title: 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.
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.
authors.affiliation: Department of Haematological Medicine, King's College Hospital and King's College London,, London, UK.
authors.forename: J C
authors.initials: JC
authors.lastname: Marsh
authors.affiliation: BSBMT Data Registry, Guy's Hospital, London, UK.
authors.forename: R M
authors.initials: RM
authors.lastname: Pearce
authors.affiliation: Department of Haematology, St George's Hospital and Medical School, London, UK.
authors.forename: M B C
authors.initials: MB
authors.lastname: Koh
authors.affiliation: Department of Haematology-Oncology, National University Cancer Institute, National Hospital Singapore, Singapore.
authors.forename: Z
authors.initials: Z
authors.lastname: Lim
authors.affiliation: Department of Haematological Medicine, King's College Hospital and King's College London,, London, UK.
authors.forename: A
authors.initials: A
authors.lastname: Pagliuca
authors.affiliation: Department of Haematological Medicine, King's College Hospital and King's College London,, London, UK.
authors.forename: G J
authors.initials: GJ
authors.lastname: Mufti
authors.affiliation: BSBMT Data Registry, Guy's Hospital, London, UK.
authors.forename: J
authors.initials: J
authors.lastname: Perry
authors.affiliation: 1] Department of Haematology, Sheffield Teaching Hospitals, Sheffield, UK [2] Department of Oncology, University of Sheffield, Sheffield, UK.
authors.forename: J A
authors.initials: JA
authors.lastname: Snowden
authors.affiliation: Department of Haematology, Sheffield Children's Hospital, Sheffield, UK.
authors.forename: A J
authors.initials: AJ
authors.lastname: Vora
authors.affiliation: Department of Paediatric Blood and Marrow Transplant, Royal Manchester Children's Hospital, Manchester, UK.
authors.forename: R T
authors.initials: RT
authors.lastname: Wynn
authors.affiliation: Department of Haematology, Nottingham University Hospital, Nottingham, UK.
authors.forename: N
authors.initials: N
authors.lastname: Russell
authors.affiliation: Department of Haematology, Royal Hospital for Sick Children, Glasgow, Scotland, UK.
authors.forename: B
authors.initials: B
authors.lastname: Gibson
authors.affiliation: Department of Haematology, St James's Institute of Oncology, St James's University Hospital, Leeds, UK.
authors.forename: M
authors.initials: M
authors.lastname: Gilleece
authors.affiliation: Centre for Haematology and Transplantation, Heartlands Hospital, Birmingham, UK.
authors.forename: D
authors.initials: D
authors.lastname: Milligan
authors.affiliation: Department of Haematology, Great Ormond Hospital for Sick Children, London, UK.
authors.forename: P
authors.initials: P
authors.lastname: Veys
authors.affiliation: Department of Paediatric and Adolescent Haematology, Great North Children's Hospital, Newcastle-Upon-Tyne, UK.
authors.forename: S
authors.initials: S
authors.lastname: Samarasinghe
authors.affiliation: Centre for Cancer Research and Cell Biology, Queen's University, Belfast, UK.
authors.forename: M
authors.initials: M
authors.lastname: McMullin
authors.affiliation: BSBMT Data Registry, Guy's Hospital, London, UK.
authors.forename: K
authors.initials: K
authors.lastname: Kirkland
authors.affiliation: Department of Haematology, St James's Institute of Oncology, St James's University Hospital, Leeds, UK.
authors.forename: G
authors.initials: G
authors.lastname: Cook
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000074323:Alemtuzumab; D000741:Anemia, Aplastic / Q000628:therapy*; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D001854:Bone Marrow Cells / Q000166:cytology; D016026:Bone Marrow Transplantation / Q000379:methods*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D036102:Peripheral Blood Stem Cell Transplantation / Q000379:methods*; D012042:Registries; D012189:Retrospective Studies; D012307:Risk Factors; D035781:Siblings; D014019:Tissue Donors; D019172:Transplantation Conditioning / Q000379:methods*; D016896:Treatment Outcome; D006113:United Kingdom; D055815:Young Adult
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D000961:Antilymphocyte Serum; D000074323:Alemtuzumab
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