Association Between Low Bone Density, Vertebral Fractures, and Pain in Sickle Cell Disease
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 53
- 试验地点
- 2
- 主要终点
- Lumbar Spine Bone Mineral Density
研究概览
简要总结
A prospective study to determine how low bone mineral density and/or vertebral compression fractures associate with pain in adults with sickle cell disease
详细描述
The investigators hypothesize that adults with sickle cell disease (SCD) and low bone density and/or vertebral compression fractures on a dual X-ray absorptiometry (DXA) scan (adjusted for age, sex, SCD genotype, relevant labs, presence of osteonecrosis, and SCD-modifying therapies) will report more severe pain than those with normal bone density or no vertebral fractures. The Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) is a validated patient-reported outcome measure of physical, mental, and social health in adults with SCD. This cross-sectional observational study involves obtaining a baseline DXA scan, vertebral fracture analysis (VFA) and pain assessment using ASCQ-Me pain impact scores. The investigators plan to recruit 50 adults with SCD followed at University of California Davis Medical Center between Nov 2022- Dec 2023 and anticipate enrolling up to 4 adults with SCD per month. The study endpoints are listed below:
- To determine the association between bone density Z-scores and ASCQ-Me pain impact scores in a prospective cohort of adults with SCD
- To study the association between Spine Deformity Index scores (SDI, a proxy for vertebral fracture analysis) and ASCQ-Me pain impact scores in a prospective cohort of adults with SCD
- To assess the correlation between baseline hematological and biochemical laboratory parameters (including bone biomarkers), bone density, and/or vertebral fractures in a prospective cohort of adults with SCD
The investigators' goal is to complete primary data analysis by Mar 2024. As an exploratory endpoint, 1cc of serum and 5cc of urine will be collected from each study participant once (at baseline), after an overnight fast, for bone biomarker analyses.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Screening
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 18-80 years with SCD (any genotype, confirmed by hemoglobin electrophoresis or high performance liquid chromatography)
- •Ability to provide written informed consent
- •Ability to lay on a DXA scanner
- •Negative urine pregnancy test for women of childbearing potential at study entry
排除标准
- •Pregnant women
- •Adults unable to consent
- •Individuals who are not yet adults (infants, children, teenagers)
- •Prisoners
- •Hospitalizations (any cause) within 2 weeks of study entry
结局指标
主要结局
Lumbar Spine Bone Mineral Density
时间窗: Baseline
Areal bone mineral density of the lumbar spine measured by dual-energy X-ray absorptiometry (DXA) scan and reported in grams per square centimeter.
Femoral Neck Bone Mineral Density (BMD)
时间窗: Baseline
Areal bone mineral density of the femoral neck measured by dual-energy X-ray absorptiometry (DXA) scan and reported in grams per square centimeter.
Femoral Neck Bone Mineral Density Z-scores
时间窗: Baseline
Number of standard deviations between measured lumbar spine bone mineral density (g/cm2) for each participant and mean lumbar spine bone mineral density (g/cm2) of the reference population. A Z-score of 0 represents the mean of the reference population. A negative Z-score means the measured bone mineral density values are lower (worse) the reference mean, while positive Z-scores mean they are above/higher. Bone mineral density Z-scores ≤ -2 indicates low bone density.
Lumbar Spine Bone Mineral Density-Z-scores
时间窗: Baseline
Number of standard deviations between measured lumbar spine bone mineral density (g/cm2) for each participant and mean lumbar spine bone mineral density (g/cm2) of the reference population. A Z-score of 0 represents the mean of the reference population. A negative Z-score means the measured bone mineral density values are lower (worse) the reference mean, while positive Z-scores mean they are above/higher. Bone mineral density Z-scores ≤ -2 indicates low bone density.
Total Hip Bone Mineral Density (BMD)
时间窗: Baseline
Areal bone mineral density of the total hip measured by dual-energy X-ray absorptiometry (DXA) scan and reported in grams per square centimeter.
Total Hip Bone Mineral Density-Z-scores
时间窗: At enrollment
Number of standard deviations between measured total hip bone mineral density (g/cm2) for each participant and mean total hip bone mineral density (g/cm2) of the reference population. A Z-score of 0 represents the mean of the reference population. A negative Z-score means the measured bone mineral density values are lower (worse) the reference mean, while positive Z-scores mean they are above/higher. Bone mineral density Z-scores ≤ -2 indicates low bone density.
Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact T-scores
时间窗: Baseline
Patient-reported outcome measure of pain impact in the preceding 7 days before bone density measurements. The Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) pain impact T-scores range from about 30-100. The ASCQ-Me pain impact T-score has standardized mean T-score of 50 and standard deviation of 10, which were derived from a reference population of ambulatory adult with sickle cell disease across the United States. ASCQ-Me pain impact T-scores less than 50 are lower/worse than the reference mean (more severe pain impact), while pain impact T-scores greater than 50 are above/better than the reference mean (less severe pain impact)
次要结局
- Spinal Deformity Index(Baseline)