Twelve immunotherapy-naïve children with opsoclonus-myoclonus syndrome and CSF B cell expansion received rituximab, adrenocorticotropic hormone (ACTH), and IVIg. Motor severity lessened 73% by 6 mo and 81% at 1 yr (P < 0.0001). Opsoclonus and action myoclonus disappeared rapidly, whereas gait ataxia and some other motor components improved more slowly. ACTH dose was tapered by 87%. Reduction in total CSF B cells was profound at 6 mo (-93%). By study end, peripheral B cells returned to 53% of baseline and serum IgM levels to 63%. Overall clinical response trailed peripheral B cell and IgM depletion, but improvement continued after their levels recovered. All but one non-ambulatory subject became ambulatory without additional chemotherapy; two relapsed and remitted; four had rituximab-related or possibly related adverse events; and two had low-titer human anti-chimeric antibody. Combination of rituximab with conventional agents as initial therapy was effective and safe. A controlled trial with long-term safety monitoring is indicated.

To test the efficacy and safety of corticotropin-based immunotherapies in pediatric opsoclonus-myoclonus syndrome, 74 children received corticotropin alone or with intravenous immunoglobulin (groups 1 and 2, active controls); or both with rituximab (group 3) or cyclophosphamide (group 4); or with rituximab plus chemotherapy (group 5) or steroid sparers (group 6). There was 65% improvement in motor severity score across groups (P < .0001), but treatment combinations were more effective than corticotropin alone (P = .0009). Groups 3, 4, and 5 responded better than group 1; groups 3 and 5 responded better than group 2. The response frequency to corticotropin was higher than to prior corticosteroids (P < .0001). Fifty-five percent had adverse events (corticosteroid excess), more so with multiagents (P = .03); and 10% had serious adverse events. This study demonstrates greater efficacy of corticotropin-based multimodal therapy compared with conventional therapy, greater response to corticotropin than corticosteroid-based therapy, and overall tolerability.

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Aripiprazole is an atypical antipsychotic medication that is a partial dopamine D(2) and serotonin 5-hydroxytryptamine (1A) receptor agonist and 5-hydroxytryptamine (2A) receptor antagonist. It has a safer profile compared to other antipsychotic medications with regard to its effect on weight, glucose tolerance, prolactin level, and cardiac conduction. The common neurological adverse effects include headache, agitation, insomnia, sleepiness, and extrapyramidal symptoms. Seizures have not been reported in the pediatric population and only twice in adult patients. Here, we report a case of a healthy 3-year-old child who experienced prolonged lethargy, dystonia, and 2 witnessed seizures after incidental ingestion of 30 mg of aripiprazole. To our knowledge, this is the first reported case of aripiprazole-induced seizures in a child.

Seven HD gene positive individuals under the age of 21 years are described with clinical examination and proton-MR-spectroscopy ((1)H-MRS) profiles of the putamen. Despite clinical variability, the predominate (1)H-MRS abnormality is elevated glutamate, expressed well beyond the confines of the basal ganglia, and low striatal creatine.