OBJECTIVES: This study was an indirect comparison of BAY 94-9027 vs. efmoroctocog alfa, BAX 855, and rAHF-PFM for prophylactic treatment of severe hemophilia A.

METHODS: Comparisons were conducted using patient-level data from PROTECT-VIII trial for BAY 94-9027 and published data from one efmoroctocog alfa trial, one BAX 855 trial, and two rAHF-PFM trials. There were no control arms in these trials, so MAIC was used for confounding adjustment. Comparisons were conducted separately for the two rAHF-PFM trials because they calculated annualized bleed rate (ABR) differently. Patients from PROTECT-VIII were weighted to match baseline characteristics in each comparator trial, including age, race, weight (except rAHF-PFM), ≥1 target joint (except rAHF-PFM trial 1), prior treatment (except rAHF-PFM trial 2), region (efmoroctocog alfa only), and bleeding events in the prior year (efmoroctocog alfa only). ABR, percentage of patients with no bleeds, and weekly consumption were compared.

RESULTS: After matching, BAY 94-9027 had a similar ABR as the comparators: 3.77 vs. 3.90 (efmoroctocog alfa); 3.95 vs. 3.70 (BAX 855); 4.28 vs. 6.30 (rAHF-PFM trial 1); 1.87 vs. 1.80 (rAHF-PFM trial 2 transformed ABR); all insignificant. BAY 94-9027 had a similar percentage of patients with no bleeds as the comparators: 34.1% vs. 40.7% (efmoroctocog alfa); 38.9% vs. 39.6% (BAX 855); 41.5% vs. 29.9% and 40.0% vs. 33.3% (rAHF-PFM); all insignificant. The mean weekly consumption for BAY 94-9027 was significantly lower than efmoroctocog alfa (66.5 vs. 82.2 IU/kg, p<0.001). The median weekly consumption for BAY 94-9027 was lower than BAX 855 and rAHF-PFM: 64.3 vs. 87.4 (BAX 855); 64.0 vs. 107.5 and 63.6 vs. 109.9 (rAHF-PFM); p-values incalculable due to unavailability of data.

CONCLUSIONS: After adjusting for heterogeneity in patient characteristics between the trials, prophylaxis with BAY 94-9027 demonstrated a lower weekly consumption to achieve efficacy comparable to efmoroctocog alfa, BAX 855, and rAHF-PFM.