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

OBJECTIVES

To evaluate the real-world effectiveness of octocog alfa (BAY 81-8973; Kovaltry) in people with haemophilia A (PwHA), including under-represented subgroups.

METHODS

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.

RESULTS

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.

CONCLUSIONS

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.