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Study Objectives*
In this study, we would like to focus on the evaluation of joint and bone health in persons with severe hemophilia A (PwHA) treated with rFVIIIFc prophylaxis. We also aim to identify potential biomarkers for assessing the joint and bone health of PwHA. Since the joint and bone health examinations are time-consuming, expensive, and require special equipment, the patients could only receive the examinations annually to realize the status of their joint health. The biomarkers testing will provide a more convenient and time-saving option on the joint and bone health evaluation for PwHA. Therefore, surrogate of biochemical marker(s) to represent the joint and BMD progression is an unmet need for clinical practice. Investigating different biomarkers and combining them with the joint and BMD results to provide a systematic approach for elucidating the treatment outcomes of PwHA treated with rFVIIIFc is major goal of this study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Systematic joint and bone health assessments before and after enrollment |
|
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| serum biomarker. | Diagnostic Test | serum biomarker:CTX-II, COMP, hsCRP, TNF-a, CTX-I, sRANKL, OPG, and Osteopontin (OPN) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Systematic joint and bone health assessments before and after enrollment | Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) on patients' ankles, knees, and elbows. Also evaluate the Hemophilia Joint Health Score (HJHS) and Dexa scan at the hip. | up to 5 years |
| Measure | Description | Time Frame |
|---|---|---|
| Serum markers | CTX-II, COMP, HSCRP, TNF-ALPHA, CTX-I, SRANKL, OPG, OPN | up to 5 years |
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Inclusion Criteria:
Exclusion Criteria:
Males with one affected X chromosome is defined hemophilia disease; a female with one affected X chromosome is a "carrier" of hemophilia.
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severe hemophilia A patients or moderate hemophilia A patients with hemophilic arthropathy.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Yeu-Chin Chen | Contact | (+886)2-87923311 | 13654 | yeuchin99@gmail.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Tri-Service General Hospital Hemophilia Care Center | Recruiting | Taipei | Neihu Dist. | 114202 | Taiwan |
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Blood samples (serum) will be obtained for serum markers.