Sickle Cell Disease (SCD) Bone Pain Study

Not Applicable

Active, not recruiting

Conditions: Sickle Cell Disease
Sickle Cell Anemia
Osteoporosis
Low Bone Density
Vertebral Fracture
Osteopenia
Vertebral Compression
Osteonecrosis
Ischemic Necrosis
Avascular Necrosis

Registration Number: NCT05283148

Lead Sponsor: University of California, Davis

Brief Summary: A prospective study to determine how low bone mineral density and/or vertebral compression fractures associate with pain in adults with sickle cell disease

Detailed Description: 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.

Recruitment & Eligibility

Status: ACTIVE_NOT_RECRUITING

Sex: All

Target Recruitment: 53

Inclusion Criteria

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

Exclusion Criteria

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

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Lumbar Spine Bone Mineral Density	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)	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	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	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)	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	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	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)

Secondary Outcome Measures

Name	Time	Method
Spinal Deformity Index	Baseline	The spinal deformity index (SDI) is a semi-quantitative measure of number and severity of vertebral fractures observed on lateral spine X-rays of the thoracolumbar spine. To calculate the SDI, vertebrae are assigned a score as follows: 0 (no fracture), 1 (mild fracture), 2 (moderate fracture), and 3 (severe fracture). The total SDI is the summation of all T12-L4 vertebrae measured on the lateral spine X-rays. Minimum SDI =0 and Maximum SDI=15. Higher scores mean increased (worse) fracture burden, lower scores mean decreased (less) fracture burden.

Trial Locations

Locations (1): UC Davis Comprehensive Cancer Center
🇺🇸
Sacramento, California, United States
UC Davis Comprehensive Cancer Center
🇺🇸Sacramento, California, United States