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.
Department of Neurology, Royal North Shore Hospital, Reserve Road, St. Leonards, NSW 2065, Australia. Electronic address: patrick_aouad@msn.com.
Patrick
P
Aouad
ImmunoRheumatology Laboratory, Pathology North, Sydney, NSW, Australia.
Jamma
J
Li
Department of Haematology, Royal North Shore Hospital, St. Leonards, NSW, Australia.
Chris
C
Arthur
Division of Immunotherapy, Northwestern University, Chicago, IL, USA.
Richard
R
Burt
ImmunoRheumatology Laboratory, Pathology North, Sydney, NSW, Australia; Department of Clinical Immunology and Allergy, Royal North Shore Hospital, St. Leonards, NSW, Australia; Sydney Medical School Northern, University of Sydney, Sydney, NSW, Australia.
Suran
S
Fernando
Department of Neurology, Royal North Shore Hospital, Reserve Road, St. Leonards, NSW 2065, Australia; Sydney Medical School Northern, University of Sydney, Sydney, NSW, Australia.
John
J
Parratt
D051401:Aquaporin 4 / Q000276:immunology*; D001253:Astrocytes / Q000276:immunology / Q000378:metabolism; D001323:Autoantibodies / Q000032:analysis*; D015415:Biomarkers; D004185:Disability Evaluation; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008875:Middle Aged; D009471:Neuromyelitis Optica / Q000276:immunology* / Q000628:therapy*; D012008:Recurrence; D016896:Treatment Outcome
C496030:AQP4 protein, human; D051401:Aquaporin 4; D001323:Autoantibodies; D015415:Biomarkers; D007166:Immunosuppressive Agents
Antibodies; Aquaporin-4; Autologous hematopoietic stem cell transplant; Neuromyelitis optica