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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
BASIC_SCIENCE
Masking
NONE
Enrollment
20
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 ;cytarabine 1-2g/m2 or idarubicin 8mg/m2;cyclophosphamide 40mg/kg followed by CD34 selected autologous hematopoietic stem cell transplant.
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.
Time frame: 3 year follow-up post transplant
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.
Time frame: 3 years
Transplant related morbidity
Rate of transplant related events.
Time frame: 3 years
Transplant related mortality
Rate of transplant related death.
Time frame: 3 years
Immune reconstitution following transplant
Rate of immune reconstitution following transplant.
Time frame: 3 years
Hematopoietic reconstitution following transplant
Rate of hematopoietic reconstitution following transplant.
Time frame: 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.
Time frame: 3 years
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