PB0502 - Clinical outcomes in patients with hemophilia A receiving octocog alfa or rurioctocog alfa pegol versus other FVIII replacement therapies: Insights from the AHEAD, CHESS II, and CHESS PAEDs studies

Background: The international Antihemophilic factor Hemophilia A outcome Database (AHEAD) study, the Cost of Haemophilia in Europe: A Socioeconomic Survey (CHESS) II study and the CHESS in a Paediatric Population (CHESS PAEDs) study provide real-world insights into the treatment and outcomes of patients with hemophilia A (HA).

Aims: To evaluate differences in clinical outcomes (annualized bleeding rate [ABR] and target joints [TJs]) in patients with HA receiving octocog alfa or rurioctocog alfa pegol compared with other factor VIII (FVIII) replacement therapies.

Methods: Data for adult and pediatric male patients with moderate (FVIII 1–≤5%) or severe (FVIII < 1%) HA without FVIII inhibitors from the AHEAD, CHESS II, or CHESS PAEDs studies were utilized. The average treatment effect (ATE) was estimated for ABR and number of TJs between a treatment cohort (patients receiving octocog alfa/rurioctocog alfa pegol) and a control cohort (patients receiving any other FVIII concentrate). One-to-one propensity score (PS) matching models were specified for each outcome of interest to match observations across cohorts on variables selected according to correlation with the outcome (Table 1). ABR and TJ outcome differences were evaluated descriptively via ATE between PS matched cohorts; statistical significance was assessed via t-test.

Results: The analysis included 1527 patients with 909 and 618 in the treatment and control cohorts, respectively (Table 1). For ABR, no statistically significant difference in ATE was observed between cohorts in the total or prophylaxis subgroup (Table 2). ATE showed a statistically significant (p < 0.01) difference of approximately -0.2 and -0.3 TJs for the treatment cohort compared with control in both the total and prophylaxis subgroup, respectively (Table 2).

Conclusion(s): These findings suggest therapeutic equivalence between octocog alfa/rurioctocog alfa pegol and other FVIII replacement therapies in terms of ABR, with more favorable TJ outcomes in patients with HA receiving octocog alfa/rurioctocog alfa pegol.