Objective:
BAX 855 is an extended half-life recombinant Factor VIII based on ADVATE. It was developed as an option to further personalize care and improve outcomes in people with hemophilia A. As a new therapy, it is important to understand patient satisfaction and preferences relative to their prior treatment. The objective of this research was to describe patient satisfaction with and preference for their prior FVIII therapy and BAX 855 as reported in the pivotal trial.
Methods:
BAX 855 was studied in a 6-month, phase 2/3, multi-center, open label study. Subjects in Arm A received 45 ± 5 IU/kg of BAX 855 twice weekly while subjects in Arm B infused BAX 855 on-demand. Patient preference and satisfaction were included as exploratory endpoints. Patient satisfaction was captured with the following options describing their treatment: very satisfied, satisfied, neither satisfied nor dissatisfied, or very dissatisfied at baseline for their prior FVIII therapy and at follow-up for BAX 855. Patient preference for BAX 855 or their prior FVIII therapy was captured at follow-up. Ethics approval and informed consent were obtained. The percent of very satisfied and satisfied patients were combined for this analysis. Results were reported for all patients and by treatment arm.
Summary:
138 patients were in the intent to treat (ITT) sample. The majority of patients in the ITT sample completed the satisfaction questionnaire, however the number varied slightly by question and visit. While 79.4% (100 of 126) of patients were satisfied with their prior FVIII at baseline, satisfaction increased to 90.1% (109 of 121) with BAX 855 and 84.7% (100 of 118) preferred BAX 855 over their prior FVIII. Of the 85 patients in Arm A previously on prophylaxis, 18 patients in Arm A previously on-demand, and 15 patients in Arm B previously on-demand, 83.5%, 88.9% and 86.7% preferred BAX 855 over their prior FVIII, respectively.
Conclusion:
Notably, patients enrolled in the BAX 855 trial had a high degree of satisfaction with their prior FVIII therapy. Nonetheless, treatment with BAX 855 was successful in further improving patient satisfaction with the majority of patients preferring it over their prior FVIII product.