title: Impact of Autologous Mesenchymal Stem Cell Infusion on Neuromyelitis Optica Spectrum Disorder: A Pilot, 2-Year Observational Study.
abstract: We evaluate safety and efficacy of autologous bone marrow-derived mesenchymal stem cells (MSCs) as a potential treatment for neuromyelitis optica spectrum disorder (NMOSD).
  
  
  
  Fifteen patients with NMOSD were recruited. All patients received a single intravenous infusion of 1.0 × 10(8) autologous MSC within 3-4 generations derived from bone marrow. The primary endpoints of the study were efficacy as reflected by reduction in annualized relapse rates (ARRs) and inflammatory lesions observed by MRI.
  
  
  
  At 12 months after MSC infusion, the mean ARR was reduced (1.1 vs. 0.3, P = 0.002), and the T2 or gadolinium-enhancing T1 lesions decreased in the optic nerve and spinal cord. Disability in these patients was reduced (EDSS, 4.3 vs. 4.9, P = 0.021; visual acuity, 0.4 vs. 0.5, P = 0.007). The patients had an increase in retinal nerve fiber layer thickness, optic nerve diameters and upper cervical cord area. We did not identify any serious MSC-related adverse events. At 24 months of MSC infusion, of 15 patients, 13 patients (87%) remained relapse-free, the mean ARR decreased to 0.1; the disability of 6 patients (40%) was improved, and the mean EDSS decreased to 4.0.
  
  
  
  This pilot trial demonstrates that MSC infusion is safe, reduces the relapse frequency, and mitigates neurological disability with neural structures in the optic nerve and spinal cord recover in patients with NMOSD. The beneficial effect of MSC infusion on NMOSD was maintained, at least to some degree, throughout a 2-year observational period.
authors.affiliation: Departments of Neurology, Radiology, Ophthalmology, Hematology, Tianjin Medical University General Hospital, Tianjin Medical University Eye Hospital, Tianjin, China.
authors.forename: Ying
authors.initials: Y
authors.lastname: Fu
authors.affiliation: Departments of Neurology, Radiology, Ophthalmology, Hematology, Tianjin Medical University General Hospital, Tianjin Medical University Eye Hospital, Tianjin, China.
authors.forename: Yaping
authors.initials: Y
authors.lastname: Yan
authors.affiliation: Departments of Neurology, Radiology, Ophthalmology, Hematology, Tianjin Medical University General Hospital, Tianjin Medical University Eye Hospital, Tianjin, China.
authors.forename: Yuan
authors.initials: Y
authors.lastname: Qi
authors.affiliation: Departments of Neurology, Radiology, Ophthalmology, Hematology, Tianjin Medical University General Hospital, Tianjin Medical University Eye Hospital, Tianjin, China.
authors.forename: Li
authors.initials: L
authors.lastname: Yang
authors.affiliation: Departments of Neurology, Radiology, Ophthalmology, Hematology, Tianjin Medical University General Hospital, Tianjin Medical University Eye Hospital, Tianjin, China.
authors.forename: Ting
authors.initials: T
authors.lastname: Li
authors.affiliation: Departments of Neurology, Radiology, Ophthalmology, Hematology, Tianjin Medical University General Hospital, Tianjin Medical University Eye Hospital, Tianjin, China.
authors.forename: Ningnannan
authors.initials: N
authors.lastname: Zhang
authors.affiliation: Departments of Neurology, Radiology, Ophthalmology, Hematology, Tianjin Medical University General Hospital, Tianjin Medical University Eye Hospital, Tianjin, China.
authors.forename: Chunshui
authors.initials: C
authors.lastname: Yu
authors.affiliation: Departments of Neurology, Radiology, Ophthalmology, Hematology, Tianjin Medical University General Hospital, Tianjin Medical University Eye Hospital, Tianjin, China.
authors.forename: Lei
authors.initials: L
authors.lastname: Su
authors.affiliation: Departments of Neurology, Radiology, Ophthalmology, Hematology, Tianjin Medical University General Hospital, Tianjin Medical University Eye Hospital, Tianjin, China.
authors.forename: Rui
authors.initials: R
authors.lastname: Zhang
authors.affiliation: Departments of Neurology, Radiology, Ophthalmology, Hematology, Tianjin Medical University General Hospital, Tianjin Medical University Eye Hospital, Tianjin, China.
authors.forename: Yi
authors.initials: Y
authors.lastname: Shen
authors.affiliation: Departments of Neurology, Radiology, Ophthalmology, Hematology, Tianjin Medical University General Hospital, Tianjin Medical University Eye Hospital, Tianjin, China.
authors.forename: Song
authors.initials: S
authors.lastname: Lin
authors.affiliation: Departments of Neurology, Radiology, Ophthalmology, Hematology, Tianjin Medical University General Hospital, Tianjin Medical University Eye Hospital, Tianjin, China.|Department of Neurology, Barrow Neurological Institute, St. Joseph's Hospital and Medical Center, Phoenix, AZ, USA.
authors.forename: Qiang
authors.initials: Q
authors.lastname: Liu
authors.affiliation: Departments of Neurology, Radiology, Ophthalmology, Hematology, Tianjin Medical University General Hospital, Tianjin Medical University Eye Hospital, Tianjin, China.
authors.forename: Zonghong
authors.initials: Z
authors.lastname: Shao
authors.affiliation: State Key Laboratory of Experimental Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China.
authors.forename: Zhongchao
authors.initials: Z
authors.lastname: Han
authors.affiliation: Departments of Neurology, Radiology, Ophthalmology, Hematology, Tianjin Medical University General Hospital, Tianjin Medical University Eye Hospital, Tianjin, China.|Department of Neurology, Barrow Neurological Institute, St. Joseph's Hospital and Medical Center, Phoenix, AZ, USA.
authors.forename: Fu-Dong
authors.initials: FD
authors.lastname: Shi
mesh_terms: D000328:Adult; D051401:Aquaporin 4 / Q000276:immunology; D001323:Autoantibodies / Q000097:blood; D001921:Brain / Q000000981:diagnostic imaging; D064987:Cell- and Tissue-Based Therapy / Q000379:methods*; D003071:Cognition / Q000502:physiology; D004185:Disability Evaluation; D005074:Evoked Potentials, Visual / Q000502:physiology; D005260:Female; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D059630:Mesenchymal Stem Cells / Q000502:physiology*; D008875:Middle Aged; D009471:Neuromyelitis Optica / Q000097:blood / Q000000981:diagnostic imaging / Q000628:therapy*; D010865:Pilot Projects; D012189:Retrospective Studies; D013116:Spinal Cord / Q000000981:diagnostic imaging; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: C496030:AQP4 protein, human; D051401:Aquaporin 4; D001323:Autoantibodies
keywords: Mesenchymal stem cells; Neuromyelitis optica spectrum disorder; Recovery; Relapse; Treatment