previous clinical trials established the efficacy and safety of sucrose-formulated recombinant factor (F) VIII (rFVIII-FS/Kogenate FS{\textregistered}/Helixate FS{\textregistered}) and octocog alfa (BAY 81–8973/Kovaltry{\textregistered}; LEOPOLD trials). Aim: To report the results of a post hoc subgroup analysis assessing efficacy and safety outcomes in patients with hemophilia A who were receiving rFVIII-FS prior to enrolling into the LEOPOLD I Part B and LEOPOLD Kids Part A clinical trials and switching to octocog alfa. Methods: LEOPOLD I Part B (NCT01029340) and LEOPOLD Kids Part A (NCT01311648) were octocog alfa Phase 3, multi-national, open-label studies in patients with severe hemophilia A aged 12–65 years and ≤12 years, respectively. Annualized bleeding rate (ABR) was the efficacy endpoint for both studies. Safety endpoints included adverse events (AEs) and development of FVIII inhibitors. Results: Of the 113 patients in both LEOPOLD trials, 40 (35.4%) patients received rFVIII-FS prophylaxis pre-study and had data available for pre-study total ABR. In LEOPOLD I Part B (n = 22, 35.5%), median (Q1; Q3) total ABR decreased from 2.5 (0.0; 9.0) pre-study to 1.0 (0.0; 6.8), and from 1.0 (0.0; 6.0) pre-study to 0.0 (0.0; 6.02) in LEOPOLD Kids Part A (n = 18, 35.3%). Octocog alfa was well tolerated, and no patients had drug-related serious AEs or inhibitors. Conclusion: Treatment with octocog alfa prophylaxis appeared to have a favorable risk–benefit profile compared with rFVIII-FS and thus could be an effective and improved alternative strategy for individualized treatment for children, adolescent and adult patients with severe hemophilia A currently on rFVIII-FS treatment.",
keywords = "FVIII, hemophilia A, octocog alfa, prophylaxis, recombinant proteins",
author = "Gili Kenet and Thomas Moulton and Wicklund, {Brian M.} and Johnny Mahlangu and Ahuja, {Sanjay P.} and Miguel Escobar",
note = "Publisher Copyright: {\textcopyright} 2023 Kenet et al.",
year = "2023",
doi = "10.2147/jbm.s405624",
language = "אנגלית",
volume = "14",
pages = "379--388",
journal = "Journal of Blood Medicine",
issn = "1179-2736",
publisher = "Dove Medical Press Ltd.",
}