Preventing disability in children with multiple sclerosis

Summary

Multiple sclerosis (MS) is an inflammatory disease of the brain and the spinal cord. It shows the highest inflammatory activity during its earliest stages, especially in children. Poorly controlled disease in children with active MS leads to permanent disability as early as their third decade of life.

Dr Sifat Sharmin and her team hypothesise that the risks of frequent attacks of MS and faster development of disability in this vulnerable population can be reduced with the appropriate choice of highly effective disease modifying therapies (DMTs). However, the lack of robust evidence in the use highly effective therapies in children with MS poses a challenge, impeding informed treatment decisions tailored to their unique needs.

To address this, Dr Sharmin’s study will use advanced statistical methods to analyse comprehensive data from three registries, both national and international. We will simulate the natural course of MS in children, progressing from negligible disability to secondary progressive MS, while evaluating the impact of DMTs on this trajectory.

Subsequently, the team will evaluate the justification for highly effective/high risk therapies in children and determine whether those at the highest risk of accelerated disability will receive greater benefits from such interventions. Furthermore, they will compare the effectiveness of different highly effective MS therapies in children.

This study will generate novel and conclusive evidence that will enable individualised treatment decision-making by identifying the optimal high-efficacy therapy at an early stage for children at the highest risk of experiencing worsening disability.