This NIS aims to assess the patient-reported outcomes (PROs) in enrolled subjects
The design of the study is observational: both prospective and retrospective, national, multicenter, non- interventional. During this observational study, subjects diagnosed with severe or moderately severe (with severe phenotype) hemophilia A receiving routine prophylaxis treatment with ReFacto AF as per local standard clinical care, and in line with local labeling, are to be observed for 12 months. The primary objective is to assess the health-related quality of life in subjects treated with ReFacto AF routine prophylaxis at baseline and after 6 and 12 months. The secondary objectives are: * To assess the subjective physical functioning of subjects treated with ReFacto AF routine prophylaxis at baseline and after 6 and 12 months; * To assess the treatment satisfaction of caregivers of children and subjects above 18 years treated with ReFacto AF routine prophylaxis at baseline and after 6 and 12 months; * To determine in routine clinical practice the mean annualized bleeding rate (ABR) in subjects treated with ReFacto AF routine prophylaxis; * To describe the status of joint health measured by HJHS before and after starting routine prophylaxis for subjects above 18 years.
Study Type
OBSERVATIONAL
Observational - Non-Interventional Study
The health-related quality of life in subjects treated with ReFacto AF routine prophylaxis
HRQoL will be assessed by applying the self-reported hemophilia-specific quality of life Haemo-QoL questionnaire. The endpoint will include the domains and total scores of the questionnaire. The results of this study will be presented using descriptive statistics and there will be no hypothesis testing. The domain scores and total scores will be summarized by: N, mean (sd), median, min-max and the 95% confidence interval at baseline, end of study, and the change from baseline.
Time frame: Baseline and after 6 and 12 months.
The health-related quality of life
HRQoL will be be evaluated also by recording the number of days of absence from kindergarten, school or high-school. There are no a priori hypotheses specified and results will be presented as descriptive statistics.
Time frame: Baseline and after 6 and 12 months.
The subjective physical functioning of subjects treated with ReFacto AF routine prophylaxis
Subjects will be asked to report their subjective physical functioning using the HEP-Test-Q. The endpoint will include the domains and total scores of the questionnaire. The results of this study will be presented using descriptive statistics and there will be no hypothesis testing. The domain scores and total scores will be summarized by: N, mean (sd), median, min-max and the 95% confidence interval at baseline, end of study, and the change from baseline.
Time frame: Baseline and after 6 and 12 months;
The treatment satisfaction of caregivers of children and subjects above 18 years treated with ReFacto AF routine prophylaxis
Caregivers will evaluate their satisfaction with the treatment of their child and the above 18 years subjects will evaluate their own satisfaction with the treatment using the Hemo-Satp questionnaire. The endpoint will include the domains and total scores of the questionnaire. The results of this study will be presented using descriptive statistics and there will be no hypothesis testing. The domain scores and total scores will be summarized by: N, mean (sd), median, min-max and the 95% confidence interval at baseline, end of study, and the change from baseline.
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Time frame: Baseline and after 6 and 12 months;
The mean annualized bleeding rate (ABR) in subjects treated with ReFacto AF routine prophylaxis;
ABR will be calculated at the end of the study based on the number of bleeds recorded during the 12-months observation period. The ABR will be presented as N, mean (sd), median, min-max and the 95% confidence interval.
Time frame: After 12 months
Joint health for subjects above 18 years
Status of joint health will be described for subjects above 18 years using the HJHS (Hemophilia Joint Health Score) before and after starting routine prophylaxis. HJHS for one particular joint (elbow, knee or ankle) represents the sum between the joint totals and the global gait score.The minimum score is 0 and the maximum is 24. The joint totals is the sum between the scores for swelling, duration of swelling, muscle atrophy, crepitus on motion, flexion loss, extension loss, joint pain and strength; the maximum score is 20. The global gait score evaluates walking, stairs, running and hopping in one leg; the maximum score is 4.
Time frame: After 12 months