Peripheral-Blood Stem Cells versus Bone Marrow from Unrelated Donors N Engl J Med 2012;367: R3 Sunhee park/Prof. Kyung-sam cho

BACKGROUND Allogeneic hematopoietic stem-cell transplantation Unrelated donors : generic diversity  results obtained with transplants from HLA-identical siblings may not be applicable BMFilgrastim-stimulated peripheral blood Source : Transplantation between HLA-identical sibling -better engraftment & survival, relapse rate↓ -higher risk of acute/chronic GVHD Aim To determine the effects of graft source for unrelated-donor transplants A randomized trial comparing outcomes of peripheral-blood stem-cell and bone marrow transplantations.

METHODS Open-label, phase 3, multicenter, randomized trial Blood and Marrow Transplant Clinical Trials Network ~ donor- recipient pairs Randomization 1:1 Randomization 1:1 f/u for 3yrs Transplantation DNA typing Transplantation DNA typing BMSC donor PBSC donor GCSF PBSC donor GCSF Recipient Conditioning GVHD prophylaxis Recipient Conditioning GVHD prophylaxis

Inclusion criteria -<66 yrs -Acute leukemia, myelodysplasia, chronic myeloid or myelomonocytic leukemia, or myelofibrosis  transplantation Exclusion criteria -donor-specific anti-HLA ab, prior allogeneic or autologous transplantation, HIV infection, pregnancy or breast feeding, cardiac insufficiency or coronary artery dz, active infection -Cr, TB, AST/ALT ≥ 2x Primary end point : 2-year survival rate Secondary end point : Post-transplantation incidences of neutrophil & platelet engraftment, graft failure, acute & chronic GVHD, relapse, infection, adverse events Primary end point : 2-year survival rate Secondary end point : Post-transplantation incidences of neutrophil & platelet engraftment, graft failure, acute & chronic GVHD, relapse, infection, adverse events

RESULTS

2-year overall survival rate 51% 46% Fig 1. Survival after Randomization in the Intention-to-Treat Analysis.

Fig 2. Outcomes after Transplantation, According to Study Group. No significant difference

5 days 7 days

CONCLUSIONS  This trial did not detect significant survival differences between peripheral-blood stem-cell and bone marrow transplantation from unrelated donors.  Secondary end points indicated that peripheral-blood stem cells may reduce the risk of graft failure, whereas bone marrow may reduce the risk of chronic GVHD.