小児多発性硬化症の予後予測における発達年齢の患者報告アウトカム評価の使用：補完的ツール

In pediatric multiple sclerosis (MS), assessment of disease burden predominantly relies on outcome measures developed and validated in adult populations. The application of these tools to children and adolescents may compromise sensitivity and specificity, potentially resulting in an underestimation of disease impact. Symptoms such as fatigue, mood disorders, and cognitive impairment may present subtly in younger patients and are therefore prone to underrecognition in routine clinical practice. Comprehensive evaluation of pediatric MS requires integration of the perspectives of both patients and their caregivers to fully capture the multidimensional effects of the disease, including psychosocial and functional consequences. Incorporation of patient-reported outcome measures (PROMs) into routine clinical care enables a more patient-centered assessment and supports informed clinical decision-making and personalized disease management. Furthermore, PROMs may provide insight into long-term health trajectories and help identify early predictors of adult outcomes. In recognition of the need for a coordinated global approach, the PROMS Initiative was established in 2019 to promote the development, validation, and implementation of PROMs in both research and clinical settings. Its objectives include facilitating the integration of PROMs into clinical trials, routine care, and regulatory frameworks across adult and pediatric populations. To optimize clinical utility, PROMs should demonstrate validity, reliability, and responsiveness, and their use should be standardized while remaining developmentally appropriate and tailored to age and disease stage. This article summarizes the current landscape of PROM use in pediatric MS and highlights key gaps to guide future research and clinical implementation.