Resolution of aquaporin-4 antibodies in a woman with neuromyelitis optica treated with human autologous stem cell transplant

Patrick Aouad; Jamma Li; Chris Arthur; Richard Burt; Suran Fernando; John Parratt

doi:10.1016/j.jocn.2015.02.007

Resolution of aquaporin-4 antibodies in a woman with neuromyelitis optica treated with human autologous stem cell transplant

J Clin Neurosci. 2015 Jul;22(7):1215-7. doi: 10.1016/j.jocn.2015.02.007. Epub 2015 Apr 22.

Authors

Patrick Aouad¹, Jamma Li², Chris Arthur³, Richard Burt⁴, Suran Fernando⁵, John Parratt⁶

Affiliations

¹ Department of Neurology, Royal North Shore Hospital, Reserve Road, St. Leonards, NSW 2065, Australia. Electronic address: patrick_aouad@msn.com.
² ImmunoRheumatology Laboratory, Pathology North, Sydney, NSW, Australia.
³ Department of Haematology, Royal North Shore Hospital, St. Leonards, NSW, Australia.
⁴ Division of Immunotherapy, Northwestern University, Chicago, IL, USA.
⁵ 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.
⁶ Department of Neurology, Royal North Shore Hospital, Reserve Road, St. Leonards, NSW 2065, Australia; Sydney Medical School Northern, University of Sydney, Sydney, NSW, Australia.

PMID: 25913278
DOI: 10.1016/j.jocn.2015.02.007

Abstract

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.

Keywords: Antibodies; Aquaporin-4; Autologous hematopoietic stem cell transplant; Neuromyelitis optica.

Publication types

Case Reports

MeSH terms

Aquaporin 4 / immunology*
Astrocytes / immunology
Astrocytes / metabolism
Autoantibodies / analysis*
Biomarkers
Disability Evaluation
Female
Hematopoietic Stem Cell Transplantation / methods*
Humans
Immunosuppressive Agents / therapeutic use
Middle Aged
Neuromyelitis Optica / immunology*
Neuromyelitis Optica / therapy*
Recurrence
Treatment Outcome

Substances

AQP4 protein, human
Aquaporin 4
Autoantibodies
Biomarkers
Immunosuppressive Agents