Hemophilia A, Congenital, Hemophilia B, Congenital
Conditions
Keywords
Hemophilia, Recombinant factor VIII, Recombinant factor VIII Fc, Recombinant factor IX, Patient-reported outcomes, Recombinant factor IX Fc
Brief summary
Recombinant factor VIII Fc (rFVIIIFc) and recombinant factor IX Fc (rFIXFc) are extended half-life coagulation factors approved by Health Canada in 2014 for the treatment of severe hemophilia A and B, respectively. The objectives of this observational study is to describe the change in annual factor consumption, clinical and patient-reported outcomes for patients who switch from recombinant factor VIII (rFVIII) and recombinant factor IX (rFIX) to rFVIIIFc/ rFIXFc in Canada, and to explore clinicians' and patients' reasons for switching or not switching.
Interventions
None listed
Sponsors
Biogen
University of British Columbia
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Males ≥12 years of age with severe and moderate congenital hemophilia A or B (baseline factor activity\<5%) 2. Ability to understand the purpose and risks of the study and provide signed and dated informed consent or assent and authorization to use protected health information (PHI) in accordance with national and local privacy regulations.
Exclusion criteria
1. Unable or unwilling to provide informed consent 2. Patients with an existing bleeding disorder other than hemophilia A or B 3. History of hypersensitivity or severe allergic reactions to factor products 4. Patients currently participating in a phase 1-3 study with another factor replacement product 5. Unable to adhere to the study requirements based on the judgment of the Prescribing Physician (e.g. unable to enter accurate and timely infusion and bleeding records)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in the total annualized factor consumption (in units/kilogram/year) | From baseline to 24-month period on rFVIIIFc or rFIXFc |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in health-related quality of life (HRQoL) SF-36 | From baseline to 3 months, 12 months and 24 months | HRQoL will be measured using Short Form 36 (SF-36) in all patients |
| Change in health-related quality of life (HRQoL) Haem-A-Qol | From baseline to 3 months, 12 months and 24 months | Haem-A-QoL in patients over age 18 years |
| Change in health-related quality of life (HRQoL) CHO-KLAT | From baseline to 3 months, 12 months and 24 months | The Canadian Hemophilia Outcomes- Kids Life Assessment Tool (CHO-KLAT) in patients between ages 13-18 years |
| Change in the Work Productivity and Impairment Questionnaire (WPAI+CIQ: HS) score | From baseline to 3 months, 12 months and 24 months | — |
| Change in chronic pain Numeric Rating Scale (0-10) | From baseline to 3 months, 12 months and 24 months | — |
| Change in chronic pain Bodily Pain subscale of SF-36 | From baseline to 3 months, 12 months and 24 months | — |
| Change in physical activity (IPAQ) | From baseline to 3 months, 12 months and 24 months | Physical activity will be measured using the International Physical Activity Questionnaire (IPAQ) |
| Change in physical activity Physical Functioning subscale of SF-36. | From baseline to 3 months, 12 months and 24 months | — |
| Change in treatment satisfaction Treatment domain of Haem-A-QoL | From baseline to 3 months, 12 months and 24 months | — |
| Change in treatment satisfaction abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) questionnaire. | From baseline to 3 months, 12 months and 24 months | Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) questionnaire. |
| Serious adverse events | From baseline to 24-month period after product switch (or 24-month period on study for non-switchers) | — |
| Change in mood partner/caregiver subjective assessment numeric rating scale (0-10) | From baseline to 3 months only | Partner or caregiver's subjective assessment of subject's mood from baseline to 3 months. |
| Clinicians' and patients' reason for switching to rFVIIIFc | Baseline through study completion, an average of 2 years | Choice among list of common reasons for changing product |
| Clinicians' and patients' reason for switching to rFIXFc | Baseline through study completion, an average of 2 years | Choice among list of common reasons for changing product |
| Product used for treatment of breakthrough bleeding and surgical procedures | Baseline through study completion, an average of 2 years | Choice among list of products |
| Total annualized number of factor infusions | From baseline to 24-month period after product switch (or 24-month period on study for non-switchers) | — |
| Annualized bleeding rate | From baseline to 24-month period after product switch (or 24-month period on study for non-switchers) | — |
| Ratio of annual factor consumption-to-annual factor prescription | From baseline to 24-month period after product switch (or 24-month period on study for non-switchers) | — |
| Number of infusions required to treat a breakthrough bleed | From baseline to 24-month period after product switch (or 24-month period on study for non-switchers) | — |
| Incremental factor utilization per joint bleed avoided | From baseline to 24-month period after product switch (or 24-month period on study for non-switchers) | Difference in annualized factor utilization between Fc and regular non-Fc prophylaxis, divided by the difference in annualized joint bleeding rate between the two groups. |
| Adverse events leading to permanent discontinuation of rFVIIIFc or rFIXFc | From baseline to 24-month period after product switch (or 24-month period on study for non-switchers) | — |
| Change in mood Mental Health subscale of SF-36 | From baseline to 3 months, 12 months and 24 months | partner or caregiver's subjective assessment of subject's mood from baseline to 3 months. |
Countries
Canada
Outcome results
None listed