A Prospective Study to Collect High-Quality Documentation of Bleeds, Health-Related Quality of Life (HRQoL), and Safety Outcomes in Patients With Hemophilia A Treated With Standard-of-Care Treatment

Hoffmann-La Roche221 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

221

Conditions

Hemophilia A

Interventions

Bypassing AgentsDRUG

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.

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.

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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)

Locations (35)

Santa Monica Oncology Center

Santa Monica, California, United States

University of Colorado Denver, Children's Hospital

Aurora, Colorado, United States

Georgetown Uni Medical Center; Lombardi Cancer Center

Washington D.C., District of Columbia, United States

Winship Cancer Institute

Atlanta, Georgia, United States

Tulane Uni Health Sciences Center

New Orleans, Louisiana, United States

Outcomes

Primary Outcomes

Number of Bleeds

Time frame: Approximately 6 months (from Baseline until study completion)

Secondary Outcomes

European Quality of Life-5 Dimensions (EQ-5D-5L) Questionnaire Score Among Adult and Adolescent Patients

Time frame: 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

Time frame: Approximately 6 months (every 4 weeks from Baseline until study completion)

Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Score

Time frame: Approximately 6 months (every 4 weeks from Baseline until study completion)