Yaguchi Hiroaki, Sakushima Ken, Takahashi Ikuko, Nishimura Hiroaki, Yashima-Yamada Moemi, Nakamura Masakazu, Tsuzaka Kazufumi, Maruo Yasunori, Takahashi Toshiyuki, Yabe Ichiro, Sasaki Hidenao
Department of Neurology, Hokkaido University Graduate School of Medicine, Japan.
Intern Med. 2013;52(9):969-72. doi: 10.2169/internalmedicine.52.7885. Epub 2012 Mar 1.
Neuromyelitis optica (NMO) is an inflammatory disease that affects the optic nerve and spinal cord. Optic neuritis and longitudinally extensive myelitis associated with systemic autoimmune disease have been recently defined as NMO spectrum disorder (NMOSD). In this study, we report the efficacy of intravenous cyclophosphamide (IVCY) therapy for NMOSD.
Four patients diagnosed with NMOSD were enrolled in this study. The expanded disability status scale (EDSS) score was used to evaluate the degree of severity. All of the patients received intravenous methylprednisolone (IVMP; 1 g/day for three days), and two patients also received plasmapheresis (PP). All of the patients were administered IVCY treatment.
Anti-AQP4 antibodies were present in the sera of all patients. All patients exhibited longitudinally extensive transverse myelitis (LETM). Only one patient who fulfilled the criteria for a diagnosis of NMO exhibited optic neuritis. Two patients developed relapse under treatment with low-dose prednisolone (PSL) before the administration of IVCY. The patients in this study exhibited a median improvement in the EDSS score following IVCY treatment from 8.0 to 5.75. Adverse effects were observed in only one patient.
This study, despite its retrospective design, demonstrated the therapeutic efficacy of IVCY for NMOSD in both the acute and chronic phases of the disease and determined the IVCY dosage for Japanese women with NMOSD. Additionally, this study provided evidence that for NMOSD patients with severe disabilities, IVCY added to IVMP and PP may be a useful therapeutic modality.
视神经脊髓炎（NMO）是一种影响视神经和脊髓的炎症性疾病。与系统性自身免疫性疾病相关的视神经炎和纵向广泛横贯性脊髓炎最近被定义为视神经脊髓炎谱系障碍（NMOSD）。在本研究中，我们报告了静脉注射环磷酰胺（IVCY）治疗NMOSD的疗效。
4例诊断为NMOSD的患者纳入本研究。采用扩展残疾状态量表（EDSS）评分评估严重程度。所有患者均接受静脉注射甲泼尼龙（IVMP；1 g/天，共3天），2例患者还接受了血浆置换（PP）。所有患者均接受IVCY治疗。
所有患者血清中均存在抗水通道蛋白4（AQP4）抗体。所有患者均表现为纵向广泛横贯性脊髓炎（LETM）。只有1例符合NMO诊断标准的患者出现视神经炎。2例患者在接受IVCY治疗前使用低剂量泼尼松龙（PSL）治疗时病情复发。本研究中的患者在接受IVCY治疗后，EDSS评分中位数从8.0改善至5.75。仅1例患者观察到不良反应。
本研究尽管为回顾性设计，但证明了IVCY在NMOSD疾病的急性期和慢性期均具有治疗效果，并确定了日本NMOSD女性患者的IVCY剂量。此外，本研究提供了证据表明，对于重度残疾的NMOSD患者，IVCY联合IVMP和PP可能是一种有效的治疗方式。
