Source:Pediatric Neurology
Author(s): Michael R. Pranzatelli, Elizabeth D. Tate
BackgroundAlthough pulse-dose dexamethasone is increasingly favored for treating pediatric opsoclonus-myoclonus syndrome (OMS), and multimodal immunotherapy is associated with improved clinical response, there have been no neuroimmunological studies of dexamethasone-based multimodal disease-modifying therapy.MethodsIn this observational retrospective study, 19 children with OMS (with or without associated neuroblastoma) underwent multi-biomarker evaluation for neuroinflammation. Nine of varying OMS severity, duration, and treatment status were treated empirically with pulse dexamethasone, IVIg, and rituximab combination immunotherapy (DEXIR-CI). Another 10 children on dexamethasone alone or with IVIg at initial evaluation only provided a comparison group. Motor severity (Total Score) was scored blinded from videotapes using the validated OMS Evaluation Scale.ResultsDEXIR-CI was associated with a 69% reduction in group Total Score (P = 0.004), and was well-tolerated clinically. Patients given the dexamethasone combination exhibited significantly lowered B cell frequencies in CSF (-94%) and blood (-76%), normalizing the CSF B cell percentage. The number of patients with positive inflammatory markers dropped 87% (P = 0.002), as did the number of markers. CSF OCB were positive in 4/9 pre- but 0/6 post-treatment. In the comparison group, partial response to dexamethasone alone or with IVIg was associated with multiple positive markers for neuroinflammation despite an average of 7 months of treatment.ConclusionsMulti-mechanistic dexamethasone-based combination immunotherapy increases the therapeutic armamentarium for OMS, providing a viable option for less severe cases. Partial response to dexamethasone with or without IVIg is indicative of ongoing neuroinflammation and should be treated promptly and accordingly.
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