Resolution of aquaporin-4 antibodies in a woman with neuromyelitis optica treated with human autologous stem cell transplant
Copyright policy )Resolution of aquaporin-4 antibodies in a woman with neuromyelitis optica treated with human autologous stem cell transplant
We report a 47-year-old woman with highly active neuromyelitis optica (NMO) and persistent high titre anti-aquaporin-4 antibodies (anti-AQP-4) who was resistant to multiple immune therapies until she underwent autologous hematopoietic stem cell transplant (auto-HSCT). NMO is the only demyelinating disease with a clinically useful serum biomarker, aquaporin-4, a water channel protein expressed on astrocytes. Anti-AQP-4 antibodies correlate with NMO disease activity and animal models strongly suggest the antibody is pathogenic. Auto-HSCT was associated with clinical and radiological remission, improved disability and resolution of AQP-4 antibodies which are still undetectable 12 months later. The utility of auto-HSCT for refractory NMO warrants further investigation, particularly with regards to pre-conditioning regimens and the utility of AQP-4 antibodies as a biomarker for immunological and clinical remission.
- Northwestern University United States
- University of Sydney Australia
- Royal North Shore Hospital Australia
Aquaporin 4, Neuromyelitis Optica, Hematopoietic Stem Cell Transplantation, Middle Aged, Disability Evaluation, Treatment Outcome, Recurrence, Astrocytes, Humans, Female, Biomarkers, Immunosuppressive Agents, Autoantibodies
Aquaporin 4, Neuromyelitis Optica, Hematopoietic Stem Cell Transplantation, Middle Aged, Disability Evaluation, Treatment Outcome, Recurrence, Astrocytes, Humans, Female, Biomarkers, Immunosuppressive Agents, Autoantibodies
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