Treosulfan, cyclophosphamide and antithymocyte globulin for allogeneic hematopoietic cell transplantation in acquired severe aplastic anemia.
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.
Giebel
Sebastian
S
Dept. of Haematology and Bone Marrow Transplantation, Silesian Medical University, Katowice, Poland. sgiebel@poczta.onet.pl
Wojnar
Jerzy
J
Krawczyk-Kulis
Malgorzata
M
Markiewicz
Miroslaw
M
Wylezoł
Iwona
I
Seweryn
Marek
M
Holowiecka-Goral
Aleksandra
A
Holowiecki
Jerzy
J
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
D000961:Antilymphocyte Serum; D003520:Cyclophosphamide; C018404:treosulfan; D002066:Busulfan