Secondary Osteoporosis & Its Therapy in Duchenne Muscular Dystrophy
Secondary Osteoporosis & Its Therapy in Duchenne Muscular Dystrophy – ScOT-DMD
Syed Faisal Ahmed
Duration of Study in the UK
2 years, 11 months, 28 days
There is increasing evidence that boys with Duchenne Muscular Dystrophy (DMD) have a greater fracture risk. Up to 40% may sustain a fracture. Fractures not only cause pain but may lead to premature loss of walking ability in this group.
Until now, the natural history of osteoporosis and development of bone fragility in DMD boys have not been studied throughly. The risk of fractures may be influenced by multiple factors including inflammation, physical inactivity, delay in puberty, deficiency of growth factors, vitamin D insufficiency and steroid therapy. The relative importance of these factors on development of osteoporosis remains unclear in this group. The evidence base for managing secondary osteoporosis therefore, remains scant and more evidence is required to improve bone health.
To understand the current practice and quantify the extent of bone morbidity, I have been funded by the CSO to perform the following set of studies.
1. A survey of the Scottish Muscle Network and the North Star Database to understand current practice.
2. Over a period of 9 months, affected boys with DMD between the ages of 5 and 16 years will be recruited into a 2-year prospective study. The changes in bone health will be studied using combination of imaging techniques including dual energy x-ray absorptiometry (DXA), lateral vertebral morphometry, plain X-rays and bone biomarkers. In a subset of patients, high resolution magnetic resonance imaging (MRI) will be employed to look at bone microarchitecture and bone marrow adiposity.
3. The opinion of affected families and boys as well as the clinicians will be collected to assess the feasibility of a future interventional trial of bone protective therapy.
This application for ethics approval applies specifically to the prospective study outlined under '2' above.
West of Scotland REC 5
Date of REC Opinion
5 Oct 2015
Further Information Favourable Opinion