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. Keywords: Cognitive dysfunction; Digital tools; Global Patient-Reported Outcomes for Multiple Sclerosis (PROMS) Initiative; Patient-reported outcome measures (PROMs); Pediatric multiple sclerosis; Responsible research and innovation.

The Use of Patient-Reported Outcome Measures in Developmental Age: A Complementary Tool for Pediatric Multiple Sclerosis Prognosis / Maria Pia Amato. - In: NEUROLOGY AND THERAPY. - ISSN 2193-8253. - ELETTRONICO. - (2026), pp. 0-0.

The Use of Patient-Reported Outcome Measures in Developmental Age: A Complementary Tool for Pediatric Multiple Sclerosis Prognosis

Maria Pia Amato
2026

Abstract

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. Keywords: Cognitive dysfunction; Digital tools; Global Patient-Reported Outcomes for Multiple Sclerosis (PROMS) Initiative; Patient-reported outcome measures (PROMs); Pediatric multiple sclerosis; Responsible research and innovation.
2026
0
0
Maria Pia Amato
File in questo prodotto:
File Dimensione Formato  
40120_2026_Article_902.pdf

Accesso chiuso

Tipologia: Pdf editoriale (Version of record)
Licenza: Tutti i diritti riservati
Dimensione 646.9 kB
Formato Adobe PDF
646.9 kB Adobe PDF   Richiedi una copia all'autore

I documenti in FLORE sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificatore per citare o creare un link a questa risorsa: https://hdl.handle.net/2158/1480329
Citazioni
  • ???jsp.display-item.citation.pmc??? ND
  • Scopus ND
  • ???jsp.display-item.citation.isi??? ND
social impact