Hematopoietic Stem Cell Transplantation in Neuromyelitis Optica-Spectrum Disorders (NMO-SD): State-of-the-Art and Future Perspectives

Int J Mol Sci. 2020 Jul 26;21(15):5304. doi: 10.3390/ijms21155304.


Neuromyelitis optica (NMO) and neuromyelitis optica spectrum disorders (NMOSD) are a group of autoimmune inflammatory disorders of the central nervous system (CNS). Understanding of the molecular basis of these diseases in the last decades has led to an important improvement in the treatment of this disease, in particular, to the use of immunotherapeutic approaches, such as monoclonal antibodies and Hematopoietic Stem Cell Transplantation (HSCT). The aim of this review is to summarize the pathogenesis, biological basis and new treatment options of these disorders, with a particular focus on HSCT applications. Different HSCT strategies are being explored in NMOSD, both autologous and allogeneic HSCT, with the new emergence of therapeutic effects such as an induction of tolerance to auto-antigens and graft versus autoimmunity effects that can be exploited to hopefully treat a disease that still has prognosis.

Keywords: HSCT; NMO; NMOSD; monoclonal antibodies; multiple sclerosis.

Publication types

  • Review

MeSH terms

  • Allografts
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Neuromyelitis Optica / immunology
  • Neuromyelitis Optica / pathology
  • Neuromyelitis Optica / therapy*