An aggressive form of MOGAD treated with aHSCT: A case report

Elvira Sbragia1,2, Giacomo Boffa1, Riccardo Varaldo3

  • 1Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genoa, Genoa, Italy.

Multiple Sclerosis (Houndmills, Basingstoke, England)
|December 20, 2023
PubMed
Abstract

Insights

Autologous hematopoietic stem cell transplantation (aHSCT) offers a safe and effective treatment for aggressive myelin-oligodendrocyte-glycoprotein antibody-associated disease (MOGAD), suppressing disease activity for over two years in a refractory case.

Area of Science:

  • Neuroimmunology
  • Demyelinating diseases of the central nervous system
  • Autoimmune disorders

Background:

  • Myelin-oligodendrocyte-glycoprotein antibody-associated disease (MOGAD) can present with severe relapses and disability, challenging treatment approaches.
  • Current treatments for MOGAD are often adapted from neuromyelitis optica spectrum disorder (NMOSD) and rely on clinical experience.
  • Aggressive forms of MOGAD necessitate the exploration of novel and effective therapeutic strategies.

Observation:

  • A 56-year-old male diagnosed with MOGAD experienced recurrent optic neuritis followed by severe spinal cord inflammation with tumefactive lesions.
  • The patient exhibited persistent disease activity and relapses despite multiple treatments including high-dose steroids, rituximab, tocilizumab, IVIg, and plasma exchange.
  • Conventional therapies failed to control the aggressive, treatment-refractory MOGAD.

Findings:

  • Autologous hematopoietic stem cell transplantation (aHSCT) was performed following a myeloablative conditioning regimen.
  • The patient achieved sustained disease remission with no evidence of disease activity for over two years post-aHSCT.
  • aHSCT demonstrated a favorable safety profile in this patient with aggressive MOGAD.

Implications:

  • Autologous hematopoietic stem cell transplantation (aHSCT) represents a viable therapeutic option for severe, treatment-refractory MOGAD.
  • This case highlights the potential of aHSCT to induce long-term remission in aggressive demyelinating disorders.
  • Further research is warranted to establish the role of aHSCT in the management of MOGAD and other refractory autoimmune neurological conditions.