• J C Marsh, R M Pearce, M B C Koh, Z Lim, A Pagliuca, G J Mufti, J Perry, J A Snowden, A J Vora, R T Wynn, N Russell, B Gibson, M Gilleece, D Milligan, P Veys, S Samarasinghe, M McMullin, K Kirkland, G Cook, and British Society for Blood and Marrow Transplantation, Clinical Trials Committee.
• Department of Haematological Medicine, King's College Hospital and King's College London,, London, UK.
• Bone Marrow Transplant. 2014 Jan 1; 49 (1): 42-8.

AbstractThis retrospective national study compared the use of alemtuzumab-based conditioning regimens for hematopoietic SCT (HSCT) in acquired severe aplastic anemia with antithymocyte globulin (ATG)-based regimens. One hundred patients received alemtuzumab and 55 ATG-based regimens. A matched sibling donor (MSD) was used in 87 (56%), matched unrelated donor (MUD) in 60 (39%) and other related or mismatched unrelated donor (UD) in 8 (5%) patients. Engraftment failure occurred in 9% of the alemtuzumab group and 11% of the ATG group. Five-year OS was 90% for the alemtuzumab and 79% for the ATG groups, P=0.11. For UD HSCT, OS of patients was better when using alemtuzumab (88%) compared with ATG (57%), P=0.026, although smaller numbers of patients received ATG. Similar outcomes for MSD HSCT using alemtuzumab or ATG were seen (91% vs 85%, respectively, P=0.562). A lower risk of chronic GVHD (cGVHD) was observed in the alemtuzumab group (11% vs 26%, P=0.031). On multivariate analysis, use of BM as stem cell source was associated with better OS and EFS, and less acute and cGVHD; young age was associated with better EFS and lower risk of graft failure. This large study confirms successful avoidance of irradiation in the conditioning regimens for MUD HSCT patients.

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