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| Name | Class |
|---|---|
| Biogen | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Hemophilia A patients on rFVIIIFc | Patients with hemophilia A who switch from on-demand or prophylactic treatment with rFVIII to rFVIIIFc | ||
| Hemophilia A patients on rFVIII | Patients with hemophilia A who remain on on-demand or prophylactic treatment with rFVIII | ||
| Hemophilia B patients on rFIXFc | Patients with hemophilia B who switch from on-demand or prophylactic treatment with rFIX to rFIXFc | ||
| Hemophilia A patients on rFIX | Patients with hemophilia B who remain on on-demand or prophylactic treatment with rFIX |
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| Measure | Description | Time Frame |
|---|---|---|
| Change in the total annualized factor consumption (in units/kilogram/year) | From baseline to 24-month period on rFVIIIFc or rFIXFc |
| Measure | Description | Time Frame |
|---|---|---|
| Change in health-related quality of life (HRQoL) SF-36 | HRQoL will be measured using Short Form 36 (SF-36) in all patients | From baseline to 3 months, 12 months and 24 months |
| Change in health-related quality of life (HRQoL) Haem-A-Qol |
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Inclusion Criteria:
Exclusion Criteria:
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This study will be conducted in male patients ≥12 years of age with severe and moderate hemophilia A or hemophilia B (baseline factor level <5%) who are able to sign the informed consent or assent.
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| Name | Affiliation | Role |
|---|---|---|
| Shannon Jackson, MD | Division of Hematology, Department of Medicine, University of British Columbia | Principal Investigator |
| Robert Klaassen, MD | Division of Hematology/Oncology, Department of Pediatrics, University of Ottawa | Study Chair |
| Man-Chiu Poon, MD | Division of Hematology, Department of Medicine, University of Calgary | Study Chair |
| Sue Robinson, MD | Division of Hematology, Department of Medicine, Dalhousie University | Study Chair |
| John Wu, MD | BC Children's hospital, Division of Hematology, Department of Medicine, University of British Columbia | Study Chair |
| Alfonso Iorio, MD | Hemophilia Program, Hamilton Health Services Program, McMaster University | Study Chair |
| Michelle Sholzberg, MD | Hemophilia Program, St. Michael's Hospital, University of Toronto | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| BC Hemophilia Adult Program | Vancouver | British Columbia | V6T 2G2 | Canada |
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| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| D002836 | Hemophilia B |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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Haem-A-QoL in patients over age 18 years
| 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 | From baseline to 3 months, 12 months and 24 months |
| 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) | Physical activity will be measured using the International Physical Activity Questionnaire (IPAQ) | From baseline to 3 months, 12 months and 24 months |
| 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. | Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) questionnaire. | 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. | 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. | From baseline to 3 months only |
| Clinicians' and patients' reason for switching to rFVIIIFc | Choice among list of common reasons for changing product | 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 | Baseline through study completion, an average of 2 years |
| Product used for treatment of breakthrough bleeding and surgical procedures | Choice among list of products | Baseline through study completion, an average of 2 years |
| 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 | 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. | 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 | From baseline to 24-month period after product switch (or 24-month period on study for non-switchers) |
| Serious adverse events | From baseline to 24-month period after product switch (or 24-month period on study for non-switchers) |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |