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| ID | Type | Description | Link |
|---|---|---|---|
| Ratchadapisek Research Fund | Other Grant/Funding Number | Chulalongkorn University |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Standard low dose FVIII prophylaxis | Experimental | Standard weight based low dose FVIII concentrates prophylaxis |
|
| PK guided Extended half life FVIII prophylaxis | Active Comparator | Pharmacokinetic guided extended half life FVIII prophylaxis |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| HEAD-US | Diagnostic Test | HEAD-US for evaluate haemophilic arthropathy in 6 joints. (Elbow, Knee, Ankle) |
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| Measure | Description | Time Frame |
|---|---|---|
| The effectiveness of standard low dose FVIII concentrate prophylaxis and PK-guided eHL FVIII prophylaxis | 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. | From enrollment to the end of treatment at 32 weeks |
| The effectiveness of standard low dose FVIII concentrate prophylaxis and PK-guided eHL FVIII prophylaxis | To compare the effectiveness of standard weight base FVIII concentrate prophylaxis and pharmacokinetic guided extended half life FVIII prophylaxis in managing hemophilic arthropathy using HEAD-US score. | From enrollment to the end of treatment at 32 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Correlation between cartilage biomarkers and Inflammatory markers | 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 | From enrollment to the end of treatment at 32 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Pediatric Department, Faculty of Medicine, Chulalongkorn University and King Chulalongkorn Memorial Hospital | Bangkok | Patumwan | 10330 | Thailand |
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1 year
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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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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.
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| Urinary carboxy-terminal cross linked telopeptide of type II collagen | Diagnostic Test | Cartilage biomarker represent cartilage degradation |
|
| Inflammatory markers | Diagnostic Test | Inflammatory markers included CBC, Ferritin, ESR, CRP |
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| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |