Abstract Number: PB0919
Meeting: ISTH 2020 Congress
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|
|Mean number of FVIII administrations per week||Severe||41||2.9||2.1||-26.9|
|Mean FVIII dose per week, IU/kg||Severe||41||129.0||107.0||-17.1|
[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|
[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 September 19, 2020.
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