Bone Pain in Adults With Sickle Cell Disease

Sponsor

University of California, Davis (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT05283148

Collaborator

(none)

Enrollment

Anticipated Duration (Months)

Study Details

Study Description

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

Condition or Disease	Intervention/Treatment	Phase
Sickle Cell Disease Low Bone Density Osteonecrosis Avascular Necrosis Sickle Cell Anemia Ischemic Necrosis Vertebra Compression Fracture Vertebral Fracture Vertebral Compression Osteoporosis Osteopenia

Detailed Description

The investigators hypothesize that adults with sickle cell disease (SCD) and low bone density or vertebral compression fractures on a dual X-ray absorptiometry (DXA) scan-adjusted for sex, SCD genotype, vitamin D levels 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 June 2022- September 2023, and anticipate enrolling 2-3 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 Dec 2023. 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.

Study Design

Study Type:

Observational

Anticipated Enrollment :

50 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Association Between Low Bone Density, Vertebral Fractures, and Pain in Sickle Cell Disease

Anticipated Study Start Date :

Jun 1, 2022

Anticipated Primary Completion Date :

Sep 1, 2023

Anticipated Study Completion Date :

Nov 1, 2023

Outcome Measures

Primary Outcome Measures

Bone mineral density Z-scores of lumbar spine, hip, forearm, and whole body [0-2 weeks from enrollment]
Bone mineral density (g/cm2) and standard deviation from reference mean
Vertebral fracture analysis [0-2 weeks from enrollment]
Qualitative measure of thoracolumbar vertebral compression fractures
Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) pain scores [0-2 weeks from enrollment]
Patient-reported outcome measure of pain level in the past 7 days. The ASCQ-Me pain scale ranges from 0-100, with a standardized sickle cell disease population mean of 50 (standard deviation=10), where lower scores signify worse disease impact.

Secondary Outcome Measures

Serum CTX-1 [0-2 weeks from enrollment]
Measurement of C-terminal telopeptide of type I collagen (CTX) in serum
Serum P1NP [0-2 weeks from enrollment]
Measurement of Procollagen type I N-terminal propeptide (P1NP) in serum

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 80 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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