Bone marrow transplantation for severe aplastic anemia: a randomized controlled study of conditioning regimens.

Author(s): Champlin RE, Perez WS, Passweg JR, Klein JP, Camitta BM, Gluckman E, Bredeson CN, Eapen M, Horowitz MM

Affiliation(s): University of Texas M.D. Anderson Cancer Center, Houston, TX, United States.

Publication date & source: 2007-02-01, Blood., [Epub ahead of print]

Publication type:

The addition of antithymocyte globulin (ATG) to a regimen of high dose cyclophosphamide has been advocated to enhance engraftment after allogeneic bone marrow transplant (BMT) for severe aplastic anemia (SAA). In a prospective clinical trial, 134 patients were randomized 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=0.44). Graft failure and GVHD rates were similar in both groups. With the survival rates achieved, this study is not adequately powered to detect significant differences between the two 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.