B cell depletion therapy for new-onset opsoclonus-myoclonus.

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.

• Authors: Michael R Pranzatelli, Elizabeth D Tate, Jennifer A Swan, Anna L Travelstead, Jerry A Colliver, Steven J Verhulst, Carl J Crosley, William D Graf, Suja A Joseph, Howard M Kelfer, G Praveen Raju
• Journal: Movement disorders : official journal of the Movement Disorder Society (Mov Disord) Vol. 25 Issue 2 Pg. 238-42 (Jan 30 2010) ISSN: 1531-8257 [Electronic] United States
• PMID: 20063398 (Publication Type: Clinical Trial, Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: (c) 2009 Movement Disorder Society.

Chemical References

• Antibodies, Monoclonal
• Antibodies, Monoclonal, Murine-Derived
• Immunoglobulin M
• Immunoglobulins
• Immunologic Factors
• Rituximab
• Adrenocorticotropic Hormone

Topics

• Adrenocorticotropic Hormone (adverse effects, therapeutic use)
• Analysis of Variance
• Antibodies, Monoclonal (adverse effects, therapeutic use)
• Antibodies, Monoclonal, Murine-Derived
• Antineoplastic Combined Chemotherapy Protocols (adverse effects, therapeutic use)
• Ataxia (drug therapy)
• B-Lymphocytes (drug effects, immunology, pathology)
• Child, Preschool
• Drug Administration Schedule
• Drug Therapy, Combination (methods)
• Female
• Humans
• Immunoglobulin M (blood)
• Immunoglobulins (adverse effects, therapeutic use)
• Immunologic Factors (adverse effects, therapeutic use)
• Infant
• Lymphocyte Depletion (methods)
• Male
• Myoclonus (drug therapy)
• Opsoclonus-Myoclonus Syndrome (physiopathology, therapy)
• Rituximab
• Treatment Outcome