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

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

37 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 (US)
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

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Clinical Neurology

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