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Is a Benign Disease Course Possible in Untreated AQP4-IgG NMOSD?

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Some patients with aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (AQP4-IgG NMOSD) may experience a benign disease course without immunosuppression. This suggests potential for safe treatment de-escalation in select NMOSD cases.

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Area of Science:

  • Neurology
  • Immunology
  • Neuroimmunology

Background:

  • Aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (AQP4-IgG NMOSD) typically necessitates lifelong immunosuppression to prevent severe relapses.
  • Untreated or de-escalated therapy in AQP4-IgG NMOSD often leads to significant disability.

Purpose of the Study:

  • To investigate the occurrence and characteristics of a benign disease course in AQP4-IgG NMOSD patients not receiving immunosuppression.
  • To explore the possibility of safe treatment discontinuation or avoidance in AQP4-IgG NMOSD.

Main Methods:

  • A case series was conducted at a UK national NMOSD referral center.
  • Benign disease was defined as a disability status scale score of ≤3 after at least 4 years without immunotherapy.

Main Results:

  • Out of 153 AQP4-IgG NMOSD patients, 8 (5.2%) exhibited a benign disease course after a median follow-up of 7.5 years without immunotherapy.
  • All identified patients were female, with 7 of 8 being of White racial background.
  • Clinical manifestations included isolated optic neuritis, transverse myelitis, area postrema syndrome, or combinations thereof.

Conclusions:

  • The existence of benign AQP4-IgG NMOSD and the feasibility of safe therapy de-escalation remain uncertain.
  • This study indicates that both a benign disease course and potential for safe treatment discontinuation may be possible in AQP4-IgG NMOSD.
  • Further research into similar cases could identify biomarkers to guide safe treatment cessation.