Rituximab, IVIg, and Tetracosactide (ACTH1-24) Combination Immunotherapy (RITE-CI) for Pediatric Opsoclonus-Myoclonus Syndrome: Immunomarkers and Clinical Observations

Michael R. Pranzatelli*, Elizabeth D. Tate, Michael Alber, Maha Awadalla, Lubov Blumkin, Elena S. Lina, Steffen Leiz, Judit Móser

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

7 Scopus citations


Opsoclonus-myoclonus syndrome (OMS) is a neuroinflammatory disorder with pervasive morbidity that warrants better treatments. Twelve children with moderate/severe OMS (total score 23 ± 6) who did not remit to multiple immunotherapies were evaluated for neuroinflammation in a case-control study using cerebrospinal fluid (CSF) lymphocyte subset analysis by flow cytometry, chemokine/cytokine analysis by enzyme-linked immunoadsorption assay (ELISA), and oligoclonal bands by immunofixation with isoelectric focusing. Observations made on empirical treatment with rituximab, IVIg, and tetracosactide combination immunotherapy (coined RITE-CI) were analyzed. All of the patients tested for multiple inflammatory markers were positive; 75% had ≥3 CSF markers. Fifty percent had CSF oligoclonal bands; 58%, B cell expansion; and 50 to 100%, elevated concentrations of multiple chemokines and neuronal/axonal marker neurofilament light chain. After RITE-CI, total score dropped significantly in the group (-85%, p < 0.0001) from moderate to trace, and by 2 to 4 severity categories in each patient. The 24-week schedule was well tolerated and clinically effective for moderate or severe OMS, as were other schedules. RITE-CI is feasible and effective as rescue therapy and presents an initial option for children with moderate/severe OMS. Though preliminary, the schedule can be adjusted to patient severity, propensity for relapse, and other factors.

Original languageEnglish
Pages (from-to)123-134
Number of pages12
Issue number2
StatePublished - 1 Apr 2018


FundersFunder number
Federal State Medical Institution
National Pediatric Neuroinflammation Organization, Inc.
Russian Children’s Clinical Hospital
Tel-Aviv University
School of Medicine
Wolfson College, University of Oxford
Ministry of Education and Science of the Russian Federation


    • chronic-relapsing OMS
    • cosyntropin
    • neuroblastoma
    • neuroimmunology
    • paraneoplastic syndrome
    • pediatric neuroinflammation


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