Table I.

Hematopoietic stem cell transplant trials for patients with sickle cell disease without an HLA-matched related donor.

Ref	Donor	Conditioning Regimen GVHD prophylaxis	Patient Numbers (Age range in yrs.)	EFS/OS % at 1yr	Acute GVHD	Chronic GVHD
[31] CTN	8/8 MUD	Alemtuz + Flu + Mel CSA/Tacro + MTX + Methylpred	29 (5.9–19.3)	76/86	31%	62% 38% extensive
[34] CTN	5-6/6 UCB	Alemtuz + Flu + Mel CSA/Tacro + MMF	8 (7.4–16.2)	38/100	66%*	33%*
[35] Johns Hopkins	Haplo BM	rATG + Flu + CY + TBI 2Gy CY 50mg/kg Day3&4 + MMF + tacro/siro	14 (15–42)	50/100	0%	0%
[37] St. Jude	Haplo PBSC, Ex-vivo T cell depleted	1) rATG+Flu+Thio+Bu+OKT3 or 2)HU+Aza+Bu+Thio+CY+OKT3 MMF	8 (4–17)	50/88 38/75 at 3 years	40%* (Grade II only)	60%*
[39] NYMC multi- center	Haplo PBSC, Ex-vivo T cell depleted	HU+Aza+Flu+Bu+Thio+CY+TLI + rATG Tacro	13 Study still accruing	92/ N/A	N/A	N/A

Studies shown have event-free survival (events = graft rejection, death) data of at least 1 year

^*Values calculated as a percentage of engrafted patients at risk for GVHD

Alemtuz = alemtuzumab, BM = bone marrow, Bu = busulfan, CSA = cyclosporine, CTN = Clinical Trials Network, CY = cyclophosphamide, EFS = event-free survival, Flu = fludarabine, Haplo = haploidentical donor, HU = hydroxyurea, Mel = melphalan, Methylpred = methylprednisolone, MMF = mycophenolate mofetil, MTX = methotrexate, MUD = matched unrelated donor, N/A = not available, NYMC =New York Medical College, OS = overall survival, PBSC = peripheral blood stem cells, rATG = rabbit anti-thymocyte globulin, Siro = sirolimus, Tacro = tacrolimus, TBI = total body irradiation, TLI = total lymphoid irradiation, UCB =unrelated cord blood.