Patient characteristics that influence efficacy of prophylaxis with rFVIII-FS three times per week: A subgroup analysis of the LIPLONG study

Prospective data on the efficacy of secondary prophylaxis in adults with haemophilia A are limited. To analyse bleeding outcomes in the sucrose-formulated recombinant factor VIII [rFVIII-FS (control)] arm of the LIPLONG study, a randomized, double-blind, 52-week trial was conducted in patients with severe haemophilia A receiving prophylaxis with the investigational product BAY 79-4980 or rFVIII-FS. The per-protocol population of previously treated patients with severe haemophilia A without a history of inhibitors (n = 68 males; mean age, 34.4 years) received 25 IU kg^-1 rFVIII-FS three times per week for a median of 50.7 weeks. Annualized bleeding rates were assessed and analysed according to predefined target joint status at study start, prestudy treatment type (prophylaxis vs. on demand), age (<30 or ≥30 years), geographical region, bleeding frequency during the previous 6 months and physical activity status during the study using the Student t-test. The annualized median (range) number of bleeds was 2.2 (0.0-23) bleeds per year. The median (range) number of bleeds per year was significantly lower in patient subgroups without vs. with target joints [0.5 (0.0-17.1) vs. 4.2 (0.0-22.8); P = 0.02] and in those with ≤9 vs. >9 bleeds during the previous 6 months [1.1 (0.0-19.2) vs. 5.3 (0.0-22.8); P = 0.01]. Following randomization to prophylaxis with rFVIII-FS, bleeding frequency was effectively reduced. Absence of target joints and prestudy bleeding frequency were predictors of a low bleeding frequency during prophylaxis treatment.