Real-World Age-Stratified FVIII Consumption and Bleed Outcomes Before and After Switching to Rurioctocog Alfa Pegol in a Retrospective, Observational Study Using US Specialty Pharmacy Data

Introduction: The safety and efficacy of rurioctocog alfa pegol (BAX 855, SHP-660, TAK-660; Adynovate®; Baxalta US Inc., a Takeda company, Lexington, MA, USA) in patients with severe hemophilia A has been reported previously (Konkle BA et al., Blood 2015, 126:1078-85; Brand B et al., Haemophilia 2016, 22:e251-8; Mullins ES et al., Haemophilia 2017, 23:238-46); however, research describing patient experience with extended half-life (EHL) recombinant factor VIII (FVIII) products outside clinical trials is limited. The objective of this study was to assess real-world utilization of TAK-660 in patients with hemophilia A and describe their clinical profiles before and after switching to TAK-660. Factor consumption and bleed outcomes stratified by age ( Methods: This was a retrospective, observational database study of patient data from US specialty pharmacies. Pharmacy data sources included patient records, prescriptions, and patient-reported bleed logs. Informed consent was obtained for all analyzed patient data. Eligible patients with hemophilia A were treated with prophylactic TAK-660 with on-label dosing from November 2015 to September 2018, and had received ≥90 days of FVIII (standard half-life [SHL] or EHL) therapy before switching to TAK-660. Main exclusion criteria were participation in a TAK-660 clinical trial before/during this study, only on-demand treatment before switching to TAK-660, or presence of active FVIII inhibitor requiring treatment and/or use of immune tolerance induction during the study period. Assessments included prior hemophilia therapy, FVIII administration frequency and consumption, and annualized bleeding rate (ABR) before and after switching to TAK-660. Results: Data was collected from 82 patients (of 61 providers in 44 practices across 25 states in the United States): 44% of the patients (36/82) were Conclusions: In patients with hemophilia A previously treated with SHL- or EHL-FVIII products, switching to TAK-660 prophylaxis resulted in a significant decrease in ABR of 40-50% in both age groups analyzed. The adult population (ie, ≥18 years old) showed a tendency for reduced weekly FVIII consumption. These findings from real-world data are in agreement with TAK-660 clinical trial results. The observed differences in FVIII consumption between patients Disclosures Watt: Shire International GmbH, a Takeda company: Employment, Other: a Takeda stock owner. Milligan:Sanofi: Research Funding; Merck: Research Funding; Gilead: Research Funding; Amgen: Research Funding; AbbVie: Research Funding; Trio Health: Employment; Viiv: Research Funding.