Table 3.

Randomised controlled trials of autologous HSCT in autoimmune diseases

DiseaseTrialNumber of patientsMobilisation regimenConditioning regimenComparator armResultsReference (number)
MSASTIMS21Cy 4 g/m2 + G-CSFBEAM + rATGMitoxantrone + methylprednisolone
monthly for 6 months
aHSCT significantly superior to mitoxantrone in reducing MRI activity in severe MS (79% reduction in T2 lesions). No difference in disease progression. No TRM12
Interim analysis
Cy 2 g/m2 + G-CSFCy 200 mg/kg + rATGStandard of DMT care (interferons, glatiramer acetate, dimethyl fumarate, fingolimod, natalizumab, but not alemtuzumab),aHSCT statistically superior to continued DMTs in terms of disability scores (EDSS) and progression in patients with RRMS with >2 relapses a year. No TRM13
SScASSIST19Cy 2g/m2 + G-CSFCy 200 mg/kg + rATGCy 1 g/m2 monthly for 6 monthsAll aHSCT (10/10) patients improved in skin score and lung function at 12 months compared to 0/9 in control. No TRM14
SScASTIS156Cy 4 g/m2 + G-CSFCy 200 mg/kg + rATGCy 750 mg/m2
monthly for 12 months
EFS and OS favoured aHSCT at 5.8 years. TRM 10%15
SScSCOT75G-CSFCy 200 mg/kg + hATG + TBI 800 cGy (with lung and kidney shielding)Cy 500 mg/m2 for 1 month, then 750 mg/m2 for 11 monthsSuperiority of aHSCT for GRCS (at 54 months), and EFS and OS (at 72 months). TRM 3% at 54 months and 6% at 72 months. Three malignancies in aHSCT group (possibly TBI related) compared with one in control group16
CDASTIC45Cy 4 g/m2 + G-CSFCy 200 mg/kg + rATGEarly versus late aHSCTFailed primary endpoint (sustained clinical remission off medication with no evidence of active disease on endoscopy or imaging). TRM in 1 patient18
CDASTIC secondary analysis38Cy 4 g/m2 + G-CSFCy 200 mg/kg + rATGOpen label cohort of all patients undergoing aHSCT in ASTIC trial3 months steroid-free remission at 1 year: 38%, 95% CI 22–55. Complete endoscopic healing: 50%, 95% CI 34–6619
  • ASSIST = American Scleroderma Stem Cell versus Immune Suppression Trial; ASTIC = Autologous Stem Cell Transplantation International Crohn's disease; ASTIMS = Autologous Stem Cell Transplantation International MS; ASTIS = Autologous Stem Cell Transplantation International Scleroderma; BEAM = BCNU/Etoposide/ARA-C/Melphalan; Cy = cyclophosphamide; EDSS = expanded disability status scale; EFS = event-free survival; G-CSF = granulocyte colony-stimulating factor; GRCS = global rank composite score; hATG = horse antithymocyte globulin; MIST = Multiple Sclerosis International Stem cell Transplant trial; OS = overall survival; rATG = rabbit antithymocyte globulin; SCOT = Scleroderma: Cyclophosphamide or Transplantation; TBI = total body irradiation; TRM = transplant-related mortality