Treatment Updates in Paediatric-onset Multiple Sclerosis

Abstract

A number of new disease-modifying therapies have recently been developed and approved for use in adult-onset multiple sclerosis. However, few treatment options are approved for patients with paediatric-onset multiple sclerosis. There are an increasing number of clinical trials evaluating the efficacy and safety of disease-modifying therapies in children and teens living with multiple sclerosis. Clinical trials are difficult to complete in rare diseases like paediatric-onset multiple sclerosis; however, it is critical to assess safety and monitoring in this vulnerable population by applying robust research methodology to randomized controlled clinical trials. Longer-term extension analyses are also needed to better evaluate the efficacy, dosing and long-term safety of adult disease-modifying therapy for use in paediatric-onset multiple sclerosis. Future research should focus on defining optimal first-line disease-modifying therapy in paediatric-onset multiple sclerosis as related to both efficacy and safety, improving recruitment and completion rates of clinical trials, identifying relevant biomarkers of disease activity, analysing outcome measures related to treatment response and assessing long-term safety for this unique population living with a chronic disease.