Effectiveness of Octocog Alfa (BAY 81‐8973) to Treat People With Haemophilia A Enrolled in the ATHNdataset, Including Under‐Represented Subgroups

To evaluate the real-world effectiveness of octocog alfa (BAY 81-8973; Kovaltry) in people with haemophilia A (PwHA), including under-represented subgroups. We retrospectively analysed the American Thrombosis and Hemostasis Network (ATHN) dataset to identify the characteristics, treatment/clinical histories and annualised bleeding rates (ABRs) of PwHA receiving prophylactic or on-demand octocog alfa. Subgroup analyses included females and those switching to octocog alfa. Data are reported descriptively. Among 270 PwHA treated with octocog alfa with available bleeding rate data between 1 January 2010 and 31 October 2020, mean (standard deviation) total ABRs were low (0.59 [2.22]) and > 70% of those treated for > 12 months had 0 bleeds. At the second data cutoff (30 April 2022; n = 354), subgroups who switched to octocog alfa from previous treatment had low mean total ABRs after switching, including adolescents (0.83; n = 22), and those previously receiving emicizumab (0.17; n = 17). In females, 10/11 reported 0 bleeds, and of those with joint range of motion data available (n = 5), 100% had values ≥ 10% below the normal range in ≥ 2 joints. PwHA treated with octocog alfa in the ATHNdataset, including important subgroups, reported a low number of bleeds; thus, octocog alfa offers an effective therapeutic option.