Journal
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
Volume 7, Issue 1, Pages 39-44Publisher
CARDEN JENNINGS PUBL CO LTD
DOI: 10.1053/bbmt.2001.v7.pm11215697
Keywords
aplastic anemia; allogeneic marrow grafts; cyclophosphamide and antithymocyte globulin conditioning
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Funding
- NATIONAL CANCER INSTITUTE [P01CA030206, P01CA049605] Funding Source: NIH RePORTER
- NATIONAL HEART, LUNG, AND BLOOD INSTITUTE [P01HL036444] Funding Source: NIH RePORTER
- NCI NIH HHS [CA30206, CA49605] Funding Source: Medline
- NHLBI NIH HHS [HL36444] Funding Source: Medline
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This report summarizes the experience with a conditioning regimen of cyclophosphamide and antithymocyte globulin in patients with severe aplastic anemia given HLA-matched related marrow grafts at 4 transplantation centers. Enrolled were 94 consecutive patients, of whom 87 had received multiple transfusions and 38 had failed immunosuppressive therapy. Their ages ranged from 2 to 59 years. After transplantation, 89 patients received a methotrexate/cyclosporine regimen for graft-versus-host disease (GVHD) prevention. Cyclosporine with or without prednisone was given in 4 patients, and no immunosuppression was given in 1 patient. Ninety-six percent of patients had sustained grafts, whereas 4% rejected grafts between 2 and 7 months after transplantation. Of the 4 rejecting patients, 3 are alive with successful second engraftments. Acute grade II GVHD was seen in 21% of patients, grade III in 7%, and grade IV in 1% of patients. Chronic GVHD was seen in 32% of patients, most of whom responded completely to immunosuppressive therapy. With a median follow-up of 6.0 years (range, 0.5-11.6 years), the survival rate was 88%. No unusual long-term side effects have been seen with the regimen. We conclude that the cyclophosphamide/antithymocyte globulin regimen combined with methotrexate/cyclosporine after transplantation is well tolerated and effective in heavily pretreated patients with aplastic anemia.
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