Hemophilia A
Conditions
Brief summary
This non-interventional study will prospectively collect detailed, high-quality documentation of bleeds, HRQoL, and safety in patients with hemophilia A with or without FVIII inhibitors treated according to local routine clinical practice (receiving FVIII replacement or bypassing agents as either episodic or prophylactic treatment). Actual patients will be enrolled from routine clinical practice in this observational study.
Interventions
DRUGBypassing Agents
Episodic or prophylactic treatment with the use of bypassing agents must be documented for at least the last 6 months prior to the study. During the study, treatment for bleeds will be documented. The choice of coagulation product is at the discretion of the investigator according to local practice standards in this non-interventional study and there are no specific protocol-defined interventions.
DRUGFVIII Replacement
Episodic or prophylactic treatment with the use of FVIII replacement must be documented for at least the last 6 months prior to the study. During the study, treatment for bleeds will be documented. The choice of coagulation product is at the discretion of the investigator according to local practice standards in this non-interventional study and there are no specific protocol-defined interventions.
Sponsors
Hoffmann-La Roche
Study design
Observational model
COHORT
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Cohort A: Patients greater than or equal to (\>/=) 12 years of age at time of informed consent * Cohort A: Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is, \>/= 5 Bethesda units \[BU\]) * Cohort B: Pediatric patients less than (\<) 12 years of age * Cohort B: Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is, \>/=5 BU) * Cohort C: Patients \>/=12 years of age * Cohort C: Diagnosis of congenital hemophilia A and FVIII activity \<1 percent (%) * Cohort C: No prior history of a positive inhibitor against FVIII
Exclusion criteria
* Prior RO5534262 (not applicable if patient agrees that prior RO5534262 will preclude participation in a future investigational RO5534262 study) * Bleeding disorder other than congenital hemophilia A * Ongoing (or planned during the study) immune tolerance induction therapy with FVIII or FVIII prophylaxis if currently/previously exposed to an inhibitor * Previous or concomitant thromboembolic disease * Known human immunodeficiency virus (HIV) infection with cluster of differentiation (CD) 4 count \<200 cells per microliter (cells/mcL)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Bleeds | Approximately 6 months (from Baseline until study completion) |
Secondary
| Measure | Time frame |
|---|---|
| Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Score | Approximately 6 months (every 4 weeks from Baseline until study completion) |
| European Quality of Life-5 Dimensions (EQ-5D-5L) Questionnaire Score Among Adult and Adolescent Patients | Approximately 6 months (every 4 weeks from Baseline until study completion and on days that bleeds are reported) |
| Hemophilia A-Specific Quality of Life (Haem-A-QoL) Questionnaire Score Among Adult Patients | Approximately 6 months (every 4 weeks from Baseline until study completion) |
Countries
Australia, China, Costa Rica, Germany, Italy, Japan, Poland, South Africa, South Korea, Spain, Taiwan, United States
Outcome results
None listed