Cartilage Biomarkers and HEAD-US Score in Severe Hemophilia A Patients Receiving FVIII Prophylaxis (HEADUS_SHA8)

Chulalongkorn University

Status: Invitation-only

Conditions: Hemophilia A

Treatments: Diagnostic Test: Inflammatory markers; Diagnostic Test: HEAD-US; Diagnostic Test: Urinary carboxy-terminal cross linked telopeptide of type II collagen

Study type: Interventional

Funder types: Other

Identifiers

NCT06816953

0736/67

Ratchadapisek Research Fund (Other Grant/Funding Number)

Details and patient eligibility

About

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.

Enrollment: 19 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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19 participants in 2 patient groups

Standard low dose FVIII prophylaxis

Experimental group

Description:

Standard weight based low dose FVIII concentrates prophylaxis

Treatment:

Diagnostic Test: Urinary carboxy-terminal cross linked telopeptide of type II collagen

Diagnostic Test: HEAD-US

Diagnostic Test: Inflammatory markers

PK guided Extended half life FVIII prophylaxis

Active Comparator group

Description:

Pharmacokinetic guided extended half life FVIII prophylaxis

Treatment:

Diagnostic Test: Urinary carboxy-terminal cross linked telopeptide of type II collagen

Diagnostic Test: HEAD-US

Diagnostic Test: Inflammatory markers