Developing a tool to estimate the chance of developing spinal fractures in Duchenne muscular dystrophy (DMD)

Some people with DMD are at high risk of developing weak bones. This is partly because of the condition itself and partly due to the steroid medicines that help slow muscle weakness but can also reduce bone strength over time. 

One of the most common problems of having weak bones is fractures (breaks) in the bones of the spine. These fractures can happen without causing pain, so they are often missed. Once a child has one spinal fracture, they are much more likely to have more fractures in the future, which can lead to back pain, changes in posture, reduced mobility and breathing problems. 

There are medicines that can help strengthen bones and reduce the risk of further fractures. However, these treatments can cause unpleasant side effects, especially when they are first given. This means doctors need a better way to identify which boys are most likely to develop fractures so that treatment can be offered to those who need it most, while avoiding unnecessary treatment for those at lower risk. 

There is currently no reliable way of predicting which boys with DMD will develop spinal fractures.

This project aims to develop a simple tool that helps doctors estimate a boy’s chance of developing a spinal fracture over the next two years. 

Information from hundreds of boys with DMD across the UK will be collected, including some details already gathered during routine clinic visits as part of their NorthStar assessment. This will include age, steroid treatment, mobility, bone scans and spine X-rays. By comparing boys who develop fractures with those who do not, factors will be identified that best predict future fracture risk. 

These will then be combined into an easy-to-use risk score that doctors can use in routine NHS clinics. The team will also work closely with families, young people with DMD and healthcare professionals to make sure the tool is practical, easy to understand and helps support decisions about when to start treatment to protect bone health. 

Broken bones can have a major impact on the health, independence and quality of life of people with DMD. Finding those at highest risk before a fracture happens could allow treatment to start earlier and prevent avoidable pain and disability. 

This research will help doctors make more informed decisions about when to offer bone-strengthening treatment, ensuring children most likely to benefit receive it while avoiding unnecessary treatment for others. Ultimately, the project aims to reduce fractures, improve quality of life and support better long-term care for people living with DMD.