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Pediatric multiple sclerosis: Improving outcome through high-efficacy therapies
Lama Saleh Aljomah1, E Ann Yeh1
1Department of Pediatrics (Neurology), SickKids Research Institute, Division of Neurosciences and Mental Health, Hospital for Sick Children, University of Toronto, Toronto, Canada.
Insights
Pediatric-onset multiple sclerosis (MS) presents unique challenges, including cognitive decline and aggressive disease activity. Early diagnosis and treatment are crucial for altering the POMS disease course.
Area of Science:
- Neurology
- Pediatrics
- Immunology
Background:
- Pediatric-onset multiple sclerosis (POMS) affects individuals under 18, presenting with worse cognitive function and higher disease activity than adult-onset MS.
- Prompt diagnosis and early intervention are critical to modify POMS progression.
Purpose of the Study:
- To review diagnostic criteria for pediatric multiple sclerosis.
- To discuss differential diagnoses for POMS.
- To examine current and emerging therapeutic strategies for POMS.
Main Methods:
- Literature review of diagnostic criteria for pediatric multiple sclerosis.
- Analysis of differential diagnoses relevant to POMS.
- Evaluation of existing clinical trials and real-world data on disease-modifying therapies (DMTs) for POMS.
- Brief overview of ongoing clinical trials and novel therapies for POMS.
Main Results:
- Few DMTs are FDA-approved for pediatric MS, necessitating off-label use supported by real-world evidence.
- Existing evidence supports the effectiveness and safety of certain DMTs in pediatric populations.
- Ongoing research and emerging therapies show promise for POMS treatment.
Conclusions:
- Early recognition and intervention in POMS are vital for improving long-term outcomes.
- Off-label use of DMTs, guided by real-world data, is currently essential for managing POMS.
- Continued research into pediatric-specific therapies is necessary to optimize POMS care.
Abstract:
Pediatric-onset multiple sclerosis (POMS) refers to multiple sclerosis occurring in individuals under 18 years of age. It is characterized by poor cognitive outcomes and a more inflammatory course, more frequent clinical relapses, and a greater number of MRI lesions compared to adult-onset MS (AOMS). Prompt recognition of multiple sclerosis in this population is essential, as early intervention with disease-modifying therapies may change the trajectory of disease progression. In this paper, we will review diagnostic criteria for pediatric multiple sclerosis, differential diagnosis, and current and emerging therapeutic approaches. While a number of DMTs are approved for adult MS, few are approved for pediatric use. Many of these DMTs are used off-label, with real-world evidence demonstrating their effectiveness and safety. The review evaluates existing evidence for the use of these therapies in pediatric populations, with an emphasis on both existing clinical trials and real-world data that supports their use. In addition, we will briefly highlight ongoing clinical trials and emerging therapies for POMS.
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