Evaluation of Joint Arthropathy Using Ultrasound Technique in Children With Severe Hemophilia Undergoing Prophylaxis Regimen
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 18
- 试验地点
- 4
- 主要终点
- Prevalence and natural progression of subclinical arthropathy (joint changes) in children with severe hemophilia undergoing prophylaxis using a validated ultrasound (HEAD-US) protocol.
研究概览
简要总结
To evaluate the prevalence of subclinical arthropathy in children with severe hemophilia undergoing a prophylaxis regimen and without evidence of target joints, using a validated ultrasound scoring method.
详细描述
Persons with Hemophilia (A or B) often experience recurrent joint bleeds, most commonly affecting the ankles, knees and elbows. These bleeds can lead to significant pain and disability over time. If recurrent joint bleeds are not managed with prompt and adequate infusions of factor concentrate, the damage caused by the presence of blood in the joint space will eventually result in a condition called debilitating chronic hemophilic arthropathy.
The initiation of and adherence to a prophylactic infusion regimen, starting with the first or second joint bleed, is essential for prevention of progression to arthropathy.
Studies have demonstrated that prophylaxis with recombinant or plasma-derived factor VIII or IX concentrates is effective in preventing clinical joint bleeds and the progression to debilitating joint disease in patients with severe hemophilia A or B respectively. However, for patients on prophylaxis, the absence of symptomatic joint bleeds and/or structural and functional abnormalities of joints on physical examination and plain radiographic images can lead to the erroneous assumption that the prophylaxis is completely effective. It has been established that patients with severe hemophilia are still at risk for subclinical bleeding ("microbleeds") despite seemingly adequate prophylaxis.
Young adults, despite a lifetime on prophylaxis and apparently normal joints are developing arthropathy in their 20's and 30's. Prophylaxis as currently practiced may only be delaying the onset of clinical joint disease.
The recent advancements in ultrasound imaging (US) have been proven to be effective in confirming a joint bleed, monitoring the evolution of a joint bleed and assessing the resolution or recurrence of a bleed. Previous studies have evaluated the prevalence of subclinical arthropathy in young hemophilic adults using both US and MRI techniques and concluded that US is as effective and sensitive as MRI identifying these subclinical joint abnormalities.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 30 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Severe Hemophilia Cohort:Patients from 0 up to 30 months of age with diagnosis of severe hemophilia A or B defined as a factor VIII:C/IX:C of <1% undergoing prophylaxis regimen with any factor VIII or IX concentrate and without evidence (clinical or by history) of target joint disease.
- •Mild/moderate Hemophilia Cohort:Patients from 0 up to 30 months of age with diagnosis of mild hemophilia A or B defined as a factor VIII:C/IX:C of 5-50% and those with diagnosis of moderate hemophilia A or B defined a s a factor VIII:C/IX:C of 1-5% without evidence (clinical or by history) of target joint disease and no history of spontaneous joint bleeds.
排除标准
- •Patients with concomitant Hepatitis B, Hepatitis C and HIV viral infections (because of a recognized arthritogen effect).
- •Present or prior history of anti FVIII or IX inhibitors.
- •Known inflammatory joint disease.
- •Established target joint.
结局指标
主要结局
Prevalence and natural progression of subclinical arthropathy (joint changes) in children with severe hemophilia undergoing prophylaxis using a validated ultrasound (HEAD-US) protocol.
时间窗: 5 years
Two independent readers will describe all the ultrasonography positive joint findings and/or changes on a standardized spreadsheet; they will also rate and score these changes based on the HEAD-US scale providing a final score for each of the evaluated joints (ankles and knees).
Prevalence and natural progression of subclinical arthropathy (joint changes) in children with mild and/or moderate hemophilia using a validated ultrasound (HEAD-US) protocol.
时间窗: 5 years
Two independent readers will describe all the ultrasonography positive joint findings and/or changes on a standardized spreadsheet; they will also rate and score these changes based on the HEAD-US scale providing a final score for each of the evaluated joints (ankles and knees).
次要结局
- Characteristics of subclinical joint changes (time of presentation, natural evolution, etc.) between patients with severe hemophilia and those with mild/moderate hemophilia.(5 years)
研究者
Fernando F. Corrales-Medina
MD
University of Miami