The Biochemical and Radiological Evaluation of Bone Health in MPS
Research type
Research Study
Full title
The Biochemical and Radiological Evaluation of Bone Health in Patients with Mucopolysaccharidoses
IRAS ID
113030
Contact name
Reena Sharma
Contact email
Sponsor organisation
Salford Royal NHS Foundation Trust & The Pennine Acute Hospitals NHS Trust
Duration of Study in the UK
1 years, 11 months, 27 days
Research summary
Patients with Mucopolysaccharidoses (MPS) are characterized by a reduction in bone mineral density (BMD), deformities of bones and an increased risk of fractures. It is important to be able to take the reliable measurements of BMD in order to offer treatment and also to be able to assess the impact of interventions. DXA (dual energy X ray absorptiometry) is often used to assess BMD (bone mineral density) in MPS patients in clinical practice. There is no evidence to suggest that it is an accurate method to assess BMD in patients with MPS disorders. Volumetric Bone Mineral Density (vBMD) by Quantitative computed tomography (QCT) is the standard method to assess BMD, however it is not practical due its lack of availability (it is mainly used as a research tool), its cost being four times higher than DXA, and the increased exposure to radiation which can be up to ten times.
The overall aim of the study it to compare how close are the results by DXA in MPS patients in comparison to vBMD by QCT (reference method) and whether new biochemical approaches to assessment of BMD can offer clinical advantages for MPS patients. The information gathered from the study will help clinical practice in order to improve assessment of bone density in MPS patients who require serial scans for monitoring. Improved assessments will lead to better treatment and outcomes for patients.
We aim to recruit patients with MPS disorders who will have BMD assessment both by DXA and QCT. Additional information will be obtained from bone markers (blood and urine specimens) to gain a better understanding of disease progression and effect of treatment. Physical activity and balanced healthy diet are important variables that can affect BMD and will be assessed in the participating patients.REC name
Yorkshire & The Humber - Bradford Leeds Research Ethics Committee
REC reference
16/YH/0104
Date of REC Opinion
12 Jul 2016
REC opinion
Further Information Favourable Opinion