Rituximab (anti-CD20) adjunctive therapy for Opsoclonus-Myoclonus syndrome

Michael R. Pranzatelli, Elizabeth D. Tate, Anna L. Travelstead, Jerry Barbosa, Robert A. Bergamini, Lucy Civitello, David N. Franz, Brian S. Greffe, Robin D. Hanson, Craig A. Hurwitz, Karen A. Kalinyak, Howard Kelfer, Yasmin Khakoo, John F. Mantovani, Stacy H. Nicholson, Joann M. Sanders, Stephen Wegner

Research output: Contribution to journalArticlepeer-review

115 Scopus citations


PURPOSE: To determine if rituximab, an anti-CD20 monoclonal antibody, reduces cerebrospinal fluid (CSF) B-cell expansion in opsoclonus-myoclonus syndrome (OMS) and results in clinical improvement. METHODS: Sixteen children with OMS and increased % CD20 B-cells in CSF received 4 rituximab infusions (375 mg/m IV) as add-on therapy to corticotropin (ACTH), intravenous immunoglobulins, or both, and were reevaluated 6 months later. Outcome measures were clinical (motor function, behavior, sleep) and immunologic (CSF and blood immunophenotype and Ig levels). Controls were 16 age-matched and sex-matched children, who did not have OMS. RESULTS: After rituximab, 81% of OMS had a lower motor severity score, and 44% improved one severity category. Mean total score decreased by 44% (P=0.0005). Rituximab reduced rage score, nighttime awakenings, and the number of children with opsoclonus, action myoclonus, drooling, gait ataxia, and rage. Despite a 51% reduction in ACTH dose, 9 of 11 children on ACTH did not relapse. The percentage of CSF CD19 (and CD20) B-cells was lowered in all children (undetectable in 6), with a 90% reduction in the group mean (P=0.00003). CSF B-cells were no longer expanded compared with controls. In blood, CD19 B-cells decreased (-90%, P=0.0003), as did the CSF:blood CD19 B-cell ratio (P=0.00003). Serum IgM fell by 69% (below reference range), with no statistically significant change in IgG or IgA. CONCLUSIONS: Rituximab seems efficacious and safe as adjunctive therapy for OMS. Selective targeting of CSF B lymphocytes represents a novel and valuable paradigm shift in the therapy for centrally mediated paraneoplastic disorders.

Original languageEnglish (US)
Pages (from-to)585-593
Number of pages9
JournalJournal of Pediatric Hematology/Oncology
Issue number9
StatePublished - Sep 2006
Externally publishedYes


  • B lymphocytes
  • B-cell trafficking
  • CSF immunophenotyping
  • Corticotropin
  • Dancing eyes
  • IVIg
  • Kinsbourne syndrome
  • Neuroblastoma
  • Paraneoplastic syndrome

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology


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