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

50 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

Funding

The authors disclosed receipt of the following financial support for the research, authorship and/or publication of this article: This work was supported by research grants to Dr Pranzatelli from Questcor Pharmaceuticals (Union City, CA), Genentech, Inc. (South San Francisco, CA)/Biogen IDEC (San Diego, CA), the Thrasher Research Fund (Salt Lake City, UT), the Chicago Institute of Neurosurgery and Neuroresearch Foundation (Chicago, IL), and the Spastic Paralysis Research Foundation (Illinois–Eastern Iowa District of Kiwanis International). No funding organization had any role in the conduct of the study, data analysis, the writing or content of the manuscript, or the decision to publish the manuscript.

Keywords

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

ASJC Scopus subject areas

  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health

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