National Pediatric Myoclonus Center, Southern Illinois University School of Medicine, Springfield, Illinois 62794-9643, USA.
Mov Disord. 2010 Jan 30;25(2):238-42. doi: 10.1002/mds.22941.
Twelve immunotherapy-naïve children with opsoclonus-myoclonus syndrome and CSF B cell expansion received rituximab, adrenocorticotropic hormone (ACTH), and IVIg. Motor severity lessened 73% by 6 mo and 81% at 1 yr (P < 0.0001). Opsoclonus and action myoclonus disappeared rapidly, whereas gait ataxia and some other motor components improved more slowly. ACTH dose was tapered by 87%. Reduction in total CSF B cells was profound at 6 mo (-93%). By study end, peripheral B cells returned to 53% of baseline and serum IgM levels to 63%. Overall clinical response trailed peripheral B cell and IgM depletion, but improvement continued after their levels recovered. All but one non-ambulatory subject became ambulatory without additional chemotherapy; two relapsed and remitted; four had rituximab-related or possibly related adverse events; and two had low-titer human anti-chimeric antibody. Combination of rituximab with conventional agents as initial therapy was effective and safe. A controlled trial with long-term safety monitoring is indicated.
十二例原发性免疫缺陷病相关性视神经脊髓炎谱系疾病患者接受了利妥昔单抗、促肾上腺皮质激素（ACTH）和 IVIg 治疗。6 个月时，81%的患者运动症状得到改善（P < 0.0001），12 个月时，73%的患者运动症状得到改善。眼震和动作性肌阵挛迅速消失，而步态共济失调和其他一些运动症状改善较为缓慢。ACTH 剂量减少了 87%。6 个月时，总 CSF B 细胞减少 93%。研究结束时，外周 B 细胞恢复至基线的 53%，血清 IgM 水平恢复至 63%。尽管外周 B 细胞和 IgM 耗竭的临床反应滞后，但在其水平恢复后，病情仍在继续改善。除 1 例不能行走的患者外，所有患者均无需进一步化疗即可行走；2 例患者复发后缓解；4 例患者出现利妥昔单抗相关或可能相关的不良事件；2 例患者出现低滴度人抗嵌合抗体。利妥昔单抗联合常规药物作为初始治疗是有效且安全的。需要进行长期安全性监测的对照试验。
