title: Efficacy of intravenous cyclophosphamide therapy for neuromyelitis optica spectrum disorder.
abstract: OBJECTIVE
  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.
  
  
  METHODS
  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.
  
  
  RESULTS
  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.
  
  
  CONCLUSIONS
  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.
authors: Department of Neurology, Hokkaido University Graduate School of Medicine, Japan. yaguchi@xa2.so-net.ne.jp
authors: Hiroaki
authors: H
authors: Yaguchi
authors: Ken
authors: K
authors: Sakushima
authors: Ikuko
authors: I
authors: Takahashi
authors: Hiroaki
authors: H
authors: Nishimura
authors: Moemi
authors: M
authors: Yashima-Yamada
authors: Masakazu
authors: M
authors: Nakamura
authors: Kazufumi
authors: K
authors: Tsuzaka
authors: Yasunori
authors: Y
authors: Maruo
authors: Toshiyuki
authors: T
authors: Takahashi
authors: Ichiro
authors: I
authors: Yabe
authors: Hidenao
authors: H
authors: Sasaki
mesh_terms: D000328:Adult; D051401:Aquaporin 4 / Q000276:immunology; D001323:Autoantibodies / Q000097:blood / Q000276:immunology; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage / Q000627:therapeutic use*; D004185:Disability Evaluation; D004341:Drug Evaluation; D005260:Female; D006801:Humans; D007262:Infusions, Intravenous; D008279:Magnetic Resonance Imaging; D008775:Methylprednisolone / Q000008:administration & dosage / Q000627:therapeutic use; D008875:Middle Aged; D009187:Myelitis / Q000188:drug therapy* / Q000276:immunology / Q000628:therapy; D009471:Neuromyelitis Optica / Q000188:drug therapy* / Q000276:immunology / Q000628:therapy; D010956:Plasmapheresis; D012189:Retrospective Studies; D012720:Severity of Illness Index; D013577:Syndrome; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: C496030:AQP4 protein, human; D051401:Aquaporin 4; D001323:Autoantibodies; D003520:Cyclophosphamide; D008775:Methylprednisolone