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| ID | Type | Description | Link |
|---|---|---|---|
| KG1411GR | Other Identifier | Company Internal |
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This study is a prospective, single center, observational, 2-cohort study of adult patients with severe Hemophilia A. There is no randomization procedure and all patients will be treated as per usual clinical practice. Patients will be followed up for 18 months after enrolment.
The primary therapy for hemophilia is coagulation factor replacement, given either episodically on demand (OD) for the treatment of acute bleeds or prophylactically to prevent them.
The last years there has been an interest to study the comparative effectiveness of OD vs. SP (Secondary Prophylaxis) treatment strategies so as to create an evidence based platform to guide both clinical decision making and quantify the economic aspects of each treatment alternative. In Greece relevant information is lacking, thus it is imperative to study how patients on different treatment strategies are managed in a real-life setting in Greece, including the main factors associated with increased health care utilization, clinical success and patient well being.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Adult patients with severe Hemophilia A.(25 patients on Secondary Prophylaxis treatment) |
| |
| Cohort 2 | Adult patients with severe Hemophilia A.(50 patients on On Demand treatment) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Survey | Other | Recombinant antihemophilic factor VIII (FVIII) on Secondary Prophylaxis treatment scheduled according to daily routine |
|
| Measure | Description | Time Frame |
|---|---|---|
| Annual direct medical cost of specialized hemophilia centre for patients with the on-demand strategy | At 18 months | |
| Annual direct medical cost of specialized hemophilia centre for patients with secondary prophylaxis | At 18 months |
| Measure | Description | Time Frame |
|---|---|---|
| Mean cost per joint bleed episodes for overall patients | At 18 months | |
| Mean cost per joint bleed episodes by cohort for patients | At 18 months | |
| Number of joint bleed episodes for overall patients |
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Inclusion Criteria:
Exclusion Criteria:
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This will be a single center prospective two-cohort observational study conducted in a hospital setting.Patients will be enrolled from the biggest specialty management center in Greece
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| Name | Affiliation | Role |
|---|---|---|
| Bayer Study Director | Bayer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Multiple Locations | Greece |
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| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| D011795 | Surveys and Questionnaires |
| ID | Term |
|---|---|
| D003625 | Data Collection |
| D004812 | Epidemiologic Methods |
| D008919 | Investigative Techniques |
| D017531 | Health Care Evaluation Mechanisms |
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| Survey | Other | Recombinant antihemophilic factor VIII (FVIII) On Demand treatment scheduled according to daily routine |
|
| At 18 months |
| Number of joint bleed episodes by cohort for patients. | At 18 months |
| Severity of joint bleed episodes for overall patients. | At 18 months |
| Severity of joint bleed episodes by cohort for patients. | At 18 months |
| Mean EQ-VAS score as reported from patients at the time of a major bleed. | EQ-VAS score is Standardized questionnaire for hemophilia | At 18 months |
| Mean EQ-5D score as reported from patients at the time of a major bleed. | EQ-5D The EuroQol standardized instrument of health outcome | At 18 months |
| Mean change from baseline in Quality of Life scores derived from the Haem-A-Qol and EQ-5D questionnaires | Haem-A-Qol Specific questionnaire for hemophilia and EQ-5D The EuroQol standardized instrument of health outcome | Baseline and at 18 months |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D011787 | Quality of Health Care |
| D017530 | Health Care Quality, Access, and Evaluation |
| D011634 | Public Health |
| D004778 | Environment and Public Health |