Presentation Details
| Title: Real-World Efficacy Data on adolescent Hemophilia A Patients Transitioning to BAY 81-8973 or BAY 94-9027 in the ATHNdataset Martin Chandler1, Jessica Charlet2, Thomas Moulton2, Michael Recht3, 4. 1American Thrombosis & Hemostasis, Rochester, NY, USA.2Bayer Pharmaceuticals, USA, Whippany, NJ, USA.3National Bleeding Disorders Foundation, New York City, NY, USA.4Yale University School of Medicine, New Haven, CT, USA |
Abstract
Background: Real-world data on recombinant factor VIII (rFVIII) product effectiveness are sparse; especially for pediatric patients transitioning between multiple products and changing treatment regimens. The ATHNdataset, sponsored by the American Thrombosis and Hemostasis Network, includes 17,109 people with hemophilia A (PwHA) as of 4/30/22, a potential source of data to evaluate therapeutic effectiveness. Objective: To evaluate the effectiveness and treatment regimens of adolescent PwHA transitioning from multiple previous products to a prophylaxis regimen with BAY 81-8973 (Kovaltry®) or BAY 94-9027 (Jivi®) in a real-world setting. Methods: The ATHNdataset was queried for adolescents aged between 12 and less than 18 years with using BAY 81-8973 or BAY 94-9027. Data included demographics, treatment history, bleed rates and treatment regimens for individuals treating prophylactically. Query dates were between January 1, 2010 and April 30, 2022. Results: A total of 13 adolescents were treating prophylactically with BAY 94-9027 and with some of their previous products, while 22 were treating prophylactically with BAY 81-8973 and with some of their previous products at data cut-off. Thirteen adolescents were treated prophylactically previously with another product and subsequently prophylactically treated with Jivi. All had severe disease. 80% of individuals were treating at least 3x/week with any of their previous products, while after transitioning to BAY 94-9027, 69% had a reduced treatment frequency of 2 or less infusions per week. In conjunction, their mean total annual bleed rate (ABR) was reduced from 1.96 to 0.19 after transitioning to BAY 94-9027. (Figure 1) Of the 22 patients treating with BAY 81-8973, 20 had severe, and 2 had mild disease. All were treating prophylactically previously with another product and subsequently prophylactically treated with BAY 81-8973. Twenty-eight per cent of patients were treated at least every other day with any of their previous products. After transitioning to BAY 81-8973, only 14% were still treated every other day. In conjunction, their mean total ABR was reduced from 1.44 to 0.83 after transitioning to BAY 81-8973. (Figure 2) Conclusions: The data show that adolescents with hemophilia A who transitioned to BAY 94-9027 or BAY 81-8973 experienced overall a decrease in their prophylaxis regimen frequency and mean total annual bleed rates. The therapeutic burden of frequent infusions can be reduced and treatment efficacy can be improved when adolescent patients transition to either BAY94-9027 or BAY81‑8973. These data should be interpreted with caution owing to limitations of real-world studies. Further studies are needed to confirm the impact of switching to BAY94-9027 and BAY81‑8973 in real-world settings.
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