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| Name | Class |
|---|---|
| Hemab ApS | INDUSTRY |
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To understand the lived experiences of people with Glanzmanns Thrombasthenia
A qualitative study of people with Glanzmanns Thrombasthenia including an on-line survey, qualitative in-depth interviews and bleed diary completion.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Qualitative assessment | Survey (using validated quality of life assessment questionnaires) and one to one interviews of people and family members living with Glanzmanns Thrombasthenia |
| |
| Bleed diary | Daily bleed diary completion over 12 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| survey and interview | Other | qualitative assessment |
|
| Measure | Description | Time Frame |
|---|---|---|
| to understand the impact of GT on quality of life of affected individuals and their families. | Incidence of reported impact of GT on daily life using validated questionnaires EQ5D, MIQ, PHQ9, Rosenbergs self esteem scale. | one year |
| Measure | Description | Time Frame |
|---|---|---|
| Identify levels of acceptability of current treatments and management approaches | Rate of reported satisfaction with current treatments using self efficacy to manage disease assessment tool | one year |
| Identify areas of unmet need among people with GT |
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Inclusion Criteria:
Clinical diagnosis of congenital (inherited) Glanzmanns Thrombasthenia
Exclusion Criteria:
Acquired Glanzmanns Thrombasthenia
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People with Glanzmanns Thrombasthenia or parents of children with Glanzmanns Thrombasthenia.
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| Name | Affiliation | Role |
|---|---|---|
| Mike Holland, BSC | Haemnet | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Oxford University Hospitals NHS Foundation Trust | Oxford | Oxfordshire | OX3 7LE | United Kingdom |
The second part of the study will be conducted by Haemnet and Hemab - participant information will be shared after reconsenting
3-6 months
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| ID | Term |
|---|---|
| D013915 | Thrombasthenia |
| 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 |
| D007407 | Interviews as Topic |
| ID | Term |
|---|---|
| D003625 | Data Collection |
| D004812 | Epidemiologic Methods |
| D008919 | Investigative Techniques |
| D017531 | Health Care Evaluation Mechanisms |
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Rate of reported unmet need using ED5D and MIQ
| one year |
| D001791 | Blood Platelet 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 |