Early Predictors of Outcome in Pediatric Acquired Demyelinating Syndromes: A Retrospective Study Stratified by Final Diagnosis

<b>Background/Objectives</b>: Pediatric acquired demyelinating syndromes (ADSs) encompass a heterogeneous group of disorders, including multiple sclerosis (MS), MOG antibody-associated disease (MOGAD), and neuromyelitis optica spectrum disorder (NMOSD), with distinct clinical trajectories and prognoses. While analyzed collectively at baseline to reflect real-world diagnostic uncertainty, outcome predictors were also examined according to final diagnosis. Identifying early predictors is crucial for optimizing long-term outcomes. <b>Methods</b>: We retrospectively analyzed 30 pediatric patients (mean onset age: 11.3 years) with ADSs. Clinical, radiological, CSF, antibody, and neurophysiological data were collected and analyzed alongside treatment strategies. Outcomes-EDSS scores, neuroradiological changes, and clinical status-were evaluated over a 3-year period. <b>Results</b>: Final diagnoses included MOGAD (36.6%), MS (33.3%), NMOSD (6.6%), ADEM (10%), and other ADSs (13.3%). At onset, ≥3 brain lesions were present in 76.7% of patients. Disease-modifying therapies (DMTs) were used in 37% and acute immunotherapy in 90%. EDSS progression was significantly associated with DMT use at multiple timepoints, with additional predictors including MRI lesion type, CSF findings, antibody status, and evoked potentials. At 3 years, neurocognitive function predicted clinical outcome. <b>Conclusions</b>: Early immunotherapy and baseline instrumental findings are key predictors of outcome in pediatric ADSs. MOGAD showed a more favorable course, while MS and NMOSD were associated with greater long-term disability. A comprehensive, early diagnostic approach is essential for improving prognosis.

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Journal
Children
Published
2025-07-24
DOI
https://doi.org/10.3390/children12080975
Primary Topic
Hereditary Neurological Disorders
Type
article
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article

Early Predictors of Outcome in Pediatric Acquired Demyelinating Syndromes: A Retrospective Study Stratified by Final Diagnosis

Susanna Esposito, Emanuela Claudia Turco, Silvia Giordani, Cosimo Neglia et al.
Children
Hereditary Neurological Disorders
article

Early Predictors of Outcome in Pediatric Acquired Demyelinating Syndromes: A Retrospective Study Stratified by Final Diagnosis

Susanna Esposito, Emanuela Claudia Turco, Silvia Giordani, Cosimo Neglia, Benedetta Piccolo, Valentina Baldini, Martina Gnazzo, Maria Carmela Pera, Giulia Pisanò, Elena Giroldini
article en

Abstract

<b>Background/Objectives</b>: Pediatric acquired demyelinating syndromes (ADSs) encompass a heterogeneous group of disorders, including multiple sclerosis (MS), MOG antibody-associated disease (MOGAD), and neuromyelitis optica spectrum disorder (NMOSD), with distinct clinical trajectories and prognoses. While analyzed collectively at baseline to reflect real-world diagnostic uncertainty, outcome predictors were also examined according to final diagnosis. Identifying early predictors is crucial for optimizing long-term outcomes. <b>Methods</b>: We retrospectively analyzed 30 pediatric patients (mean onset age: 11.3 years) with ADSs. Clinical, radiological, CSF, antibody, and neurophysiological data were collected and analyzed alongside treatment strategies. Outcomes-EDSS scores, neuroradiological changes, and clinical status-were evaluated over a 3-year period. <b>Results</b>: Final diagnoses included MOGAD (36.6%), MS (33.3%), NMOSD (6.6%), ADEM (10%), and other ADSs (13.3%). At onset, ≥3 brain lesions were present in 76.7% of patients. Disease-modifying therapies (DMTs) were used in 37% and acute immunotherapy in 90%. EDSS progression was significantly associated with DMT use at multiple timepoints, with additional predictors including MRI lesion type, CSF findings, antibody status, and evoked potentials. At 3 years, neurocognitive function predicted clinical outcome. <b>Conclusions</b>: Early immunotherapy and baseline instrumental findings are key predictors of outcome in pediatric ADSs. MOGAD showed a more favorable course, while MS and NMOSD were associated with greater long-term disability. A comprehensive, early diagnostic approach is essential for improving prognosis.

ChildrenVol. 12(8)
University of Modena and Reggio Emilia (IT), University of Parma (IT), University of Bologna (IT)
Openalex Percentile: Top 81%
Hereditary Neurological Disorders
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