Active comparator-controlled, rater-blinded study of corticotropin-based immunotherapies for opsoclonus-myoclonus syndrome

Elizabeth D. Tate, Michael R. Pranzatelli, Steven J. Verhulst, Stephen J. Markwell, David Neal Franz, William D. Graf, S. Anne Joseph, Yasmin N. Khakoo, Warren D. Lo, Wendy G. Mitchell, Lalitha Sivaswamy

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47 Scopus citations

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.

Original languageEnglish
Pages (from-to)875-884
Number of pages10
JournalJournal of Child Neurology
Volume27
Issue number7
DOIs
StatePublished - Jul 2012

Keywords

  • ACTH
  • Adrenocorticotropic hormone
  • Corticosteroid
  • Corticotropin
  • Dancing eyes
  • Intravenous immunoglobulin
  • Kinsbourne syndrome
  • Neuroblastoma
  • Paraneoplastic
  • Rituximab

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