Cartilage Biomarkers and HEAD-US Score in Severe Hemophilia A Patients Receiving FVIII Prophylaxis

N/AEnrolling By InvitationNCT06816953

Chulalongkorn University19 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hemophilia A

Interventions

HEAD-USDIAGNOSTIC_TEST

HEAD-US for evaluate haemophilic arthropathy in 6 joints. (Elbow, Knee, Ankle)

Urinary carboxy-terminal cross linked telopeptide of type II collagenDIAGNOSTIC_TEST

Cartilage biomarker represent cartilage degradation

Inflammatory markersDIAGNOSTIC_TEST

Inflammatory markers included CBC, Ferritin, ESR, CRP

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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.

Locations (1)

Pediatric Department, Faculty of Medicine, Chulalongkorn University and King Chulalongkorn Memorial Hospital

Bangkok, Patumwan, Thailand

Outcomes

Primary Outcomes

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.

Time frame: 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

Time frame: From enrollment to the end of treatment at 32 weeks

Secondary Outcomes

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

Time frame: From enrollment to the end of treatment at 32 weeks

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.