Allogeneic hematopoietic stem cell transplantation for neuromyelitis optica

Ann Neurol. 2014 Mar;75(3):447-53. doi: 10.1002/ana.24079. Epub 2014 Mar 7.


Neuromyelitis optica is a rare neurological autoimmune disorder characterized by a poor prognosis. Immunosuppression can halt disease progression, but some patients are refractory to multiple treatments, experiencing frequent relapses with accumulating disability. Here we report on durable clinical remissions after allogeneic hematopoietic stem cell transplantation in 2 patients suffering from severe forms of the disease. Immunological data evidenced disappearance of the pathogenic antibodies and regeneration of a naive immune system of donor origin. These findings correlated with evident clinical and radiological improvement in both patients, warranting extended clinical trials to investigate this promising therapeutic option.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aquaporin 4 / immunology
  • Autoantibodies / immunology
  • Bone Marrow / surgery
  • Female
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Lymphocyte Subsets / immunology
  • Male
  • Neuromyelitis Optica / immunology
  • Neuromyelitis Optica / therapy*
  • Transplantation, Homologous*


  • AQP4 protein, human
  • Aquaporin 4
  • Autoantibodies