Canadian Hemophilia Prophylaxis Study

The Hospital for Sick Children56 enrolled

Overview

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.

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Severe Hemophilia A

Interventions

Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)BIOLOGICAL

escalating dose prophylaxis

Recombinant Factor VIII (Advate/Helixate FS/KoegenateFS)BIOLOGICAL

escalating dose

Eligibility

Sex: MALEMin age: 12 MonthsMax age: 30 Months

Medical Language ↔ Plain English

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.)

Locations (1)

The Hospital for Sick Children

Toronto, Ontario, Canada

Outcomes

Primary Outcomes

Number of Participants Who Developed Target Joint Bleeding

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.

Time frame: 6 months

Secondary Outcomes

Annualized Bleeding Rate

Number of index hemarthorses (bleeds into ankles, elbows or knees) per patient per year

Time frame: 6 months

Annualized Factor Use

annual factor usage per subject

Time frame: 12 months

Number of Patients Who Developed an Inhibitor to FVIII

The number of patients who developed an inhibitor for FVIII, defined as \>= 0.5 Bethesda Units

Time frame: 6 months

Physical Disability as Measured by the CHAQ

complete the Child Health Assessment Questionnaire (CHAQ) at each 6 month visit. The CHAQ is a validated tool to measure a disability index, with a possible score range of 0-3, where 0 represents no disability and 3 represents maximal disability. The CHAQ is known to have a strong ceiling effect. The CHAQ was collected at each study visit (i.e. every 6 months for the duration each patient was on study). The reported score represents the median end of study score.

Time frame: through study completion, a median of 10 years

Joint Damage as Determined by the Physiotherapy Score

Complete the modified Colarado Physiotherapy Assessment every 6 months at each visit with a score range 0-30 for ankles and knees and 0-26 for elbows), measured at all study visits, which we modified by not assessing crepitus or the ankle joint circumference measurement. For each scale, 0 represents no joint damage, with 26/30 representing maximum possible joint damage. The reported score represents the median end of study score

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.