Factor Product Utilization and Health Outcomes in Patients With Hemophilia

University of British Columbia

Status: Completed

Conditions: Hemophilia B, Congenital; Hemophilia A, Congenital

Study type: Observational

Funder types: Other; Industry

Identifiers

NCT02796222

CAN-FAB-15-10911

Details and patient eligibility

About

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.

Enrollment: 61 patients

Sex: Male

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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)

Trial design

61 participants in 4 patient groups

Hemophilia A patients on rFVIIIFc

Description:

Patients with hemophilia A who switch from on-demand or prophylactic treatment with rFVIII to rFVIIIFc

Hemophilia A patients on rFVIII

Description:

Patients with hemophilia A who remain on on-demand or prophylactic treatment with rFVIII

Hemophilia B patients on rFIXFc

Description:

Patients with hemophilia B who switch from on-demand or prophylactic treatment with rFIX to rFIXFc

Hemophilia A patients on rFIX

Description:

Patients with hemophilia B who remain on on-demand or prophylactic treatment with rFIX