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Multiple sclerosis in children: an update on clinical diagnosis, therapeutic strategies, and research
Amy Waldman1, Angelo Ghezzi2, Amit Bar-Or3
1Division of Neurology, The Children's Hospital of Philadelphia, University of Pennsylvania, Philadelphia, PA, USA.
Insights
Recent advancements offer new insights into childhood-onset multiple sclerosis (MS). Research has refined diagnostic criteria, identified age-specific features, and detailed treatment options for pediatric MS patients.
Area of Science:
- Pediatric Neurology
- Neuroimmunology
- Clinical Neuroscience
Background:
- Childhood- and adolescent-onset multiple sclerosis (MS) has seen significant research progress in the last seven years.
- International collaborative efforts have led to revised diagnostic definitions and age-specific clinical feature identification for pediatric MS.
Purpose of the Study:
- To summarize recent advancements in understanding childhood- and adolescent-onset multiple sclerosis.
- To review updated diagnostic criteria, clinical features, neuroimaging, therapeutic options, and pathobiology of pediatric MS.
Main Methods:
- Review of clinical diagnostic definitions and age-specific features.
- Analysis of genetic and environmental risk factors through cohort studies.
- Characterization of MRI features and quantitative brain volume assessments.
- Identification of immunological markers using humoral and cell-based assays.
- Evaluation of safety and efficacy of first-line therapies in pediatric MS populations.
Main Results:
- Revised diagnostic criteria and age-dependent clinical presentations for pediatric MS have been established.
- Genetic and environmental factors' relative risks for pediatric MS are better understood.
- MRI findings are increasingly integrated into diagnostic criteria, with quantitative assessments of lesion burden and brain volume.
- Immunological studies reveal adult-like T-cell responses and antibodies against myelin and potassium-channel proteins in pediatric MS.
- Detailed understanding of first-line therapy safety and efficacy in pediatric MS populations has emerged.
Conclusions:
- Significant progress has been made in understanding pediatric multiple sclerosis, leading to improved diagnostics and treatment recommendations.
- Future research directions and trial designs for new therapies in pediatric MS are being proposed.
Abstract:
The clinical features, diagnostic challenges, neuroimaging appearance, therapeutic options, and pathobiological research progress in childhood-and adolescent-onset multiple sclerosis have been informed by many new insights in the past 7 years. National programmes in several countries, collaborative research efforts, and an established international paediatric multiple sclerosis study group have contributed to revised clinical diagnostic definitions, identified clinical features of multiple sclerosis that differ by age of onset, and made recommendations regarding the treatment of paediatric multiple sclerosis. The relative risks conveyed by genetic and environmental factors to paediatric multiple sclerosis have been the subject of several large cohort studies. MRI features have been characterised in terms of qualitative descriptions of lesion distribution and applicability of MRI aspects to multiple sclerosis diagnostic criteria, and quantitative studies have assessed total lesion burden and the effect of the disease on global and regional brain volume. Humoral-based and cell-based assays have identified antibodies against myelin, potassium-channel proteins, and T-cell profiles that support an adult-like T-cell repertoire and cellular reactivity against myelin in paediatric patients with multiple sclerosis. Finally, the safety and efficacy of standard first-line therapies in paediatric multiple sclerosis populations are now appreciated in more detail, and consensus views on the future conduct and feasibility of phase 3 trials for new drugs have been proposed.
