Autologous stem cell transplantation for stiff person syndrome: two cases from the Ottawa blood and marrow transplant program
| Autor: | Sheilagh Sanders, Lothar Huebsch, Linda Hamelin, Isabelle Bence-Bruckler, Dawn Sheppard, C. Elizabeth Pringle, Christopher Bredeson, Lisa Martin, Jason Tay, Mitchell Sabloff, Harold L. Atkins, Harry S. Hopkins, David S. Allan |
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| Rok vydání: | 2014 |
| Předmět: |
Adult
medicine.medical_specialty medicine.medical_treatment Disease Hematopoietic stem cell transplantation Stiff-Person Syndrome Severity of Illness Index Transplantation Autologous Pathogenesis Autologous stem-cell transplantation Severity of illness medicine Humans business.industry Hematopoietic Stem Cell Transplantation Hematopoietic stem cell Middle Aged equipment and supplies medicine.disease Surgery Transplantation surgical procedures operative medicine.anatomical_structure Treatment Outcome Female Neurology (clinical) business Stiff person syndrome |
| Zdroj: | JAMA neurology. 71(10) |
| ISSN: | 2168-6157 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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