1.
Spinart trial 3-year results with Bayer’s sucrose formulated recombinant factor VIII: relationship between bleeding frequency and joint health in adults with severe hemophilia A using prophylaxis
Reding MT, Kempton CL, Funk S, Engelen S, Walker D, Hong W
Journal of Thrombosis and Haemostasis. 2015;13((Suppl. 2)):132.. Abstract No. OR101.
3.
Bleeding phenotype with various bay 94-9027 dosing regimens: subanalyses from the Protect VIII study
Boggio LN, Hong W, Wang M, Eyster ME, Michaels LA
Blood. 2014;124((21)): Abstract No. 1526
4.
Effect on joint health of routine prophylaxis with Bayer’s sucrose-formulated recombinant factor VIII (rFVIII-FS) in adolescents and adults previously treated on demand: MRI analyses from the 3-Year Spinart study
Lundin B, Hong W, Raunig D, Engelen S, Peterfy C, Werk, Manco-Johnson MJ
Blood. 2014;124((21)): Abstract No. 2854
5.
Randomized, controlled, parallel-group trial of routine prophylaxis vs. on-demand treatment with sucrose-formulated recombinant factor VIII in adults with severe hemophilia A (SPINART)
Manco-Johnson MJ, Kempton CL, Reding MT, Lissitchkov T, Goranov S, Gercheva L, Rusen L, Ghinea M, Uscatescu V, Rescia V, et al
Journal of Thrombosis & Haemostasis. 2013;11((6):):1119-27.
Abstract
BACKGROUND The benefits of routine prophylaxis vs. on-demand treatment with factor VIII products have not been evaluated in controlled clinical trials in older patients with hemophilia A. OBJECTIVES To report results from a preplanned analysis of data from the first year of the 3-year SPINART study, which compares routine prophylaxis with on-demand treatment with sucrose-formulated recombinant FVIII (rFVIII-FS). PATIENTS/METHODS SPINART is an open-label, randomized, controlled, parallel-group, multinational trial. Males aged 12-50 years with severe hemophilia A, > 150 days of exposure to FVIII, no FVIII inhibitors, no prophylaxis for > 12 consecutive months in the past 5 years and 6-24 bleeding episodes in the preceding 6 months were randomized 1 : 1 to rFVIII-FS prophylaxis (25 IU kg(-1) , three times weekly) or on-demand treatment. The primary efficacy endpoint, number of total bleeding episodes in the intent-to-treat population, was analyzed after the last patient had completed 1 year of follow-up. A negative binomial model was used for the primary endpoint analysis; analysis of variance was used for confirmatory analysis of annualized bleeding rates. RESULTS Eighty-four patients were enrolled and analyzed (n = 42 per group; mean age, 30.6 years; median treatment duration, 1.7 years). The median number of total bleeding episodes and total bleeding episodes per year were significantly lower with prophylaxis than with on-demand treatment (total, 0 vs. 54.5; total per year, 0 vs. 27.9; both P < 0.0001). No treatment-related adverse events occurred, and no patients developed FVIII inhibitors. CONCLUSIONS Routine prophylaxis with rFVIII-FS leads to a significant reduction in bleeding as compared with on-demand treatment. Adverse events were consistent with the established rFVIII-FS safety profile. 2013 International Society on Thrombosis and Haemostasis.