Abstract
Twelve immunotherapy-naïve children with opsoclonus-myoclonus syndrome and CSF B cell expansion received rituximab, adrenocorticotropic hormone (ACTH), and IVIg. Motor severity lessened 73% by 6 mo and 81% at 1 yr (P < 0.0001). Opsoclonus and action myoclonus disappeared rapidly, whereas gait ataxia and some other motor components improved more slowly. ACTH dose was tapered by 87%. Reduction in total CSF B cells was profound at 6 mo (-93%). By study end, peripheral B cells returned to 53% of baseline and serum IgM levels to 63%. Overall clinical response trailed peripheral B cell and IgM depletion, but improvement continued after their levels recovered. All but one non-ambulatory subject became ambulatory without additional chemotherapy; two relapsed and remitted; four had rituximab-related or possibly related adverse events; and two had low-titer human anti-chimeric antibody. Combination of rituximab with conventional agents as initial therapy was effective and safe. A controlled trial with long-term safety monitoring is indicated.
(c) 2009 Movement Disorder Society.
Publication types
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Clinical Trial
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Research Support, Non-U.S. Gov't
MeSH terms
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Adrenocorticotropic Hormone / adverse effects
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Adrenocorticotropic Hormone / therapeutic use
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Analysis of Variance
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Antibodies, Monoclonal / adverse effects
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Antibodies, Monoclonal / therapeutic use*
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Antibodies, Monoclonal, Murine-Derived
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Antineoplastic Combined Chemotherapy Protocols / adverse effects
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Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
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Ataxia / drug therapy
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B-Lymphocytes / drug effects*
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B-Lymphocytes / immunology
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B-Lymphocytes / pathology
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Child, Preschool
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Drug Administration Schedule
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Drug Therapy, Combination / methods
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Female
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Humans
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Immunoglobulin M / blood
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Immunoglobulins / adverse effects
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Immunoglobulins / therapeutic use
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Immunologic Factors / adverse effects
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Immunologic Factors / therapeutic use*
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Infant
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Lymphocyte Depletion* / methods
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Male
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Myoclonus / drug therapy
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Opsoclonus-Myoclonus Syndrome / physiopathology
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Opsoclonus-Myoclonus Syndrome / therapy*
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Rituximab
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Treatment Outcome
Substances
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Antibodies, Monoclonal
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Antibodies, Monoclonal, Murine-Derived
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Immunoglobulin M
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Immunoglobulins
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Immunologic Factors
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Rituximab
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Adrenocorticotropic Hormone