mycophenolic-acid and Opsoclonus-Myoclonus-Syndrome

Opsoclonus-myoclonus syndrome is characterized by abnormal lymphocyte trafficking into brain. The authors hypothesized that mycophenolate mofetil, a lymphocyte proliferation inhibitor, might be therapeutic. The cerebrospinal fluid and blood immunophenotypes of 15 children with predominantly chronic-relapsing opsoclonus-myoclonus syndrome were compared before and after treatment by flow cytometry. Mycophenolate mofetil reduced the cerebrospinal fluid expansion of HLA-DR+ activated T cells (-40%); the frequency of other T-cell or natural killer cell subsets remained unchanged, but cerebrospinal fluid B cells increased significantly. Adrenocorticotropic hormone dose was lowered by 64% over an average of 1.5 years, yet 73% eventually relapsed despite therapeutic drug levels. Prior treatment with rituximab prevented relapse-associated increase in cerebrospinal fluid B cells, without hindering mycophenolate mofetil-induced reduction in T-cell activation. These data demonstrate resistant immunologic problems in chronic-relapsing opsoclonus-myoclonus syndrome. Mycophenolate mofetil did not prevent relapse. The novel effect of mycophenolate mofetil on chronically activated T cells may contribute to its efficacy in T-cell mediated neurological disorders.

Topics: Adrenocorticotropic Hormone; Antibodies, Monoclonal; Antibodies, Monoclonal, Murine-Derived; B-Lymphocytes; Child, Preschool; Female; Flow Cytometry; HLA-DR Antigens; Humans; Immunologic Factors; Immunosuppressive Agents; Lymphocyte Activation; Male; Mycophenolic Acid; Opsoclonus-Myoclonus Syndrome; Phenotype; Pilot Projects; Recurrence; Rituximab; Severity of Illness Index; T-Lymphocytes