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Pediatric multiple sclerosis: The 2022 ECTRIMS lecture
1Brenda BanwellChildren's Hospital of Philadelphia, Grace E. Loeb Chair in Neuroscience, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Insights
Prompt recognition and treatment of pediatric-onset multiple sclerosis (POMS) are crucial. Research suggests MS is a single disease across ages, offering unique insights from studying young patients.
Area of Science:
- Neurology
- Immunology
- Pediatrics
Background:
- Multiple sclerosis (MS) diagnosis in children and youth (pediatric-onset MS or POMS) is increasingly recognized.
- The inclusion of pediatric patients in diagnostic criteria has advanced POMS identification.
- Research indicates MS is a single disease entity across all age groups.
Purpose of the Study:
- To highlight the significance of early POMS recognition and treatment.
- To discuss the unique insights gained from studying POMS pathobiology.
- To outline the evolving landscape of pediatric MS clinical trials and research.
Main Methods:
- Review of current research on POMS epidemiology, genetics, and immunology.
- Discussion of emerging pediatric clinical trial designs and regulatory considerations.
- Emphasis on international collaborative efforts and POMS research priorities.
Main Results:
- MS pathobiology may be better understood through studying younger patients.
- Pediatric trials for MS therapies are developing, requiring specific designs and safety focus.
- International collaboration is essential due to the rarity of POMS.
Conclusions:
- Early diagnosis and treatment are vital for POMS.
- Further research and international collaboration are needed to advance POMS care and treatment options.
- Long-term safety and efficacy data are critical for pediatric MS therapies.
Abstract:
The 2022 ECTRIMS lecture focused on pediatric-onset multiple sclerosis (POMS), in recognition of the pivotal importance of prompt recognition and treatment of children and youth diagnosed with multiple sclerosis (MS), enabled over the past decade by the formal inclusion of pediatric patients in the McDonald diagnostic criteria. Epidemiologic, genetic and immunologic research has supported the concept that MS is a single disease across the age span and that clues to the inciting and early facets of MS pathobiology might be uniquely discerned through study of the youngest MS patients. Pediatric trials of pharmaceutical agents approved in adult-onset MS are emerging, although innovative study designs, alignment of regulatory agency requirements for trial design, family-centric models for study visits and emphasis on long-term safety and tolerability are essential. Evidence of safety and efficacy of key therapies is key if POMS patients are to be availed of the full armamentarium of MS therapeutic options. Finally, the rarity of POMS necessitates an international community effort to advance care and research. Such collaborations have been facilitated through the International Pediatric Multiple Sclerosis Group, Multiple Sclerosis International Federation, and by national multiple sclerosis societies. International efforts and priorities for the next decade will be highlighted.
