A Prospective, International, Longitudinal, Observational Disease Registry of Patient-reported Outcomes (PROs) and the Association With Hemophilia A and Its Treatment in Patients With Moderate to Severe Hemophilia A
Overview
- Phase
- Not Applicable
- Intervention
- Available Recombinant, and Human Factor VIII products incl. Kogenate FS (Recombinant Factor VIII, BAY14-2222)
- Conditions
- Hemophilia A
- Sponsor
- Bayer
- Enrollment
- 272
- Primary Endpoint
- Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).
- Status
- Completed
- Last Updated
- 7 years ago
Overview
Brief Summary
The purpose of the study is to improve the understanding of key patient reported outcomes such as quality of life as well as clinical outcomes in hemophilia A, in a global real world setting.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Age 16 or over.
- •Have documentation of physician-confirmed diagnosis of moderate or severe Hemophilia A (severity defined as moderate = FVIII activity 1% to 5% and severe = FVIII activity ≤1%).
- •Signed written informed consent provided by the patient or the patient's parents for patients under the age of 18 (dependent of local regulations).
- •Signed written assent is also required for patients under the age 18 years (dependent on local regulations).
- •Plan to receive at least half of their Hemophilia care at the registry site.
- •Willing and able to enter data as per the data collection schedule.
- •Currently receiving prophylactic or on demand treatment (including within last 6months for on demand).
- •Expected life expectancy of at least 2 years.
Exclusion Criteria
- •Patients with Hemophilia B
- •Patients with von Willebrand disease (vWD)
- •Patients with other rare bleeding disorders
- •Unable to comply with the study protocol
Arms & Interventions
Patients with moderate to severe Hemophilia A / Cohort 1
Patients aged over 16 years, with documented physician-confirmed diagnosis of moderate or severe Hemophilia A (severity defined as moderate = FVIII activity 1% to 5% and severe = FVIII activity ≤1%)
Intervention: Available Recombinant, and Human Factor VIII products incl. Kogenate FS (Recombinant Factor VIII, BAY14-2222)
Outcomes
Primary Outcomes
Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI).
Time Frame: Baseline and at 60 months
Patient Reported Outcome: Change of Quality of Life as measured by EuroQol-5 Dimension questionnaire 5 Level (EQ5D), Short Form Health Survey (SF-12), Hemophilia Quality of Life Measure for Adults (HAEMO-QoL-A)
Time Frame: Baseline and at 60 months
Patient Reported Outcome: Change of treatment patterns as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT) and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
Time Frame: Baseline and at 60 months
Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN)
Time Frame: Baseline and at 60 months
Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT)
Time Frame: Baseline and at 60 months
Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (Short Form) (BPI-SF)
Time Frame: Baseline and at 12 months
Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF)
Time Frame: Baseline and at 60 months
Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective
Time Frame: Baseline and at 60 months
Patient Reported outcome: Change of well-being as measured by Psychological General Well Being Schedule (PGWBI) as well as Smoking and drinking behavior and recreational drug use
Time Frame: Baseline and at 60 months
Secondary Outcomes
- Clinical Outcome: Change of Hemophilia A status(Baseline and every 12 months up to 60 months)
- Clinical Outcome: Change of Joint Status(Baseline and every 12 months up to 60 months)
- Clinical Outcome: Baseline disease characteristics(Baseline)
- Clinical Outcome: Change of treatment patterns including Hemophilia treatments via patient chart(Baseline and every 12 months up to 60 months)