58908
document
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 ) . 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 .
58909
Intervention_Pharmacological
routine prophylaxis
58910
Intervention_Pharmacological
on-demand treatment with sucrose-formulated recombinant factor VIII
58911
Intervention_Pharmacological
with sucrose-formulated recombinant FVIII
58912
Outcome_Physical
number of total bleeding episodes
58913
Outcome_Physical
total bleeding episodes and total bleeding
58914
Participant_Age
adults
58915
Participant_Condition
severe hemophilia A ( SPINART )
58916
Participant_Age
older patients
58917
Participant_Condition
hemophilia
58918
Participant_Condition
trial
58919
Participant_Sex
Males
58920
Participant_Age
aged 12-50 years
58921
Participant_Condition
severe hemophilia A , ≥ 150 days of exposure to FVIII , no FVIII inhibitors , no prophylaxis for > 12 consecutive months in the past 5 years
58922
Participant_Condition
6-24 bleeding episodes in the preceding 6 months
58923
Participant_Sample-size
Eighty-four patients
58924
Participant_Sample-size
n = 42 per group
58925
Participant_Age
mean age , 30.6 years
58926
Participant_Condition
median treatment duration , 1.7 years