Abstract
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.
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Acknowledgements
We would like to thank the data managers and transplant physicians at participating centers for providing data: Dr MBC Koh, data manager S Kukkapalli, St George’s Hospital, London; Dr J Snowden, data managers B Holt and L Scott, Sheffield teaching Hospitals, Sheffield; Dr AJ Vora, data manger J Williams, Sheffield Children’s Hospital, Sheffield; Dr RT Wynn, data manager M Coussons, Royal Manchester Children’s Hospital, Manchester; Professor N Russell, data manager P Nelson, Nottingham University Hospital, Nottingham; Dr B Gibson, Yorkhill Children’s Hospital, data manager G Stewart, Glasgow; Dr M Gilleece, data managers S Hardaker, R Goodall, St James University Hospital, Leeds; Dr D Milligan, data manager W Clay, Heartlands Hospital, Birmingham; Dr P Veys, Great Ormond Street Hospital, London; Dr S Samarasinghe, Great North Children’s Hospital, Newcastle; Dr M McMullin, data manager S Piggott, Belfast City Hospital, Belfast; Prof G Jackson, data manager L McNally, Royal Victoria Infirmary, Newcastle; Dr K Orchard, data manager C Hurlock, Southampton General Hospital, Southampton; Dr A Hunter, data manager R Lewin, Leicester Royal Infirmary, Leicester; Prof C Craddock, data manager J Ward, Queen Elizabeth Hospital, Birmingham; Dr A O’Meara, data managers H Kerrigan and C Cawley, Our Lady’s Hospital for Sick Children, Dublin; Prof J Apperley, data manager F O’Boyle, Hammersmith Hospital, London; Dr K Wilson, data manager S Nicholas, University Hospital of Wales, Cardiff; Dr A Bloor, data manager T Dalton, Christie Hospital, Manchester; Dr P Johnson, data manager A Robertson, Western General Hospital, Edinburgh; Dr S Lawson, data manager J Rogers, Birmingham Children’s Hospital, Birmingham; Dr J dela Fuente, data manager F O’Boyle, St Mary’s Hospital, London; Dr K Thomson, University College Hospital, London.
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JCM held a consultancy with Genzyme from May 2008 to May 2009 and from June 2009 to July 2009. The other authors declare no competing financial interests.
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JCM, RMP, GC, ZL, GJM, AP contributed to the design of the study. JCM drafted the manuscript. KK and JP were responsible for coordination of data acquisition. JP, KK, MBCK, JAS, AJV, RTW, NR, BG, MG, DM, PV, SS, MM and GC contributed patient data to the study. RMP performed statistical analysis of the data. All authors were responsible for critical review and revision of the manuscript.
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Marsh, J., Pearce, R., Koh, M. et al. 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. Bone Marrow Transplant 49, 42–48 (2014). https://doi.org/10.1038/bmt.2013.115
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DOI: https://doi.org/10.1038/bmt.2013.115
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