title: Autologous stem cell transplantation for stiff person syndrome: two cases from the Ottawa blood and marrow transplant program.
abstract: OBJECTIVE
  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.
  
  
  METHODS
  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
  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.
authors.affiliation: Department of Medicine, University of Ottawa, Ottawa, Ontario, Canada.
authors.forename: Sheilagh
authors.initials: S
authors.lastname: Sanders
authors.affiliation: Ottawa Hospital Blood and Marrow Transplant Program, Ottawa, Ontario, Canada3Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
authors.forename: Christopher
authors.initials: C
authors.lastname: Bredeson
authors.affiliation: Department of Medicine, University of Ottawa, Ottawa, Ontario, Canada.
authors.forename: C Elizabeth
authors.initials: CE
authors.lastname: Pringle
authors.affiliation: Canadian Blood Services, Ottawa, Ontario, Canada.
authors.forename: Lisa
authors.initials: L
authors.lastname: Martin
authors.affiliation: Ottawa Hospital Blood and Marrow Transplant Program, Ottawa, Ontario, Canada3Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
authors.forename: David
authors.initials: D
authors.lastname: Allan
authors.affiliation: Ottawa Hospital Blood and Marrow Transplant Program, Ottawa, Ontario, Canada.
authors.forename: Isabelle
authors.initials: I
authors.lastname: Bence-Bruckler
authors.affiliation: Ottawa Hospital Blood and Marrow Transplant Program, Ottawa, Ontario, Canada.
authors.forename: Linda
authors.initials: L
authors.lastname: Hamelin
authors.affiliation: Ottawa Hospital Blood and Marrow Transplant Program, Ottawa, Ontario, Canada.
authors.forename: Harry S
authors.initials: HS
authors.lastname: Hopkins
authors.affiliation: Ottawa Hospital Blood and Marrow Transplant Program, Ottawa, Ontario, Canada.
authors.forename: Mitchell
authors.initials: M
authors.lastname: Sabloff
authors.affiliation: Ottawa Hospital Blood and Marrow Transplant Program, Ottawa, Ontario, Canada.
authors.forename: Dawn
authors.initials: D
authors.lastname: Sheppard
authors.affiliation: Ottawa Hospital Blood and Marrow Transplant Program, Ottawa, Ontario, Canada3Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
authors.forename: Jason
authors.initials: J
authors.lastname: Tay
authors.affiliation: Ottawa Hospital Blood and Marrow Transplant Program, Ottawa, Ontario, Canada.
authors.forename: Lothar
authors.initials: L
authors.lastname: Huebsch
authors.affiliation: Ottawa Hospital Blood and Marrow Transplant Program, Ottawa, Ontario, Canada3Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
authors.forename: Harold L
authors.initials: HL
authors.lastname: Atkins
mesh_terms: D000328:Adult; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008875:Middle Aged; D012720:Severity of Illness Index; D016750:Stiff-Person Syndrome / Q000276:immunology / Q000628:therapy*; D014182:Transplantation, Autologous; D016896:Treatment Outcome