title: Treosulfan, cyclophosphamide and antithymocyte globulin for allogeneic hematopoietic cell transplantation in acquired severe aplastic anemia.
abstract: To reduce the risk of graft rejection after allogeneic hematopoietic cell transplantation (alloHCT) for patients with acquired severe aplastic anemia (SAA), we introduced an intensified preparative regimen consisting of treosulfan 10 g/m2/d on days -7, -6, cyclophosphamide 40 mg/kg/d on days -5, -4, -3, -2 and anti-thymocyte globulin 2 mg/kg/d on days -3, -2, -1. Six patients with the history of multiple transfusions were treated with alloHCT from either HLA-identical sibling (n=3) or an unrelated volunteer (n=3). Each, bone marrow and peripheral blood was used as a source of stem cells in three cases. All patients engrafted and achieved complete donor chimerism. None of the patients experienced severe organ toxicity. No severe acute graft-versus-host-disease (GVHD) was observed; two patients experienced extensive chronic GVHD. At the median follow-up of 14.5 (13-27) months all patients remained alive and disease-free. Our observation indicates that treosulfan + cyclophosphamide + antithymocyte globulin conditioning is well-tolerated and allows stable engraftment in acquired SAA.
authors.affiliation: Dept. of Haematology and Bone Marrow Transplantation, Silesian Medical University, Katowice, Poland. sgiebel@poczta.onet.pl
authors.forename: Sebastian
authors.initials: S
authors.lastname: Giebel
authors.forename: Jerzy
authors.initials: J
authors.lastname: Wojnar
authors.forename: Malgorzata
authors.initials: M
authors.lastname: Krawczyk-Kulis
authors.forename: Miroslaw
authors.initials: M
authors.lastname: Markiewicz
authors.forename: Iwona
authors.initials: I
authors.lastname: Wylezoł
authors.forename: Marek
authors.initials: M
authors.lastname: Seweryn
authors.forename: Aleksandra
authors.initials: A
authors.lastname: Holowiecka-Goral
authors.forename: Jerzy
authors.initials: J
authors.lastname: Holowiecki
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D002066:Busulfan / Q000031:analogs & derivatives* / Q000627:therapeutic use; D003520:Cyclophosphamide / Q000627:therapeutic use*; D018572:Disease-Free Survival; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation; D006650:Histocompatibility Testing; D006801:Humans; D012189:Retrospective Studies; D012720:Severity of Illness Index; D013997:Time Factors; D014184:Transplantation, Homologous
chemical_list: D000961:Antilymphocyte Serum; D003520:Cyclophosphamide; C018404:treosulfan; D002066:Busulfan