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Early Predictors of Outcome in Pediatric Acquired Demyelinating Syndromes: A Retrospective Study Stratified by Final
Emanuela Claudia Turco1, Martina Gnazzo2, Sara Giordani1
1Child Neuropsychiatry Unit, Department of Medicine and Surgery, University of Parma, 43124 Parma, Italy.
Insights
Early immunotherapy and baseline findings predict outcomes in pediatric acquired demyelinating syndromes (ADSs). MOG antibody-associated disease (MOGAD) has a better prognosis than multiple sclerosis (MS) or neuromyelitis optica spectrum disorder (NMOSD).
Area of Science:
- Neurology
- Pediatrics
- Immunology
Background:
- Pediatric acquired demyelinating syndromes (ADSs) are diverse, including multiple sclerosis (MS), MOG antibody-associated disease (MOGAD), and neuromyelitis optica spectrum disorder (NMOSD).
- Accurate diagnosis and early outcome prediction are vital for managing pediatric ADSs, which have varied prognoses.
- Real-world data often involves initial diagnostic uncertainty, necessitating analysis of predictors based on final diagnoses.
Purpose of the Study:
- To identify early predictors of long-term outcomes in pediatric patients diagnosed with acquired demyelinating syndromes (ADSs).
- To compare the clinical trajectories and prognoses of different subtypes of pediatric ADSs, including MOGAD, MS, and NMOSD.
Main Methods:
- Retrospective analysis of 30 pediatric patients with ADSs, assessing clinical, radiological, CSF, antibody, and neurophysiological data.
- Evaluation of treatment strategies, including disease-modifying therapies (DMTs) and acute immunotherapy.
- Longitudinal assessment of outcomes over 3 years, including EDSS scores, MRI changes, and clinical status.
Main Results:
- MOGAD, MS, and NMOSD were the most common final diagnoses, with MOGAD showing a more favorable course.
- Early immunotherapy (90% of patients) and baseline factors like ≥3 brain lesions (76.7%) were prevalent.
- EDSS progression correlated with DMT use, MRI lesion type, CSF findings, antibody status, and evoked potentials; neurocognitive function predicted 3-year outcomes.
Conclusions:
- Early immunotherapy and baseline instrumental findings are critical predictors for pediatric ADSs outcomes.
- MS and NMOSD are associated with higher long-term disability compared to MOGAD.
- A prompt and comprehensive diagnostic approach is essential for improving the prognosis of pediatric ADSs.
Abstract:
Background/Objectives: 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. Methods: 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. Results: 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. Conclusions: 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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