Phase 4
Completed N=56
Canadian Hemophilia Prophylaxis Study
Source: ClinicalTrials.gov NCT01085344 ↗Enrolled (actual)
56
Serious AEs
0.0%
Results posted
Dec 2019
Primary outcomePrimary: Number of Participants Who Developed Target Joint Bleeding — 17 Participants
◆ Published Evidence
Established
38citations · ~5 / year
Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort.
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.
Linked Publications
-
Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Who Developed Target Joint Bleeding |
17 | — |
| SECONDARY Annualized Bleeding Rate |
0.95 | — |
| SECONDARY Annualized Factor Use |
3600 | — |
| SECONDARY Number of Patients Who Developed an Inhibitor to FVIII |
5 | — |
| SECONDARY Physical Disability as Measured by the CHAQ |
— | — |
| SECONDARY Joint Damage as Determined by the Physiotherapy Score |
1; 1; 0; 0; 0; 0 | — |
| SECONDARY Complications Arising From Indwelling Venous Catheter |
— | — |
Eligibility Criteria
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 > 150,000.
- 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.)
Data sourced from ClinicalTrials.gov (NCT01085344) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.