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
National Pediatric Myoclonus Center, and Departments of Neurology and Statistics and Research Consulting, Southern Illinois University School of Medicine, Springfield, IL 62794, USA.
J Child Neurol. 2012 Jul;27(7):875-84. doi: 10.1177/0883073811428816. Epub 2012 Feb 28.
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.
为了测试基于促肾上腺皮质激素的免疫疗法治疗儿童眼阵挛-肌阵挛综合征的疗效和安全性，74名儿童单独接受促肾上腺皮质激素治疗，或联合静脉注射免疫球蛋白（第1组和第2组，活性对照组）；或两者联合利妥昔单抗（第3组）或环磷酰胺（第4组）；或联合利妥昔单抗加化疗（第5组）或类固醇替代药物（第6组）。各组的运动严重程度评分改善率为65%（P <.0001），但联合治疗比单独使用促肾上腺皮质激素更有效（P =.0009）。第3、4和5组的反应比第1组更好；第3和5组的反应比第2组更好。对促肾上腺皮质激素的反应频率高于先前使用的皮质类固醇（P <.0001）。55%的患者出现不良事件（皮质类固醇过量），联合用药的情况更常见（P =.03）；10%的患者出现严重不良事件。这项研究表明，与传统疗法相比，基于促肾上腺皮质激素的多模式疗法疗效更佳，对促肾上腺皮质激素的反应优于基于皮质类固醇的疗法，且总体耐受性良好。
