Cartilage Biomarkers and HEAD-US Score in Severe Hemophilia A Patients Receiving Low Dose FVIII Prophylaxis
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 19
- 试验地点
- 1
- 主要终点
- The effectiveness of standard low dose FVIII concentrate prophylaxis and PK-guided eHL FVIII prophylaxis
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •patients with underlying condition affecting the joints such as osteoarthritis, metabolic bone disease, etc.
研究组 & 干预措施
Standard low dose FVIII prophylaxis
Standard weight based low dose FVIII concentrates prophylaxis
干预措施: HEAD-US (Diagnostic Test)
Standard low dose FVIII prophylaxis
Standard weight based low dose FVIII concentrates prophylaxis
干预措施: Urinary carboxy-terminal cross linked telopeptide of type II collagen (Diagnostic Test)
Standard low dose FVIII prophylaxis
Standard weight based low dose FVIII concentrates prophylaxis
干预措施: Inflammatory markers (Diagnostic Test)
PK guided Extended half life FVIII prophylaxis
Pharmacokinetic guided extended half life FVIII prophylaxis
干预措施: HEAD-US (Diagnostic Test)
PK guided Extended half life FVIII prophylaxis
Pharmacokinetic guided extended half life FVIII prophylaxis
干预措施: Urinary carboxy-terminal cross linked telopeptide of type II collagen (Diagnostic Test)
PK guided Extended half life FVIII prophylaxis
Pharmacokinetic guided extended half life FVIII prophylaxis
干预措施: Inflammatory markers (Diagnostic Test)
结局指标
主要结局
The effectiveness of standard low dose FVIII concentrate prophylaxis and PK-guided eHL FVIII prophylaxis
时间窗: 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.
次要结局
- Correlation between cartilage biomarkers and Inflammatory markers(From enrollment to the end of treatment at 32 weeks)