Stem Cell Transplantation for Stiff Person Syndrome (SPS)
Summary
Non-myeloablative regimens (as the investigators use herein) are designed to maximally suppress the immune system without destruction of the bone marrow stem cell compartment. When using a non-myeloablative regimen recovery occurs without infusion of stem cells and the stem cells are autologous. While not necessary for recovery, stem cell infusion may shorten the interval of neutropenia and attendant complications. Thus in reality there is no transplant only an autologous supportive blood product. Based on our encouraging results of non-myeloablative hematopoietic stem cell transplantation, for patients with multiple sclerosis and chronic inflammatory demyelinating polyneuropathy, the investigators will investigate the role of non-myeloablative hematopoietic stem cell transplantation for patients with SPS who require assistance to ambulate.
Timeline
- Start
- 2014-10
- Primary completion
- 2019-08-19
- Completion
- 2019-08-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | autologous hematopoietic stem cells | Cell therapy | — | Intravenous |
| Background | 2-MERCAPTOETHANESULFONIC ACID | Small molecule | — | Intravenous |
| Background | Cyclophosphamide | Other / unclassified | 200 mg/kg | Intravenous |
| Background | Cyclophosphamide | Other / unclassified | 2 g | Intravenous |
| Background | G-CSF | Protein / enzyme biologic | 5 ug/kg | Subcutaneous |
| Background | G-CSF | Protein / enzyme biologic | 10 ug/kg | Subcutaneous |
| Background | Methylprednisolone | Other / unclassified | 1000 mg | Intravenous |
| Background | Rituximab | Monoclonal antibody | 500 mg | Intravenous |
| Background | Thymoglobulin | Protein / enzyme biologic | 1.5 mg/kg | Intravenous |