Moderate Term Musculoskeletal Outcomes With Escalating Dose Prophylaxis: the Canadian Hemophilia Prophylaxis Study Follow-up Study
Trial Snapshot
- Phase
- Phase 4
- Status
- Completed
- Enrollment
- 56
- Locations
- 1
- Primary Endpoint
- Number of Participants Who Developed Target Joint Bleeding
Study Overview
Brief Summary
Primary prophylaxis given less frequently initially, with the infusion frequency increased if needed (Escalating Dose Prophylaxis), is likely to be less expensive and associated with fewer complications than standard prophylaxis while reducing disability to a greater degree than intermittent therapy.
Detailed Description
There are 2 specific study objectives. The first is to estimate the incidence of target joint bleeding in patients with severe hemophilia A treated (for primary prophylaxis) with Escalating Dose Prophylactic factor replacement. The second objective is to obtain accurate estimates of the direct and indirect costs associated with this protocol for use in a cost-effectiveness model (comparing Escalating Dose with standard prophylaxis and with intermittent therapy).
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 12 Months to 30 Months (Child)
- Sex
- Male
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Severe hemophilia A (factor level less than 2%).
- •Age greater than 1 year and less than or equal to 2.5 years.
- •Normal joints using the World Federation of Hemophilia orthopedic scale.
- •Normal radiographs of joints in which bleeding has occurred using the World Federation of Hemophilia radiographic scale.
- •Platelet count of > 150,
- •Informed consent to participate.
Exclusion Criteria
- •Three or more clinically determined bleeds into any single elbow, knee or ankle.
- •Presence or past history of a circulating inhibitor (level ≥ 0.5 Bethesda Units).
- •Family judged to be non-compliant by the local hemophilia clinic director.
- •Competing risk (symptomatic HIV infection, juvenile rheumatoid arthritis, metabolic bone disease, or other diseases known to cause or mimic arthritis.)
Arms & Interventions
Factor VIII
escalating dose Factor VIII
Intervention: Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS) (Biological)
Outcomes
Primary Outcomes
Number of Participants Who Developed Target Joint Bleeding
Time Frame: 6 months
The number of participants who developed target joint bleeding during the study, which was defined as 3 bleeds into any 1 joint within a period of 3 months.
Secondary Outcomes
- Number of Patients Who Developed an Inhibitor to FVIII(6 months)
- Complications Arising From Indwelling Venous Catheter(6 months)
- Annualized Factor Use(12 months)
- Annualized Bleeding Rate(6 months)
- Joint Damage as Determined by the Physiotherapy Score(through study completion, a median of 10 years)
- Physical Disability as Measured by the CHAQ(through study completion, a median of 10 years)
Investigators
Brian Feldman
Division Head, Rheumatology
The Hospital for Sick Children