title: 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.affiliation: National Pediatric Myoclonus Center, Southern Illinois University School of Medicine, Springfield, Illinois 62794-9643, USA. mpranzatelli@siumed.edu
authors.forename: Michael R
authors.initials: MR
authors.lastname: Pranzatelli
authors.forename: Elizabeth D
authors.initials: ED
authors.lastname: Tate
authors.forename: Jennifer A
authors.initials: JA
authors.lastname: Swan
authors.forename: Anna L
authors.initials: AL
authors.lastname: Travelstead
authors.forename: Jerry A
authors.initials: JA
authors.lastname: Colliver
authors.forename: Steven J
authors.initials: SJ
authors.lastname: Verhulst
authors.forename: Carl J
authors.initials: CJ
authors.lastname: Crosley
authors.forename: William D
authors.initials: WD
authors.lastname: Graf
authors.forename: Suja A
authors.initials: SA
authors.lastname: Joseph
authors.forename: Howard M
authors.initials: HM
authors.lastname: Kelfer
authors.forename: G Praveen
authors.initials: GP
authors.lastname: Raju
mesh_terms: D000324:Adrenocorticotropic Hormone / Q000009:adverse effects / Q000627:therapeutic use; D000704:Analysis of Variance; D000911:Antibodies, Monoclonal / Q000009:adverse effects / Q000627:therapeutic use*; D058846:Antibodies, Monoclonal, Murine-Derived; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D001259:Ataxia / Q000188:drug therapy; D001402:B-Lymphocytes / Q000187:drug effects* / Q000276:immunology / Q000473:pathology; D002675:Child, Preschool; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination / Q000379:methods; D005260:Female; D006801:Humans; D007075:Immunoglobulin M / Q000097:blood; D007136:Immunoglobulins / Q000009:adverse effects / Q000627:therapeutic use; D007155:Immunologic Factors / Q000009:adverse effects / Q000627:therapeutic use*; D007223:Infant; D008212:Lymphocyte Depletion / Q000379:methods; D008297:Male; D009207:Myoclonus / Q000188:drug therapy; D053578:Opsoclonus-Myoclonus Syndrome / Q000503:physiopathology / Q000628:therapy*; D000069283:Rituximab; D016896:Treatment Outcome
chemical_list: D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D007075:Immunoglobulin M; D007136:Immunoglobulins; D007155:Immunologic Factors; D000069283:Rituximab; D000324:Adrenocorticotropic Hormone