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Autologous hematopoietic stem cell transplantation (aHSCT) is the only treatment for refractory autoimmune diseases capable of inducing long-term, drug-free and asymptomatic remission. Over the past two decades, aHSCT has been used to treat inflammatory autoimmune disease of the CNS. Patients with relapsing-remitting multiple sclerosis benefit from aHSCT treatment. However, a certain percentage of patients still experience recurrence 3 or 5 years after transplantation. Therefore, exploration of conditioning regimens will drive therapeutic advances in aHSCT in autoimmune diseases of the CNS.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Hematopoietic Stem Cell Transplantation | Experimental | Patients will undergo stem cell transplantation for the treatment of refractory MS |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Autologous haemopoietic stem cell transplantation | Procedure | Immuno-ablation and autologous CD34 selected hematopoietic stem cell transplantation (HSCT). Stem cell mobilization with cyclophosphamide 2g/m2 and filgrastim 10 ug/kg/d x 5 day. Stem cell collection with cobe cpectra stem cell purification with Miltenyi CliniMACS Stem cell transplant conditioning with busulphan 3.2 mg/kg ; fludarabine 30mg/m2 or cladribine 10mg ï¼›cytarabine 1-2g/m2 or idarubicin 8mg/m2ï¼›cyclophosphamide 40mg/kg followed by CD34 selected autologous hematopoietic stem cell transplant. |
| Measure | Description | Time Frame |
|---|---|---|
| 3 year MS activity free survival | The events for the primary outcome are: clinical relapse, appearance of a new or Gd-enhancing lesion on MRI, or sustained progression of EDSS score. | 3 year follow-up post transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Time to MS treatment failure | Disease activity and disability will be assessed with clinical relapse, appearance of a new or Gd-enhancing lesion on MRI, or sustained progression of EDSS score and quality of life. | 3 years |
| Transplant related morbidity |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Qiang Liu, M.D.,Ph.D | Contact | +86 15022439149 | qliu@tmu.edu.cn |
| Name | Affiliation | Role |
|---|---|---|
| Qiang Liu, M.D.,Ph.D | Tianjin Medical University General Hospital | Principal Investigator |
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| ID | Term |
|---|---|
| D009103 | Multiple Sclerosis |
| ID | Term |
|---|---|
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D003711 | Demyelinating Diseases |
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|
Rate of transplant related events. |
| 3 years |
| Transplant related mortality | Rate of transplant related death. | 3 years |
| Immune reconstitution following transplant | Rate of immune reconstitution following transplant. | 3 years |
| Hematopoietic reconstitution following transplant | Rate of hematopoietic reconstitution following transplant. | 3 years |
| Imaging changes associated with the disease activity | Imaging changes include: new or enlarging T2-weighted lesion count and new T1-weighted lesion count at all scans after baseline; T2-weighted lesion volume; Gd-enhanced lesion count and volume; and total volume of non-enhancing T1-weighted lesions on all MRI scans. | 3 years |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |