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Copyright ©The Author(s) 2016.
World J Transplant. Mar 24, 2016; 6(1): 199-205
Published online Mar 24, 2016. doi: 10.5500/wjt.v6.i1.199
Table 1 Summary of European Group for Blood and Marrow Transplantation registry experience[6,7]
DiseaseNumberMean age at Tx (yr)TRM (100 d)5 yr progression free survival5 yr overall survivalDeath due to diseaseDeaths due Tx
Systemic sclerosis175416%55%76%2312
SLE852811%44%76%511
Rheumatoid arthritis89421%18%94%02
JIA651111%52%82%27
Table 2 Randomized control trials of hematopoietic stem cell transplantation in systemic sclerosis
Trial namePatientsControlsNumberOutcomeTRMComments
ASTIS[14]mRSS 15 for disease duration 4 yr, mRSS 20 if disease duration is 2 yr; and major organ involvementIV CYC156 (79 HSCT, 77 CYC)5 yr survival: 52% (40 patients) in CYC; 70% (55 patients) in HSCT10.01%At 2 yr: significantly better event free survival, mRSS, EuroQol. HAQ; decline in creatinine clearance and increase in FVC/VC
Median follow up 5.8 yr
ASSIST[15]mRSS 14 with internal organ involvement or coexistent pulmonary Involvement if mRSS was < 14IV CYC19 (10 HSCT, 9 CYC)HSCT: all improved; CYC: 8 progressedNoneSmall study, 7/8 that progressed in CYC group switched to HSCT. All HSCT patients (including switches) had significant improvement in mRSS and FVC and TLC
Follow up 2 yr
SCOT[16]mRSS > 16, significant visceral organ involvement, disease duration < 4 yrIV CYC75Not reported-Recruitment competed, yet to be published. Identical regimen to ASTIS except total body irradiation in HSCT