Hemophilia A
Conditions
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.
Interventions
DRUGAvailable Recombinant, and Human Factor VIII products incl. Kogenate FS (Recombinant Factor VIII, BAY14-2222)
Recombinant and Human Factor VIII / Used on demand or prophylaxis of bleeds
Sponsors
Bayer
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
16 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 | Baseline and at 12 months |
| Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT) | Baseline and at 12 months |
| Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (Short Form) (BPI-SF) | Baseline and at 12 months |
| Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF) | Baseline and at 24 months |
| Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective | Baseline and at 12 months |
| Patient Reported Outcome: Change of function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI). | Baseline and at 12 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) | Baseline and at 12 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) | Baseline and at 12 months |
| Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN) | Baseline and at 12 months |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Clinical Outcome: Change of Joint Status | Baseline and every 12 months up to 60 months | Joint Status as defined by: - Hemophilia Joint Health Score/ Pettersson Additive Scale - Range of motion - Imaging (where available) - Target joints and past surgical procedures |
| Clinical Outcome: Baseline disease characteristics | Baseline | Baseline disease characteristics and disease course as defined by: - Date of diagnosis of Hemophilia A - Age at first treatment with factor replacement therapy (child/adult) - Type of factor VIII gene mutation (if available) - Factor VIII level at initial diagnosis (if available) - History of inhibitor and assay used for detection - Date of determination of peak level of inhibitor |
| Clinical Outcome: Change of treatment patterns including Hemophilia treatments via patient chart | Baseline and every 12 months up to 60 months | Treatment patterns as measured by: - Treatment choice and dose (Factor VIII use and type) - Treatment Regimen - Immune tolerance therapy - Products for inhibitors - Blood bank products - Non plasma and topical products |
| Clinical Outcome: Change of Hemophilia A status | Baseline and every 12 months up to 60 months | Hemophilia A status as defined by: - Last measured FVIII level - History of intracranial hemorrhage and age of occurrence - Number of Bleeding episodes/events in the last year - Location of bleed - Confirmed microbleeds on past imaging - PK assessment (if available): Name of FVIII product for most recent PK evaluation - Dose of Factor VIII product administered for most recent PK evaluation - Measured Factor VIII level - Factor VIII half-life and time point of measurement -- Central Venous Access Device (CVAD) or AV (arterial-venous) fistulae |
Countries
Japan, Spain, United Kingdom, United States
Outcome results
None listed