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| ID | Type | Description | Link |
|---|---|---|---|
| KG1301 | Other Identifier | Company internal |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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%) |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Available Recombinant, and Human Factor VIII products incl. Kogenate FS (Recombinant Factor VIII, BAY14-2222) | Drug | Recombinant and Human Factor VIII / Used on demand or prophylaxis of bleeds |
| Measure | Description | Time Frame |
|---|---|---|
| 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 function as measured by Hemophilia Activities List (HAL), Daily Activities including Work Productivity and Activity Impairment Scale (WPAI). | Baseline and at 24 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 36 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 48 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 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) | 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) | Resources: Hospital and Healthcare professional visits |
| Measure | Description | Time Frame |
|---|---|---|
| Clinical Outcome: Change of Hemophilia A status | 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 |
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Inclusion Criteria:
Exclusion Criteria:
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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%)
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| Name | Affiliation | Role |
|---|---|---|
| Bayer Study Director | Bayer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Phoenix | Arizona | United States | ||||
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| Baseline and at 24 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 36 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 48 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 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) | 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 24 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 36 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 48 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 60 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 |
| Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN) | Baseline and at 24 months |
| Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN) | Baseline and at 36 months |
| Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN) | Baseline and at 48 months |
| Patient Reported outcome: Change of adherence as measured by and Validated Hemophilia Regimen Treatment Adherence Scale (Veritas Pro/PRN) | Baseline and at 60 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 satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT) | Baseline and at 24 months |
| Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT) | Baseline and at 36 months |
| Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT) | Baseline and at 48 months |
| Patient Reported outcome: Change of satisfaction as measured by Hemophilia Treatment Satisfaction Questionnaire (Hemo-SAT) | Baseline and at 60 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 pain as measured by Brief Pain Inventory (BPI-SF) | Baseline and at 36 months |
| Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF) | Baseline and at 48 months |
| Patient Reported outcome: Change of pain as measured by Brief Pain Inventory (BPI-SF) | Baseline and at 60 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 number of resource utilization as evaluated from the patient perspective | Baseline and at 24 months |
| Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective | Baseline and at 36 months |
| Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective | Baseline and at 48 months |
| Patient Reported outcome: Change of number of resource utilization as evaluated from the patient perspective | 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 | Baseline and at 12 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 | Baseline and at 24 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 | Baseline and at 36 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 | Baseline and at 48 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 | Baseline and at 60 months |
| Baseline and every 12 months up to 60 months |
| Clinical Outcome: Change of Joint Status | Joint Status as defined by: - Hemophilia Joint Health Score/ Pettersson Additive Scale - Range of motion - Imaging (where available) - Target joints and past surgical procedures | Baseline and every 12 months up to 60 months |
| Clinical Outcome: Baseline disease characteristics | 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 | Baseline |
| Clinical Outcome: Change of treatment patterns including Hemophilia treatments via patient chart | 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 | Baseline and every 12 months up to 60 months |
| Aurora |
| Colorado |
| United States |
| Washington D.C. | District of Columbia | United States |
| New Orleans | Louisiana | United States |
| Detroit | Michigan | United States |
| Minneapolis | Minnesota | United States |
| Columbus | Ohio | United States |
| Multiple Locations | Japan |
| Multiple Locations | Spain |
| Multiple Locations | United Kingdom |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| D005169 | Factor VIII |
| C414350 | BAY 14-2222 |
| ID | Term |
|---|---|
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011498 | Protein Precursors |
| D001685 | Biological Factors |
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