Neurology AdvisorHigh-Efficacy Therapies Reduce Disability Worsening in Pediatric-Onset MS

Early High-Efficacy Therapy Slows Disability Progression in Pediatric-Onset MS

A new study published in The Lancet Child and Adolescent Health reveals that early use of high-efficacy disease-modifying therapies (DMTs) significantly reduces the risk of disability worsening in patients with pediatric-onset relapsing-remitting multiple sclerosis (RRMS). The research, which analyzed data from international MS registries, provides compelling evidence for the benefits of early intervention with high-efficacy therapies, particularly in patients with minimal or mild disability at treatment initiation.

Key Points:

• The study included 5,224 participants with pediatric-onset MS, contributing to 91,613 visits.
• 87.5% of patients received DMTs, with a median follow-up of 5.05 years.
• High-efficacy therapies (HETs) showed the greatest effect in patients with early disease and minimal disability.
• Patients with minimal disability receiving HETs were 59% less likely to progress to mild disability compared to untreated patients (HR 0.41, 95% CI 0.31-0.53, P <.0001).
• Low-efficacy therapies also reduced disability progression risk, but to a lesser extent (HR 0.65, 95% CI 0.54-0.77, P <.0001).
• Patients on low-efficacy therapy had approximately twice the risk of disability worsening compared to those on HETs.
• Longer MS duration was associated with higher risk of disability worsening and lower chance of improvement.
• Male sex and older age were predictive of transition to the next disability state (HRs 1.34 and 1.04, respectively).
• The most common disability transition (32.8%) was from minimal to mild.
• The median time from first symptom to diagnosis was 2.03 years.
• Limitations included potential inaccuracies of the Expanded Disability Status Scale (EDSS) in detecting disability and lack of data on clinical characteristics like BMI and comorbidities.

Pediatric-onset MS is rarer than you might think. A recent meta-analysis pegged the global annual incidence at less than 1 per 100,000 kids. But here’s the kicker: prevalence varies wildly, from less than 1 per 100,000 in Japan to a surprising 27 per 100,000 in Sardinia, Italy. The MS Atlas estimates there are at least 30,000 children worldwide living with MS – and that’s just from 47 reporting countries! (CNS Drugs)

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