Table 1

Comparison of patient selection, treatment modality and outcomes among three randomised trials investigating HSCT in SSc.

ASSIST13ASTIS14SCOT15
Patient number1915675
Inclusion criteria<60 years of age18–65 years of age18–69 years of age
Diffuse SScDiffuse SScDiffuse SSc
mRSS≥15mRSS≥15mRSS≥16
Disease duration ≤4 yearsDisease duration ≤4 yearsDisease duration ≤4 years
Internal organ involvementInternal organ involvementInternal organ involvement
Exclusion criteriaMean PAP>25 mm Hg or PAPsys>40 mm HgMean PAP>50 mm HgMean PAP>30 mm Hg
LVEF<40%LVEF<45%LVEF<50%
FVC<45% predicted
DLCO<40% predicted
Creatinine >177 umol/LCreatinine clearance <40 mL/minCreatinine clearance <40 mL/min
Cyclophosphamide>6 intravenous coursesCyclophosphamide cumulative intravenous dose >5 g or >3 months oralCyclophosphamide cumulative intravenous dose >3 g/m2 or >4 months oral or >6 months intravenous
Active GAVE
MobilisationCyclophosphamide 2 g/m2, G-CSFCyclophosphamide 4 g/m2, G-CSFG-CSF only
ConditioningCyclophosphamide (200 mg/kg), rabbit ATGCyclophosphamide (200 mg/kg), rabbit ATGCyclophosphamide (120 mg/kg), equine ATG
Total body irradiationNoNoYes (800 cGy, lung and kidney shielding)
Stem cell manipulationNoneCD34+ selectionCD34+ selection
Comparator armCyclophosphamide 6 monthly intravenous courses (1000 mg /m2)Cyclophosphamide 12 monthly intravenous courses (750 mg/m2).Cyclophosphamide 12 monthly intravenous courses (750 mg/m2).
Primary outcome measure>25% decrease in mRSS, or >10% increase in FVC at 12 monthsSurvival without new onset heart, lung or kidney failureGlobal Rank Composite Score at month 54
Follow-up2.6 years (mean)5.8 years (median)Up to 4.5 years
12-month treatment-related mortality in comparator arm0 (0%)0 (0%)0 (0%)
12-month transplant-related mortality0 (0%)8 (10.1%)1 (3%)
  • ASSIST, American Scleroderma Stem cell versus Immune Suppression Trial; ASTIS, Autologous Stem cell Transplantation International Scleroderma Trial; ATG, antithymocyte globulin; FVC, forced vital capacity; GAVE, gastric antral vascular ectasia; G-CSF, granulocyte-colony stimulating factor; LVEF, left ventricular ejection fraction; mRSS, modified Rodnan skin score; PAP, pulmonary arterial pressure; SCOT, The Scleroderma Cyclophosphamide Or Transplantation; SSc, systemic sclerosis.