Active Comparator-Controlled, Rater-Blinded Study of Corticotropin-Based Immunotherapies for Opsoclonus-Myoclonus Syndrome

作者：Tate Elizabeth D^*; Pranzatelli Michael R; Verhulst Steven J; Markwell Stephen J; Franz David Neal; Graf William D; Joseph S Anne; Khakoo Yasmin N; Lo Warren D; Mitchell Wendy G; Sivaswamy Lalitha

来源：Journal of Child Neurology, 2012, 27(7): 875-884.

DOI：10.1177/0883073811428816

• adrenocorticotropic hormone
• ACTH
• dancing eyes
• intravenous immunoglobulin
• corticosteroid
• Kinsbourne syndrome
• neuroblastoma
• paraneoplastic
• rituximab
• corticotropin

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 %26lt; .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 %26lt; .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.

• 出版日期2012-7