Retrospective study of alemtuzumab vs ATG-based conditioning without irradiation for unrelated and matched sibling donor transplants in acquired severe aplastic anemia: a study from the British Society for Blood and Marrow Transplantation

TY - JOUR

T1 - Retrospective study of alemtuzumab vs ATG-based conditioning without irradiation for unrelated and matched sibling donor transplants in acquired severe aplastic anemia

T2 - a study from the British Society for Blood and Marrow Transplantation

AU - Marsh, J. C.

AU - Pearce, R. M.

AU - Koh, M. B. C.

AU - Lim, Z.

AU - Pagliuca, A.

AU - Mufti, G. J.

AU - Perry, J.

AU - Snowden, J. A.

AU - Vora, A. J.

AU - Wynn, R. T.

AU - Russell, N.

AU - Gibson, B.

AU - Gilleece, M.

AU - Milligan, D.

AU - Veys, P.

AU - Samarasinghe, S.

AU - McMullin, M.

AU - Kirkland, K.

AU - Cook, G.

AU - British Soc Blood Marrow

PY - 2014/1

Y1 - 2014/1

N2 - This 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.

AB - This 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.

KW - aplastic anemia

KW - alemtuzumab

KW - ATG

KW - SCT

KW - STEM-CELL TRANSPLANTATION

KW - TOTAL-BODY IRRADIATION

KW - SAA WORKING PARTY

KW - VERSUS-HOST-DISEASE

KW - TERM-FOLLOW-UP

KW - BONE-MARROW

KW - ALTERNATIVE DONOR

KW - IMMUNOSUPPRESSIVE THERAPY

KW - ANTITHYMOCYTE GLOBULIN

KW - PERIPHERAL-BLOOD

U2 - 10.1038/bmt.2013.115

DO - 10.1038/bmt.2013.115

M3 - Article

SN - 0268-3369

VL - 49

SP - 42

EP - 48

JO - Bone Marrow Transplantation

JF - Bone Marrow Transplantation

IS - 1

ER -