Abstract Number: PB0919
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: Prophylactic factor VIII (FVIII) is the standard of care for managing hemophilia A. However, real-world patient experience with extended half-life (EHL) recombinant FVIII is limited.
Aims: To analyze real-world outcomes in patients with hemophilia A in a US retrospective observational study before and after switching from any standard half-life (SHL) FVIII to EHL rurioctocog alfa pegol (TAK-660, SHP660, BAX 855). This is a modified analysis of Milligan S, et al. RPTH 2019;3(suppl 1):330.
Methods: Real-world data (patient records, prescriptions, and patient-reported bleed logs) were from US specialty pharmacies. Informed consent was obtained for all analyzed patient data. Patients receiving TAK-660 prophylaxis between Nov 2015 and Sep 2018 and ≥90 days’ FVIII before switching to TAK-660 were eligible for inclusion. Main exclusion criteria: previous or current participation in TAK-660 clinical trials, use of on-demand treatment only before switching to TAK-660, or presence of active FVIII inhibitors requiring treatment and/or immune tolerance induction during the study. In contrast to Milligan RPTH 2019, on-label dosing was not required, which resulted in more evaluable patients. Assessments included dosing frequency, factor consumption, and annualized bleeding rate (ABR) before and after switching to TAK-660. Analyses were performed on data from patients with sufficient bleed log information and >90 days’ treatment for both prior FVIII and TAK-660 after switching.
Results: Of 54 patients with available ABR data, 85% (46/54) had received prior SHL FVIII treatment. FVIII consumption decreased in all patients after switching from SHL FVIII to TAK-660, with higher dose per administration but fewer administrations per week (Table 1). Mean ABR decreased by ~50% after switching to TAK-660, with greater decreases in patients with severe disease (Table 2).
Conclusions: Switching from SHL FVIII to TAK-660, given at lower administration frequency and overall weekly consumption, resulted in reduced ABRs in patients with hemophilia A.
| Parameter | Disease severity | Patients, n | Prior SHL FVIII consumption | TAK-660 consumption | Percentage difference: FVIII consumption |
| Mean FVIII dose per administration, IU/kg | Severe | 41 | 38.5 | 50.0 | +29.7 |
| Mild-moderate | 5 | 38.0 | 42.0 | +10.5 | |
| Total | 46 | 38.5 | 49.1 | +27.7 | |
| Mean number of FVIII administrations per week | Severe | 41 | 2.9 | 2.1 | -26.9 |
| Mild-moderate | 5 | 2.7 | 1.8 | -33.3 | |
| Total | 46 | 2.9 | 2.1 | -27.6 | |
| Mean FVIII dose per week, IU/kg | Severe | 41 | 129.0 | 107.0 | -17.1 |
| Mild-moderate | 5 | 93.0 | 72.0 | -22.6 | |
| Total | 46 | 125.0 | 103.0 | -17.5 |
[FVIII consumption before/after switching from SHL FVIII to TAK-660 in patients with available ABR data (values shown are rounded)]
| Disease severity | Patients, n | Prior SHL FVIII mean ABR | TAK-660 mean ABR | Percentage difference: mean ABR |
| Severe | 41 | 3.5 | 1.7 | -52.9 |
| Mild-moderate | 5 | 3.1 | 2.5 | -18.3 |
| Total | 46 | 3.5 | 1.7 | -49.5 |
[Mean ABRs before/after switching from SHL FVIII to TAK-660 in patients with available ABR data (values shown are rounded)]
To cite this abstract in AMA style:
Sun SX, Milligan S. Real-World Outcomes before and after Switching from Standard Half-Life Factor VIII to Rurioctocog Alfa Pegol in the United States [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/real-world-outcomes-before-and-after-switching-from-standard-half-life-factor-viii-to-rurioctocog-alfa-pegol-in-the-united-states/. Accessed November 29, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/real-world-outcomes-before-and-after-switching-from-standard-half-life-factor-viii-to-rurioctocog-alfa-pegol-in-the-united-states/