Severe Hemophilia A Without Inhibitor
Conditions
Brief summary
Study Objectives\* 1. Provide a systematic evaluation of the treatment outcomes in patients with hemophilia A 2. Emphasize the importance and clinical benefits of rFVIII-Fc in joint and bone health. 3. Compare the clinical outcomes from 1 year before and after switching to EHL. 4. Exploratory: Identify biomarkers that could provide more useful and convenient evaluations of joint and bone health. (Time-saving and easy to monitor)
Detailed description
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.
Interventions
DIAGNOSTIC_TESTserum biomarker.
serum biomarker:CTX-II, COMP, hsCRP, TNF-a, CTX-I, sRANKL, OPG, and Osteopontin (OPN)
Sponsors
Tri-Service General Hospital
Study design
Observational model
OTHER
Time perspective
OTHER
Eligibility
Sex/Gender
MALE
Healthy volunteers
Yes
Inclusion criteria
1. Patients with severe hemophilia A (all ages). 2. Patients with moderate type hemophilia A with hemophilic arthropathy. 3. Currently on rFVIIIFc regular prophylaxis and have previous medical record with regular prophylaxis with standard half-life product for one year 4. Able and willing to undergo joint and bone examinations
Exclusion criteria
1. Participants of other interventional studies. 2. Patients with current inhibitors. 3. History of major neurological disease (eg. Stroke, Parkinson's disease, neuropathy, etc.) 4. History of major psychiatric disease (eg. Schizophrenia, bipolar disorder) 5. Significantly impaired vision/hearing
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Systematic joint and bone health assessments before and after enrollment | up to 5 years | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Serum markers | up to 5 years | CTX-II, COMP, HSCRP, TNF-ALPHA, CTX-I, SRANKL, OPG, OPN |
Countries
Taiwan
Contacts
Primary ContactYeu-Chin Chen
Outcome results
None listed