Hematopoietic stem cell transplantation for auto immune rheumatic diseases

Table 1 Summary of European Group for Blood and Marrow Transplantation registry experience[6,7]

Disease	Number	Mean age at Tx (yr)	TRM (100 d)	5 yr progression free survival	5 yr overall survival	Death due to disease	Deaths due Tx
Systemic sclerosis	175	41	6%	55%	76%	23	12
SLE	85	28	11%	44%	76%	5	11
Rheumatoid arthritis	89	42	1%	18%	94%	0	2
JIA	65	11	11%	52%	82%	2	7

Tx: Transplant; TRM: Transplant related mortality; SLE: Systemic lupus erythematosus; JIA: Juvenile idiopathic arthritis.

Table 2 Randomized control trials of hematopoietic stem cell transplantation in systemic sclerosis

Trial name	Patients	Controls	Number	Outcome	TRM	Comments
ASTIS[14]	mRSS 15 for disease duration 4 yr, mRSS 20 if disease duration is 2 yr; and major organ involvement	IV CYC	156 (79 HSCT, 77 CYC)	5 yr survival: 52% (40 patients) in CYC; 70% (55 patients) in HSCT	10.01%	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 < 14	IV CYC	19 (10 HSCT, 9 CYC)	HSCT: all improved; CYC: 8 progressed	None	Small 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 yr	IV CYC	75	Not reported	-	Recruitment competed, yet to be published. Identical regimen to ASTIS except total body irradiation in HSCT

mRSS: Modified Rodnan skin score; IV: Intravenous; CYC: Cyclophosphamide; HSCT: Hematopoietic stem cell transplantation; HAQ: Health Assessment Questionnaire.