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.
Study Type
OBSERVATIONAL
Enrollment
61
BC Hemophilia Adult Program
Vancouver, British Columbia, Canada
Change in the total annualized factor consumption (in units/kilogram/year)
Time frame: From baseline to 24-month period on rFVIIIFc or rFIXFc
Change in health-related quality of life (HRQoL) SF-36
HRQoL will be measured using Short Form 36 (SF-36) in all patients
Time frame: From baseline to 3 months, 12 months and 24 months
Change in health-related quality of life (HRQoL) Haem-A-Qol
Haem-A-QoL in patients over age 18 years
Time frame: From baseline to 3 months, 12 months and 24 months
Change in health-related quality of life (HRQoL) CHO-KLAT
The Canadian Hemophilia Outcomes- Kids Life Assessment Tool (CHO-KLAT) in patients between ages 13-18 years
Time frame: From baseline to 3 months, 12 months and 24 months
Change in the Work Productivity and Impairment Questionnaire (WPAI+CIQ: HS) score
Time frame: From baseline to 3 months, 12 months and 24 months
Change in chronic pain Numeric Rating Scale (0-10)
Time frame: From baseline to 3 months, 12 months and 24 months
Change in chronic pain "Bodily Pain" subscale of SF-36
Time frame: From baseline to 3 months, 12 months and 24 months
Change in physical activity (IPAQ)
Physical activity will be measured using the International Physical Activity Questionnaire (IPAQ)
Time frame: From baseline to 3 months, 12 months and 24 months
Change in physical activity "Physical Functioning" subscale of SF-36.
Time frame: From baseline to 3 months, 12 months and 24 months
Change in treatment satisfaction "Treatment" domain of Haem-A-QoL
Time frame: 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.
Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) questionnaire.
Time frame: From baseline to 3 months, 12 months and 24 months
Change in mood "Mental Health" subscale of SF-36
partner or caregiver's subjective assessment of subject's mood from baseline to 3 months.
Time frame: From baseline to 3 months, 12 months and 24 months
Change in mood partner/caregiver subjective assessment numeric rating scale (0-10)
Partner or caregiver's subjective assessment of subject's mood from baseline to 3 months.
Time frame: From baseline to 3 months only
Clinicians' and patients' reason for switching to rFVIIIFc
Choice among list of common reasons for changing product
Time frame: Baseline through study completion, an average of 2 years
Clinicians' and patients' reason for switching to rFIXFc
Choice among list of common reasons for changing product
Time frame: Baseline through study completion, an average of 2 years
Product used for treatment of breakthrough bleeding and surgical procedures
Choice among list of products
Time frame: Baseline through study completion, an average of 2 years
Total annualized number of factor infusions
Time frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Annualized bleeding rate
Time frame: 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
Time frame: 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
Time frame: 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
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.
Time frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Adverse events leading to permanent discontinuation of rFVIIIFc or rFIXFc
Time frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
Serious adverse events
Time frame: From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)
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