Hemophilia A
Conditions
Brief summary
The purpose of this post-marketing safety study is to evaluate the safety, immunogenicity, and effectiveness of ADVATE in previously untreated patients (PUPs) in China with moderate to severe hemophilia A.
Interventions
Sponsors
Baxalta now part of Shire
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
1. Previously untreated patient (PUP) in China with moderate to severe hemophilia A. 2. Investigator prescribed ADVATE as the predominant therapeutic agent for the management of hemophilia A 3. Informed consent form from patient and/or legal representative will need to be signed per local regulation
Exclusion criteria
1. Presence of an inherited or acquired hemostatic defect other than hemophilia A, and any other clinically significant chronic disease 2. Participants who participated in another investigational FVIII drug study, or had participated in any clinical study involving an investigational FVIII drug during the course of the observation period
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of high titer, low titer and transient Factor VIII (FVIII) inhibitors developed | Throughout the study period of 6 months | — |
| Number and type of other serious and non-serious adverse events (AEs) | Throughout the study period of 6 months | Other AEs refers to AEs other than Factor VIII (FVIII) inhibitors |
Secondary
| Measure | Time frame |
|---|---|
| Annualized bleeding rate (ABR) of participants treated prophylactically and on-demand with ADVATE | Throughout the study period of 6 months |
| Annualized joint bleeding rate (AJBR) of participants treated prophylactically and on-demand with ADVATE | Throughout the study period of 6 months |
| Total number of exposure days (EDs) to ADVATE | Throughout the study period of 6 months |
| Number and type of bleeding episodes treated with ADVATE | Throughout the study period of 6 months |
| Total number of exposure days (EDs) to all Factor VIII (FVIII) products | Throughout the study period of 6 months |
| Total units of all Factor VIII (FVIII) products administered | Throughout the study period of 6 months |
| Total units of ADVATE administered | Throughout the study period of 6 months |
| Number of bleeding episodes treated with 1, 2, 3, ≥ 4 infusions of ADVATE | Throughout the study period of 6 months |
Countries
China
Outcome results
None listed