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Bone marrow transplantation for severe aplastic anemia: a randomized controlled study of conditioning regimens. Blood 2007 May 15;109(10):4582-5 PMID: 17272503 PMCID: PMC1885491

Pubmed ID



The addition of antithymocyte globulin (ATG) to a regimen of high-dose cyclophosphamide has been advocated to enhance engraftment after allogeneic bone marrow transplantation (BMT) for severe aplastic anemia (SAA). In a prospective clinical trial, 134 patients were randomly assigned to receive cyclophosphamide alone or in combination with ATG. All patients received T-cell-replete bone marrow from an HLA-matched sibling. With a median follow-up of 6 years, the 5-year probabilities of survival were 74% for the cyclophosphamide alone group and 80% for the cyclophosphamide plus ATG group (P = .44). Graft failure and graft-versus-host disease (GVHD) rates were similar in both groups. With the survival rates achieved, this study is not adequately powered to detect significant differences between the 2 treatment groups. In conclusion, the results of allogeneic BMT for SAA have improved over time related to advances in supportive care. The addition of ATG to the preparative regimen did not significantly improve the outcome.

Author List

Champlin RE, Perez WS, Passweg JR, Klein JP, Camitta BM, Gluckman E, Bredeson CN, Eapen M, Horowitz MM


Bruce M. Camitta MD Clinical Professor in the Medicine department at Medical College of Wisconsin
Mary Eapen MBBS, DCh, MRCPI, MS Professor in the Medicine department at Medical College of Wisconsin
Mary M. Horowitz MD, MS Center Director, Professor in the Medicine department at Medical College of Wisconsin


2-s2.0-34248376573   114 Citations

MESH terms used to index this publication - Major topics in bold

Anemia, Aplastic
Antilymphocyte Serum
Bone Marrow Transplantation
Child, Preschool
Graft Rejection
Graft vs Host Disease
Immunosuppressive Agents
Middle Aged
Survival Analysis
Transplantation Conditioning
Transplantation, Homologous
jenkins-FCD Prod-332 f92a19b0ec5e8e1eff783fac390ec127e367c2b5