Observational Study of Long-Term Joint Health Outcome in Hemophilia Patient
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 35
- 试验地点
- 1
- 主要终点
- Musculoskeletal ultrasound/HEAD-US scoring system (Haemophilia Early Arthropathy Detection by UltraSound)
研究概览
简要总结
Patients with severe hemophilia A can experience repeated bleeding into the same joint (ie, a target joint; most frequently in the ankle, knee, and elbow), which can contribute to hemophilic arthropathy and, over time, ultimately result in chronic pain, functional limitations, deformities, reduced joint of motion and decreased quality of life.
Early use of prophylaxis is recommended following diagnosis of severe hemophilia A to maintain joint health and prevent joint destruction. Eloctate is produced using a human cell line and an addition of an Fc fusion protein to recombinant FVIII (rFVIIIFc) with prolonged half-life and was launched in Taiwan from Nov. 2018. The pivotal studies of rFVIIIFc show that patients maintained a low bleeding rate, with most experiencing a median annualized bleeding rate (ABR) of 0 and 97% of target joints were resolved across adult, adolescent, and pediatric subjects during 4 years of prophylaxis rFVIIIFc treatment. However, in Taiwan we are still lacking the real world treatment outcome data on rFVIIIFc, especially for the joint health evidence in Asian Hemophilia A patients.
Therefore the objective of this study is to evaluate the effectiveness of rFVIIIFc treatment on joint health over a long observational period of 36 month focused on physical and functional changes in hemophilia A patients.
详细描述
Thermographic assessment has been proposed to be a potential tool in evaluating the inflammatory arthritis patients. Thermography has advantages of simple, time-saving, low skill demanding, no radiation exposure and non-invasiveness. It is possible to conduct a quantitative analysis with thermography. However, it is still unknown whether thermography evaluation can provide clinical information regarding the joint health in hemophilia arthropathy patients. The project will focus on:
- Investigate the association among thermography findings and functional status in hemophilia arthroplasty
- Investigate the novel biomarkers in predicting function in hemophilia arthropathy including: soft tissue ultrasound, body composition, thermographic findings of joints, muscle composition and serum biomarkers of joint degeneration.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Other
入排标准
- 年龄范围
- 20 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Willing to sign inform consent form and complied with study procedure
- •Patient received confirmative diagnosis of Hemophilia A Disease
- •Severe hemophilia A patient currently who are on treatment with Eloctate prior to enrolment visit (Note: Prescription based on physician's clinical judgement and discussion with patients)
排除标准
- •Unwilling to sign inform consent form.
- •History of major neurological disease (eg. Stroke, Parkinson's disease, neuropathy, etc.)
- •History of major psychiatric disease (eg. Schizophrenia, bipolar disorder)
- •Significantly impaired vision / hearing
- •Cannot communicate in Mandarin Chinese
- •Recent joint bleeding and trauma 3 months prior to study
结局指标
主要结局
Musculoskeletal ultrasound/HEAD-US scoring system (Haemophilia Early Arthropathy Detection by UltraSound)
时间窗: Baseline to 24 months follow up
Musculoskeletal ultrasound could detect soft tissue alterations and be used as early detection of hemophilic arthropathy. Ultrasound assessment of bilateral knees and ankles, and bilateral gastrocnemius and quadriceps would be taken out in this study and scored with HEAD-US(Haemophilia Early Arthropathy Detection by UltraSound) scoring system. The scoring was based on three markers: synovitis (score 0-2), cartilage (score 0-4) and subchondral bone (score of 0-2) with a maximum score of eight points per joint, higher score is worse.
X-rays/Pettersson score (PS)
时间窗: Baseline to 24 months follow up
X-rays are used to evaluate structural changes in joints of patients with hemophilic arthropathy. X-rays images of bilateral knees and ankles will be taken in this study and scored with Pettersson scoring system. The maximum possible score for a given joint is 13 points and higher score is worse.
Thermography
时间窗: Baseline to 24 months follow up
Infrared thermography could be a simple tool to detect early inflammation of joints and joint associated structures. FLIR ONE imaging camera is used to take thermal images.
Hemophilia Joint Health Score (HJHS)
时间窗: Baseline to 24 months follow up
The HJHS measures joint health, in the domain of body structure and function (i.e. impairment), of the joints most commonly affected by bleeding in hemophilia: the knees, ankles, and elbows. The HJHS provides a total score (higher score is worse; max=124), joint specific scores, and a global gait score.
次要结局
- Hemophilia activities list (HAL)(Baseline to 24 months follow up)
- Quality of life assessment (EQ-5D) - Visual Analogue Scale (VAS)(Baseline to 24 months follow up)
- 6 mins walk test(Baseline to 24 months follow up)
- Serum markers: chondroitin-sulphate aggrecan turnover 846 epitope (CS846)(Baseline to 24 months follow up)
- Hemophilia Quality of Life Questionnaire for Adults (HAEM-A-QOL)(Baseline to 24 months follow up)
- Quality of life assessment (EQ-5D) - Descriptive System(Baseline to 24 months follow up)
- Serum markers: cartilage oligomeric matrix protein (COMP)(Baseline to 24 months follow up)