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Early High-Efficacy Therapy in Paediatric MS Reduces Risk of Reaching Key Disability Milestones

24 April 2024

  • MS Australia-supported researchers have revealed that early initiation of high-efficacy therapy in paediatric MS significantly reduces disability progression.
  • Treatment benefits are most pronounced in individuals with minimal or mild disability, emphasising the importance of early intervention.
  • Both high- and low-efficacy therapies demonstrated benefits in reducing the risk of disability worsening, underscoring the potential for improved outcomes with proactive treatment approaches.

The Power of Registries

The study, published online in The Lancet Child Adolescent Health, investigated the effectiveness of high-efficacy disease-modifying therapies (DMTs) on slowing disability progression in paediatric-onset multiple sclerosis (MS) by leveraging data from two MS registries.

Approximately 4–8% of people with MS have symptoms before the age of 18 years. Children are known to recover from relapses more effectively due to better remyelination capacities and stand to benefit significantly from high-efficacy treatments.

Registries are vital in advancing our understanding of MS across all age groups. They offer crucial insights into disease progression, treatment responses, and long-term outcomes.

What Did the Researchers Do?

MS Australia-funded researcher Dr Sifat Sharmin and her team analysed longitudinal data, tracking the same individuals over an extended period to observe changes, from the international MS registry, MSBase, and the Italian MS and Related Disorders Register, focusing on individuals under 18 with relapsing-remitting MS.

The study looked at 5,224 individuals with MS, out of which 70.6% (3,686) were female and 29.4% (1,538) were male. On average, these young people were about 15 years old when they first started showing signs of MS. The researchers tracked transitions across disability states using Expanded Disability Status Scale (EDSS) scores and assessed the impact of high-efficacy therapies (e.g., alemtuzumab, natalizumab) compared to low-efficacy therapies, and no treatment.

What Did the Researchers Find?

High-efficacy therapies significantly reduced the risk of disability worsening across all disability states, with the most substantial reduction observed in individuals with minimal disability.

Treatment benefits declined as disability increased. Even low-efficacy therapies showed benefits, particularly in reducing the risk of transitioning to mild disability in young people with minimal disability.

What does this mean for children with MS?

Early treatment with high-efficacy therapies in paediatric-onset relapsing-remitting MS substantially decreases the risk of reaching significant disability milestones. This effect is most pronounced when treatment begins in individuals with minimal or mild disability.

The findings suggest the importance of initiating high-efficacy therapy early to preserve neurological function in children with MS.

In Australia, there are currently only two DMTs approved for paediatric MS. This research is a significant step in broadening the range of treatment options for children with MS, aiming to support their journey towards living full and healthy lives.

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Early High-Efficacy Therapy in Paediatric MS Reduces Risk of Reaching Key Disability Milestones