Hemophilia A
Conditions
Brief summary
The project is a controlled observational, multicenter, prospective data collection on secondary prophylaxis with Kogenate Bayer in adolescents and adults with severe haemophilia A (FVIII \< 1%). The observational period will cover at least 5 years per patient. The long-term secondary prophylaxis group will be compared versus on-demand treatment group by the assessment of orthopedic status progression and pharmacoeconomics evaluation.
Interventions
Long-term secondary prophylaxis with product administered 20-30 UI /kg three times weekly
Sponsors
Bayer
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to 55 Years
Healthy volunteers
No
Inclusion criteria
* age ≥ 12 years and ≤ 55 years * severe haemophilia A (FVIII \< 1%) * absence of inhibitors (Bethesda titre \< 0.6 BU/ml) * Previous Treated Patients (prior exposure days \> 200) * Kogenate Bayer administered 20-30 IU/kg -3 times a week, for the prophylaxis group * ≥ 6 joint bleeds requiring treatment with FVIII concentrates in the previous 6 months before enrollment, for on-demand group * written informed consent
Exclusion criteria
* concomitant severe and chronic diseases or congenital skeletal malformation * unreliability of patient or likelihood of follow-up failure * presence of inhibitors or history of inhibitors (in the previous 2 years) * currently on immune tolerance treatment * hepatic cirrhosis or liver disease in rapid progression * AIDS * platelet count \< 75,000/mm3 * presence of conditions that influence negatively patient´s compliance * participation in another study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Evaluate the efficacy, safety and pharmacoeconomic of long-term secondary prophylaxis with Kogenate Bayer in comparison to on-demand treatment with Kogenate Bayer in terms of joint bleeding episodes per year | Every 6 months |
Secondary
| Measure | Time frame |
|---|---|
| Efficacy, safety, pharmacoeconomic impact of secondary prophylaxis versus on demand group in terms of overall bleeding episodes | Every 6 months |
| Muscolo skeletal evaluation by Orthopedic Joint Score | Every 12 months |
| Radiological Evaluation by Pettersson Score | Baseline and after 3-5 years |
| Health related quality of life | Every 12 months |
| cost-effectiveness and utility, patient compliance, adverse events | Every 6 months |
Countries
Italy
Outcome results
None listed