| Abstract: |
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. © The Author(s) 2012. |
| Keywords: |
child; controlled study; treatment outcome; child, preschool; fracture; retrospective studies; major clinical study; drug tolerability; drug efficacy; drug safety; drug withdrawal; hypertension; monotherapy; side effect; rituximab; controlled clinical trial; anemia; vomiting; dehydration; odor; drug administration schedule; cyclophosphamide; dexamethasone; immunoglobulin; fever; flushing; syncope; malaise; longitudinal studies; severity of illness index; immunotherapy; drug therapy, combination; infant; infant, newborn; neuroblastoma; drug response; acne; hypoglycemia; chi-square distribution; azathioprine; headache; immunologic factors; corticosteroid; motor activity; mycophenolic acid 2 morpholinoethyl ester; analysis of variance; irritability; immunosuppressive agents; leg pain; injections, intravenous; hormones; mercaptopurine; single blind procedure; weight gain; face disorder; single-blind method; swelling; septicemia; steroids; hirsutism; corticotropin; sweating; opsoclonus-myoclonus syndrome; opsoclonus myoclonus syndrome; antibodies, monoclonal, murine-derived; adrenocorticotropic hormone; stomach pain; dancing eyes; kinsbourne syndrome; paraneoplastic; acth; intravenous immunoglobulin; hunger; rotavirus infection
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