Active Comparator-Controlled, Rater-Blinded Study of Corticotropin-Based Immunotherapies for Opsoclonus-Myoclonus Syndrome
- 28 February 2012
- journal article
- research article
- Published by SAGE Publications in Journal of Child Neurology
- Vol. 27 (7), 875-884
- https://doi.org/10.1177/0883073811428816
Abstract
To test the efficacy and safety of corticotropin-based immunotherapies in pediatric opsoclonus-myoclonus syndrome, 74 children received corticotropin alone or with intravenous immunoglobulin (groups 1 and 2, active controls); or both with rituximab (group 3) or cyclophosphamide (group 4); or with rituximab plus chemotherapy (group 5) or steroid sparers (group 6). There was 65% improvement in motor severity score across groups ( P < .0001), but treatment combinations were more effective than corticotropin alone ( P = .0009). Groups 3, 4, and 5 responded better than group 1; groups 3 and 5 responded better than group 2. The response frequency to corticotropin was higher than to prior corticosteroids ( P < .0001). Fifty-five percent had adverse events (corticosteroid excess), more so with multiagents ( P = .03); and 10% had serious adverse events. This study demonstrates greater efficacy of corticotropin-based multimodal therapy compared with conventional therapy, greater response to corticotropin than corticosteroid-based therapy, and overall tolerability.Keywords
This publication has 31 references indexed in Scilit:
- Insights on Chronic-Relapsing Opsoclonus-Myoclonus From a Pilot Study of Mycophenolate MofetilJournal of Child Neurology, 2009
- Rituximab (anti-CD20) Adjunctive Therapy for Opsoclonus-Myoclonus SyndromeJournal of Pediatric Hematology/Oncology, 2006
- Neuroepidemiologic Trends in 105 US Cases of Pediatric Opsoclonus-Myoclonus SyndromeJournal of Pediatric Oncology Nursing, 2005
- B- and T-cell markers in opsoclonus–myoclonus syndromeNeurology, 2004
- New aspects on the melanocortins and their receptorsPharmacological Research, 2000
- Monoaminergic effects of high-dose corticotropin in corticotropin-responsive pediatric opsoclonus-myoclonusMovement Disorders, 1998
- On the Molecular Mechanism of Adrenocorticotrophic Hormone in the CNS: Neurotransmitters and ReceptorsExperimental Neurology, 1994
- High‐dose iv human immunoglobulin in a case with infantile opsoclonus polymyoclonia syndromeActa Paediatrica, 1992
- Treatment of infantile spasms with high‐dose ACTHNeurology, 1989
- Myoclonic encephalopathy of infantsJournal of Neurology, Neurosurgery & Psychiatry, 1962