Factor Product Utilization and Health Outcomes in Patients With Hemophilia

Sponsor

University of British Columbia (Other)

Overall Status

Completed

CT.gov ID

NCT02796222

Collaborator

Biogen (Industry)

Enrollment

Location

60.8

Actual Duration (Months)

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Hemophilia A, Congenital Hemophilia B, Congenital

Study Design

Study Type:

Observational

Actual Enrollment :

61 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Factor Product Utilization and Health Outcomes in Patients With Hemophilia A and B in Canada: An Observational Study of Real-world Outcomes

Actual Study Start Date :

Apr 1, 2016

Actual Primary Completion Date :

Apr 26, 2021

Actual Study Completion Date :

Apr 26, 2021

Arms and Interventions

Arm	Intervention/Treatment
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

Outcome Measures

Primary Outcome Measures

Change in the total annualized factor consumption (in units/kilogram/year) [From baseline to 24-month period on rFVIIIFc or rFIXFc]

Secondary Outcome Measures

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.
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.
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)]
Serious adverse events [From baseline to 24-month period after product switch (or 24-month period on study for non-switchers)]

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years and Older

Sexes Eligible for Study:

Male

Accepts Healthy Volunteers:

Inclusion Criteria:

Males ≥12 years of age with severe and moderate congenital hemophilia A or B (baseline factor activity<5%)
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:

Unable or unwilling to provide informed consent
Patients with an existing bleeding disorder other than hemophilia A or B
History of hypersensitivity or severe allergic reactions to factor products
Patients currently participating in a phase 1-3 study with another factor replacement product
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)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	BC Hemophilia Adult Program	Vancouver	British Columbia	Canada	V6T 2G2

Sponsors and Collaborators

University of British Columbia
Biogen

Investigators

Principal Investigator: Shannon Jackson, MD, Division of Hematology, Department of Medicine, University of British Columbia
Study Chair: 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