Haemophilia B
Conditions
Keywords
Blood Coagulation Disorder, Hematologic Diseases, Coagulation Protein Disorder, Hemorrhagic Disorder, Genetic Diseases, Inborn
Brief summary
Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic treatment of haemophilia B.
Interventions
DRUGAlprolix
Extended half-life factor IX product
Sponsors
Swedish Orphan Biovitrum
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Have a diagnosis of haemophilia B and been treated previously with factor IX Product * Have started Alprolix treatment prior to enrolment visit, or at enrolment prescribed treatment with Alprolix, regardless of participation in the study * Signed and dated informed consent provided by the patient, or the patient's legally acceptable representative for patients under the legal age, before any study-related activities are undertaken. Assent should be obtained from paediatric patients according to local regulations
Exclusion criteria
* Participation in an investigational medicinal product trial at enrolment visit
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Amount of factor Product used to treat a bleed (on-demand treatment) | 24 months | Assessed by diary |
| Annualised bleeding rate (ABR) (prophylactic treatment) | 24 months | Assessed by diary |
| Annualised injection frequency (prophylactic treatment) | 24 months | Assessed by diary |
| Annualised factor consumption (International Unit [IU]) (prophylactic treatment) | 24 months | Assessed by diary |
| Number of injections to treat a bleed (on-demand treatment) | 24 months | Assessed by diary |
Countries
France
Outcome results
None listed