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JAMA Neurol. 2014 Oct;71(10):1296-9. doi: 10.1001/jamaneurol.2014.1297.

Autologous stem cell transplantation for stiff person syndrome: two cases from the Ottawa blood and marrow transplant program.

Author information

1
Department of Medicine, University of Ottawa, Ottawa, Ontario, Canada.
2
Ottawa Hospital Blood and Marrow Transplant Program, Ottawa, Ontario, Canada3Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
3
Canadian Blood Services, Ottawa, Ontario, Canada.
4
Ottawa Hospital Blood and Marrow Transplant Program, Ottawa, Ontario, Canada.

Abstract

IMPORTANCE:

Stiff person syndrome (SPS) is a rare neurological disease causing significant functional disability for patients and presenting a therapeutic challenge for clinicians. Autologous hematopoietic stem cell transplantation (auto-HSCT) has been used successfully to remit autoimmune-mediated neurological diseases. We report 2 cases of severe SPS treated with auto-HSCT, a novel therapy for this disease.

OBSERVATIONS:

Two anti-glutamic acid decarboxylase antibody-positive patients with SPS had an autologous hematopoietic stem cell graft collected and stored. Subsequently, the patients underwent auto-HSCT. Both patients achieved clinical remission with sustained, marked improvement in symptoms and a return to premorbid functioning, now more than 2.5 and 4.5 years after the procedure.

CONCLUSIONS AND RELEVANCE:

Stiff person syndrome represents a novel indication for auto-HSCT. The resolution of clinical manifestations of SPS despite the persistence of anti-glutamic acid decarboxylase antibodies following auto-HSCT suggests that the antibody does not play a direct role in pathogenesis of SPS.

PMID:
25155372
DOI:
10.1001/jamaneurol.2014.1297
[Indexed for MEDLINE]

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