Long-term follow-up of pediatric MS patients starting treatment with injectable first-line agents: A multicentre,...

Area of Science:

• Neurology
• Pediatric Neurology
• Immunology

Background:

• Limited data exist on the very long-term outcomes of pediatric multiple sclerosis (MS) patients receiving disease-modifying treatments (DMTs).
• Understanding long-term efficacy and safety is crucial for managing this chronic condition in young patients.

Purpose of the Study:

• To evaluate the long-term follow-up of pediatric MS patients initially treated with injectable first-line agents.
• To assess treatment effectiveness, disability progression, and safety over an extended period.

Main Methods:

• A cohort of 97 pediatric MS patients was followed for over 12 years.
• Data collected included treatments, annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) scores, and adverse events.
• Multivariate analysis identified predictors of disease evolution, including age at treatment initiation.

Main Results:

• Initial treatment with interferons or copaxone significantly reduced ARR (from 3.2 to 0.7, p < 0.001), with sustained low ARR (0.3, p < 0.001) throughout follow-up.
• 89% of patients maintained an EDSS score below 4, though 40% experienced disability worsening.
• Early treatment initiation before age 12 was associated with a better disease course in multivariate analysis.

Conclusions:

• Injectable DMTs like interferons and copaxone are beneficial for pediatric MS patients, but many require escalation to more potent therapies.
• Initiating treatment before age 12 may lead to more favorable long-term outcomes in pediatric MS.
• Long-term monitoring is essential to manage treatment adjustments and potential adverse events, such as progressive multifocal leukoencephalopathy (PML).