Hemophilia A
Conditions
Keywords
Hemophilic arthropathy, Diagnostic ultrasound, Severe hemophilia A
Brief summary
Patients with severe hemophilia A often develop joint complication requiring careful monitoring. This study aimed to compare the effectiveness of standard low dose weight-based FVIII concentrates (CFCs) prophylaxis with pharmacokinetic-guided extended half-life FVIII concentrates (eHLFVIII) using cartilage biomarkers and HEAD-US score.
Interventions
DIAGNOSTIC_TESTHEAD-US
HEAD-US for evaluate haemophilic arthropathy in 6 joints. (Elbow, Knee, Ankle)
DIAGNOSTIC_TESTUrinary carboxy-terminal cross linked telopeptide of type II collagen
Cartilage biomarker represent cartilage degradation
DIAGNOSTIC_TESTInflammatory markers
Inflammatory markers included CBC, Ferritin, ESR, CRP
Sponsors
Chulalongkorn University
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
PwSHA receiving low-dose FVIII prophylaxis for ≥6 months were enrolled. Weight-based low-dose FVIII prophylaxis (10-15 IU/kg, 1-3 times/week) was continued for 4 months, then switched to PK-guided low-dose EHL FVIII prophylaxis, targeting a trough FVIII activity of 1 IU/dL, for another 4 months. ESR, CRP, ferritin, and CTX-II were measured at week 8 (during the weight-based low-dose regimen) and at week 24 (during the PK-guided regimen). HEAD-US scores were also compared at weeks 16 and 32. Bleeding rates and quality-of-life (QoL) score were analyzed parallelly.
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* patients with severe hemophilia A * previously receiving standard low dose FVIII prophylaxis 1-3 times per week for at least 6 months prior to enrollment in the project * no history of FVIII inhibitor (\<0.6 Bethesda unit/ml) * obtained consent from both patients and their parents
Exclusion criteria
* patients with underlying condition affecting the joints such as osteoarthritis, metabolic bone disease, etc.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The effectiveness of standard low dose FVIII concentrate prophylaxis and PK-guided eHL FVIII prophylaxis | From enrollment to the end of treatment at 32 weeks | To compare the effectiveness of standard weight base FVIII concentrate prophylaxis and pharmacokinetic guided extended half life FVIII prophylaxis in managing hemophilic arthropathy using cartilage biomarkers. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Correlation between cartilage biomarkers and Inflammatory markers | From enrollment to the end of treatment at 32 weeks | To investigate the correlation between cartilage biomarkers (Urine CTX-II), Inflammatory markers (CBC, Ferritin, ESR, CRP) and ultrasound (HEAD-US score) comparing standard weight base low dose prophylaxis with pharmacokinetic guided extended half life FVIII concentrates |
Countries
Thailand
Outcome results
None listed