Bone Microarchitecture in Men With Hemophilia
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- number of trabecular at distal tibia
研究概览
简要总结
Hemophilia A and B are hereditary sex-linked deficiencies of coagulation factors VIII and IX characterized by bleeding. Their modern therapy increases life expectancy and risk of age-related diseases, e.g., osteoporosis.
Hemophilia-specific risk factors impair formation of peak bone mass and accelerate bone loss. Fractures are more frequent in hemophilic men vs. age-matched men and induce bleeding which is aggravated by manipulations and surgical intervention.
The hypothesis of this study is that hemophilic men have poor bone microarchitecture (assessed by High-resolution peripheral quantitative computed tomography (HR-pQCT)) related to an imbalance between bone formation and resorption (assessed by bone turnover markers (BTM) and bone biomarkers).
The study aims to assess the difference in low trabecular number (Tb.N) at the distal radius between hemophilic men (cases) and age- height-weight-ethnicity and smoking-matched healthy men (controls). Correlation between BTM and Tb.N will be also studied.
Biologic markers of bone remodeling (C-terminal telopeptide of type I collagen (PINP), N-terminal propeptide of type I procollagen (CTX-I), periostin) will be studied.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 20 Years 至 60 Years（Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Men aged 20 to 60 years
- •Severe hemophilia A or B (FVIII or FIX<1%)
- •Regular followed up in Lyon Hemophilia Center
- •Ability to give free and informed consent
- •Person capable of actively participating in radiological examinations
- •Healthy Mens:
- •Aged 20 to 60 years
排除标准
- •Not covered by health system
- •Vulnerable (adults unable to consent, protected under guardianship, prisoner)
- •Any blood coagulation abnormality other than severe hemophilia A or B
- •Having one of the following treatments against osteoporosis of more than 6 months : bisphosphonates, denosumab, teriparatide
- •With a chronical disease having a high impact on bone structure and no related to hemophilia disease, such as Cushing or Crohn diseases.
研究组 & 干预措施
severe hemophilia A or B patients
30 patients with severe hemophilia A or B (Factor (F)VIII or Factor IX (FIX)≤ 1%) aged 20 to 60 years will be included in this study.
干预措施: HR-pQCT (Radiation)
severe hemophilia A or B patients
30 patients with severe hemophilia A or B (Factor (F)VIII or Factor IX (FIX)≤ 1%) aged 20 to 60 years will be included in this study.
干预措施: Blood sample (Biological)
severe hemophilia A or B patients
30 patients with severe hemophilia A or B (Factor (F)VIII or Factor IX (FIX)≤ 1%) aged 20 to 60 years will be included in this study.
干预措施: Dual energy X-ray absorptiometry (Radiation)
healthy men
Data of healthy men, matching in age- height-weight-ethnicity and smoking-matched with patient will be collected. These data are already available at the "Institut national de la santé et de la recherche médicale" (INSERM) research unit associated.
干预措施: medical data collection (Other)
结局指标
主要结局
number of trabecular at distal tibia
时间窗: 3 months following the inclusion
The number of trabecular at distal tibia will be performed and compared to the values available for the healthy control group
number of trabecular at distal radius
时间窗: 3 months following the inclusion
The number of trabecular at distal radius will be performed and compared to the values available for the healthy control group
次要结局
- Sera bone remodeling biomarkers(3 months following the inclusion)
- Trabecular Bone Score (TBS)(3 months following the inclusion)
- reflection of bone strength(3 months following the inclusion)