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Neuropediatrics 2018; 49(02): 123-134
DOI: 10.1055/s-0037-1609038
Original Article
Georg Thieme Verlag KG Stuttgart · New York

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

Authors

  • Michael R. Pranzatelli

    1   National Pediatric Myoclonus Center and National Pediatric Neuroinflammation Organization, Inc., Orlando, Florida

  • Elizabeth D. Tate

    1   National Pediatric Myoclonus Center and National Pediatric Neuroinflammation Organization, Inc., Orlando, Florida

  • Michael Alber

    2   Department of Neuropaediatrics, Developmental Neurology, Social Paediatrics, University Children's Hospital Tübingen, Tübingen, Germany

  • Maha Awadalla

    3   Pediatric Neurology Unit, Dr. Erfan and Bagedo General Hospital, Jeddah, Kingdom of Saudi Arabia

  • Lubov Blumkin

    4   Pediatric Neurology Unit, Wolfson Medical Center, Holon, Sackler School of Medicine, Tel-Aviv University, Holon, Israel

  • Elena S. Lina

    5   Department of Psychoneurology No. 2, Federal State Medical Institution, Russian Children's Clinical Hospital, Ministry of Health Russian Federation, Moscow, Russia

  • Steffen Leiz

    6   Pediatric Neurology Unit, Department of Pediatrics and Adolescent Medicine, Hospital Dritter Orden, Munich, Germany

  • Judit Móser

    7   Department of Neurology, Heim Pal Children's Hospital, Budapest, Hungary

Funding This research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors.
Further Information

Publication History

15 February 2017

30 October 2017

Publication Date:
19 December 2017 (online)

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Abstract

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.

Keywords

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

Supplementary Material