. Author manuscript; available in PMC: 2016 Aug 1.

Published in final edited form as: Rheum Dis Clin North Am. 2015 May 23;41(3):439–457. doi: 10.1016/j.rdc.2015.04.006

Table 3.

Comparison of 3 Randomized Trials of Autologous HSCT for Systemic Sclerosis

	ASTIS	SCOT	ASSIST
Study Design	Phase II, Non-Myeloablative, multicenter, event-free survival study	Phase II/III, Myeloablative, multicenter, event-free survival study	Phase II, Non-Myeloablative, singlecenter, treatment failure study
Inclusion Criteria	18–65 years old Disease duration ≤4 years, skin score ≥15, at least one predefined major organ involved or Disease duration ≤2 years, skin score ≥20, elevated acute phase reactants and/or proteinuria	18–69 years old Disease duration ≤ 5 years Diffuse cutaneous SSc, skin score ≥16 plus either pulmonary disease or prior renal crisis	18–60 years old Disease duration ≤ 4 years mRSS ≥15 and internal organ involvement or restricted skin involvement (mRSS ≤15) but coexistent pulmonary involvement
Exclusion Criteria	Predefined severe organ damage Prior cyclophosphamide total >5 g iv or >2 mg/kg po for > 3 months	Predefined severe organ damage Prior cyclophosphamide >6 months or >3 g/m²	Predefined severe organ damage Prior cyclophosphamide >6 month
Mobilizing Regimen	Cyclophosphamide and G-CSF	G-CSF	Cyclophosphamide and G-CSF
Conditioning Regimen	Cyclophosphamide 200 mg/kg Rabbit ATG	Cyclophosphamide 120 mg/kg Equine ATG TBI 800 cGy (with lung and renal shielding)	Cyclophosphamide 200 mg/kg Rabbit ATG
Graft Manipulation	CD34⁺ cell selection	CD34⁺ cell selection	No CD34⁺ cell selection
Primary End Point	Survival without organ failure at 3 years	Event-free survival without organ failure at 54 months	Improvement at 12 months defined as a decrease in mRSS (>25% for those with initial mRSS >14) or an increase in FVC > 10%
Control Arm	Cyclophosphamide 750 mg/m² iv monthly x 12; cross over to HSCT not allowed	Cyclophosphamide 750 mg/m² iv monthly x 12; cross over to HSCT not allowed	Cyclophosphamide 1000 mg/m² iv monthly x 6
Current Status	Completed 156 patients enrolled and randomized. Primary analysis reported in 2014 (van Laar et al, JAMA)	Completed enrollment 2011 Results pending follow-up	Completed 19 patients enrolled and randomized (10 HSCT, 9 cyclophosphamide of whom 7 crossed over to HSCT after cyclophosphamide failure). Primary analysis reported in 2011 (Burt et al, 2011). ASSIST IIb currently underway to test less intensive conditioning regimen (clinicaltrials.gov/ct2/show/NCT01445821)