Rituximab (anti-CD20) adjunctive therapy for opsoclonus-myoclonus syndrome

J Pediatr Hematol Oncol. 2006 Sep;28(9):585-93. doi: 10.1097/01.mph.0000212991.64435.f0.


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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antibodies, Monoclonal / therapeutic use*
  • Antibodies, Monoclonal, Murine-Derived
  • B-Lymphocytes / drug effects
  • Behavior / drug effects
  • Cerebrospinal Fluid / cytology
  • Cerebrospinal Fluid / drug effects
  • Cerebrospinal Fluid / immunology
  • Chemotherapy, Adjuvant
  • Female
  • Flow Cytometry
  • Humans
  • Immunologic Factors / therapeutic use*
  • Immunotherapy
  • Infant
  • Male
  • Motor Activity / drug effects
  • Neuroblastoma / complications
  • Neuroblastoma / drug therapy
  • Paraneoplastic Syndromes, Nervous System / cerebrospinal fluid*
  • Paraneoplastic Syndromes, Nervous System / drug therapy*
  • Rituximab
  • Sleep / drug effects


  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Murine-Derived
  • Immunologic Factors
  • Rituximab