1. Active Comparator-Controlled, Rater-Blinded Study of Corticotropin-Based Immunotherapies for Opsoclonus-Myoclonus Syndrome
- Author
-
Yasmin Khakoo, Lalitha Sivaswamy, Wendy G. Mitchell, David Neal Franz, Michael R. Pranzatelli, Elizabeth D. Tate, Stephen Markwell, William D. Graf, Steven Verhulst, S. Anne Joseph, and Warren D. Lo
- Subjects
Male ,medicine.medical_specialty ,Cyclophosphamide ,medicine.drug_class ,medicine.medical_treatment ,Severity of Illness Index ,Gastroenterology ,Dexamethasone ,Drug Administration Schedule ,Antibodies, Monoclonal, Murine-Derived ,Adrenocorticotropic Hormone ,Internal medicine ,Severity of illness ,Opsoclonus myoclonus syndrome ,Humans ,Immunologic Factors ,Medicine ,Single-Blind Method ,Longitudinal Studies ,Child ,Adverse effect ,Retrospective Studies ,Analysis of Variance ,Chemotherapy ,Chi-Square Distribution ,Opsoclonus-Myoclonus Syndrome ,business.industry ,Infant, Newborn ,Infant ,medicine.disease ,Hormones ,Treatment Outcome ,Endocrinology ,Tolerability ,Child, Preschool ,Injections, Intravenous ,Pediatrics, Perinatology and Child Health ,Corticosteroid ,Drug Therapy, Combination ,Female ,Steroids ,Rituximab ,Neurology (clinical) ,business ,Immunosuppressive Agents ,hormones, hormone substitutes, and hormone antagonists ,medicine.drug - 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.
- Published
- 2012
- Full Text
- View/download PDF