title: Active comparator-controlled, rater-blinded study of corticotropin-based immunotherapies for opsoclonus-myoclonus syndrome.
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.
authors.affiliation: National Pediatric Myoclonus Center, and Departments of Neurology and Statistics and Research Consulting, Southern Illinois University School of Medicine, Springfield, IL 62794, USA. etate@siumed.edu
authors.forename: Elizabeth D
authors.initials: ED
authors.lastname: Tate
authors.forename: Michael R
authors.initials: MR
authors.lastname: Pranzatelli
authors.forename: Steven J
authors.initials: SJ
authors.lastname: Verhulst
authors.forename: Stephen J
authors.initials: SJ
authors.lastname: Markwell
authors.forename: David Neal
authors.initials: DN
authors.lastname: Franz
authors.forename: William D
authors.initials: WD
authors.lastname: Graf
authors.forename: S Anne
authors.initials: SA
authors.lastname: Joseph
authors.forename: Yasmin N
authors.initials: YN
authors.lastname: Khakoo
authors.forename: Warren D
authors.initials: WD
authors.lastname: Lo
authors.forename: Wendy G
authors.initials: WG
authors.lastname: Mitchell
authors.forename: Lalitha
authors.initials: L
authors.lastname: Sivaswamy
mesh_terms: D000324:Adrenocorticotropic Hormone / Q000627:therapeutic use*; D000704:Analysis of Variance; D058846:Antibodies, Monoclonal, Murine-Derived / Q000008:administration & dosage*; D016009:Chi-Square Distribution; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use; D003907:Dexamethasone / Q000627:therapeutic use; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D005260:Female; D006728:Hormones / Q000627:therapeutic use*; D006801:Humans; D007155:Immunologic Factors / Q000008:administration & dosage*; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D007231:Infant, Newborn; D007275:Injections, Intravenous; D008137:Longitudinal Studies; D008297:Male; D053578:Opsoclonus-Myoclonus Syndrome / Q000188:drug therapy* / Q000276:immunology; D012189:Retrospective Studies; D000069283:Rituximab; D012720:Severity of Illness Index; D016037:Single-Blind Method; D013256:Steroids / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D006728:Hormones; D007155:Immunologic Factors; D007166:Immunosuppressive Agents; D013256:Steroids; D000069283:Rituximab; D003907:Dexamethasone; D003520:Cyclophosphamide; D000324:Adrenocorticotropic Hormone