Comparison of the effect of three times a week prophylaxis on all bleeds with on-demand treatment for children with severe Hemophilia A.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.
Unnamed facility
Guangzhou, Guangdong, China
Unnamed facility
Wuhan, Hubei, China
Unnamed facility
Beijing, China
Unnamed facility
Beijing, China
Unnamed facility
Tianjin, China
Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period
Annualized bleedings period 1 minus period 2 ITT analysis set.
Time frame: Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)
Difference of Annualized Number of Joint Bleeds Between On-demand and Prophylaxis Period
Annualized joint bleedings period 1 minus period 2 ITT analysis set.
Time frame: Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)
Difference of Intra-individual Change of Joint Function During Each Period Assessed by the Hemophilia Joint Health Score Between On-demand and Prophylaxis Period
Hemophilia Joint Health Score(HJHS) ranges from 0 to 124. Higher values in the HJHS represent worse situation for the subject. 2-sided Hodges Lehmann estimates for median 95% CI HJHS values difference of changes ITT analysis set.
Time frame: From baseline to Week 12 (on-demand treatment) and Week 24 (prophylactic treatment)
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