Improved tests for children with disabling muscle condition | Action Medical Research

Touching Lives - March 2011

Improved tests for children with disabling muscle condition

Juvenile dermatomyositis (JDM) can strike children at any age leaving them with pain, fatigue, rashes and muscle aches. Treatment, while intensive and prolonged, can help, but is not always effective. If untreated, this rare disease can make children so weak they are unable to do everyday activities such as running, walking or playing sports. The most severely affected can lose their lives.

Identifying JDM can be a complicated and lengthy process, involving varied assessments, such as blood tests, a muscle biopsy and MRI scans. Researchers had already developed a scoring tool to measure the abnormalities seen in biopsies and needed to investigate whether this method could predict how severe a child’s illness could become and their response to treatment.

With the help of funding from The Henry Smith Charity, the group began a two-year study. Led by Dr Lucy Wedderburn, who heads one of the leading JDM research centres in the world, the team also set out to find whether there is a link between the severity of disease and levels of a particular group of proteins in the blood. They brought together some international experts and studied 55 children with JDM.

First, their scoring tool proved reliable and effective; its versatility was also noted as different countries use either thigh or upper arm muscles for biopsies and it works for both. The researchers were excited to find that the amount of change or damage measured on the biopsy showed a direct link to how severe the disease is.

They also proved that myeloid related protein (MRP) is high in the blood of children with JDM, and the amount relates to the condition’s severity.

The researchers believe their tool can be used to help pathologists and paediatricians measure how badly affected the muscle is and select the right treatment. And their findings on MRP could eventually help doctors assess how the disease is responding to treatment.

This provides hope to children with this rare, disabling condition: earlier and more appropriate treatment may improve symptoms much quicker. It could reduce time spent in hospital and ease pain.

Your money in Action

At Action Medical Research, we are determined to stop the suffering of babies and children caused by disease and disability. With the help of many donors, we find and fund cutting-edge medical research across the UK most likely to deliver real benefit to babies, children and young people. In each issue of Touching Lives, we report on the outcome of projects funded – your money in Action.

“It is very difficult to get funding for studies of rare diseases,” says Dr Wedderburn, “yet this study should make a real and vital difference to families who have a child with JDM, and other patients as well. We were delighted to have been awarded this funding from Action Medical Research.”

Help us spread the word