Clinical trial NCT01085344: Canadian Hemophilia Prophylaxis Study
Official title: Moderate Term Musculoskeletal Outcomes With Escalating Dose Prophylaxis: the Canadian Hemophilia Prophylaxis Study Follow-up Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Severe Hemophilia A
Keywords: hemophilia, prophylaxis, cost effectiveness
Lead sponsor: The Hospital for Sick Children
Sponsor class: OTHER
First posted: 2010-03-11
Enrollment: 56
Sex: MALE
Minimum age (years): 12
Maximum age (years): 30
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).

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.)

Primary outcomes:
measure: Number of Participants Who Developed Target Joint Bleeding
timeFrame: 6 months
Secondary outcomes:
measure: Annualized Bleeding Rate
timeFrame: 6 months
measure: Annualized Factor Use
timeFrame: 12 months
measure: Number of Patients Who Developed an Inhibitor to FVIII
timeFrame: 6 months
measure: Physical Disability as Measured by the CHAQ
timeFrame: through study completion, a median of 10 years
measure: Joint Damage as Determined by the Physiotherapy Score
timeFrame: through study completion, a median of 10 years
measure: Complications Arising From Indwelling Venous Catheter
timeFrame: 6 months
Other outcomes:
Locations:
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
Study officials:
affiliation: The Hospital for Sick Children
name: Brian M Feldman, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Feldman BM, Rivard GE, Babyn P, Wu JKM, Steele M, Poon MC, Card RT, Israels SJ, Laferriere N, Gill K, Chan AK, Carcao M, Klaassen RJ, Cloutier S, Price VE, Dover S, Blanchette VS. Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort. Lancet Haematol. 2018 Jun;5(6):e252-e260. doi: 10.1016/S2352-3026(18)30048-6. Epub 2018 May 3.