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.
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: 1. Investigate the association among thermography findings and functional status in hemophilia arthroplasty 2. 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.
Study Type
OBSERVATIONAL
Enrollment
35
Taipei Medical University
Taipei, Taiwan
Hemophilia Joint Health Score (HJHS)
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.
Time frame: Baseline to 24 months follow up
Musculoskeletal ultrasound/HEAD-US scoring system (Haemophilia Early Arthropathy Detection by UltraSound)
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.
Time frame: Baseline to 24 months follow up
X-rays/Pettersson score (PS)
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.
Time frame: Baseline to 24 months follow up
Thermography
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.
Time frame: Baseline to 24 months follow up
Hemophilia activities list (HAL)
The HAL measures the impact of hemophilia on self-perceived functional abilities in 7 domains, including Lying/sitting/kneeling/standing, Functions of the legs , Functions of the arms, Use of transportation, Self-care, Household tasks, Leisure activities and sports.
Time frame: Baseline to 24 months follow up
Hemophilia Quality of Life Questionnaire for Adults (HAEM-A-QOL)
The HAEM-A-QOL assess health-related quality of life in haemophilia patients with 10 domains, including physical health, feelings, view, sport\& leisure, work\& school, dealing, treatment, future, family planning, and partnership.
Time frame: Baseline to 24 months follow up
Quality of life assessment (EQ-5D) - Descriptive System
The EQ-5D measures generic health status with 2 components: descriptive system and visual analogue scale. The descriptive system comprises five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression.
Time frame: Baseline to 24 months follow up
Quality of life assessment (EQ-5D) - Visual Analogue Scale (VAS)
The EQ-5D measures generic health status with 2 components: descriptive system and visual analogue scale. The visual analogue scale (VAS) records the patient's self-rated health on a vertical visual analogue scale
Time frame: Baseline to 24 months follow up
Serum markers: cartilage oligomeric matrix protein (COMP)
COMP is considered a marker of cartilage breakdown, and is diagnostic of arthritis and to correlate with the disease severity. Serum COMP level is indicative of the amount of joint damage in patients with hemophilic arthropathy.
Time frame: Baseline to 24 months follow up
Serum markers: chondroitin-sulphate aggrecan turnover 846 epitope (CS846)
CS846 is a sensitive biomarker reflecting degradation of cartilage and synovial tissues. Serum CS-846 levels is indicative of the amount of joint damage in patients with hemophilic arthropathy.
Time frame: Baseline to 24 months follow up
6 mins walk test
The 6 mins walk test (MWT) measures the distance an individual could walk over six minutes on a hard, flat surface. The test could be used as a performance-based measure of functional exercise capacity. Gait Analysis (RehaWatch): RehaWatch is based on inertial sensors that allow the quantitative measurement of the kinemetic variables
Time frame: Baseline to 24 months follow up
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