Comparison of patient selection, treatment modality and outcomes among three randomised trials investigating HSCT in SSc.
| ASSIST13 | ASTIS14 | SCOT15 | |
| Patient number | 19 | 156 | 75 |
| Inclusion criteria | <60 years of age | 18–65 years of age | 18–69 years of age |
| Diffuse SSc | Diffuse SSc | Diffuse SSc | |
| mRSS≥15 | mRSS≥15 | mRSS≥16 | |
| Disease duration ≤4 years | Disease duration ≤4 years | Disease duration ≤4 years | |
| Internal organ involvement | Internal organ involvement | Internal organ involvement | |
| Exclusion criteria | Mean PAP>25 mm Hg or PAPsys>40 mm Hg | Mean PAP>50 mm Hg | Mean PAP>30 mm Hg |
| LVEF<40% | LVEF<45% | LVEF<50% | |
| – | – | FVC<45% predicted DLCO<40% predicted | |
| Creatinine >177 umol/L | Creatinine clearance <40 mL/min | Creatinine clearance <40 mL/min | |
| Cyclophosphamide>6 intravenous courses | Cyclophosphamide cumulative intravenous dose >5 g or >3 months oral | Cyclophosphamide cumulative intravenous dose >3 g/m2 or >4 months oral or >6 months intravenous | |
| – | – | Active GAVE | |
| Mobilisation | Cyclophosphamide 2 g/m2, G-CSF | Cyclophosphamide 4 g/m2, G-CSF | G-CSF only |
| Conditioning | Cyclophosphamide (200 mg/kg), rabbit ATG | Cyclophosphamide (200 mg/kg), rabbit ATG | Cyclophosphamide (120 mg/kg), equine ATG |
| Total body irradiation | No | No | Yes (800 cGy, lung and kidney shielding) |
| Stem cell manipulation | None | CD34+ selection | CD34+ selection |
| Comparator arm | Cyclophosphamide 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 months | Survival without new onset heart, lung or kidney failure | Global Rank Composite Score at month 54 |
| Follow-up | 2.6 years (mean) | 5.8 years (median) | Up to 4.5 years |
| 12-month treatment-related mortality in comparator arm | 0 (0%) | 0 (0%) | 0 (0%) |
| 12-month transplant-related mortality | 0 (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.