Factor Product Utilization and Health Outcomes in Patients With Hemophilia

Completed

• Conditions: Hemophilia B, Congenital; Hemophilia A, Congenital

• Registration Number: NCT02796222
• Lead Sponsor: University of British Columbia

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-04
• Study First Submitted: 2016-05-26
• Study First Submitted QC: 2016-06-06
• Study First Posted: 2016-06-10
• Status Verified: 2021-04
• Primary Completion: 2021-04-26
• Study Completion: 2021-04-26
• Last Update Submitted: 2021-04-26
• Last Update Posted: 2021-04-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 61

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.

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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)

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Change in the Work Productivity and Impairment Questionnaire (WPAI+CIQ: HS) score	From baseline to 3 months, 12 months and 24 months
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 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 "Physical Functioning" subscale of SF-36.	From baseline to 3 months, 12 months and 24 months
Product used for treatment of breakthrough bleeding and surgical procedures	Baseline through study completion, an average of 2 years	Choice among list of products
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 treatment satisfaction "Treatment" domain of Haem-A-QoL	From baseline to 3 months, 12 months and 24 months
Annualized bleeding rate	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)
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.
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
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)
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.
Total annualized number of factor infusions	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)

Trial Locations

Locations (1)

BC Hemophilia Adult Program; 🇨🇦 Vancouver, British Columbia, Canada