Allogeneic Marrow Grafts for Aplastic Anemia
Cyclophosphamide and Antithymocyte Globulin to Condition Patients
With Aplastic Anemia for Allogeneic Marrow Transplantations: The
Experience in Four Centers
Rainer Storb, Karl G.Blume, Margaret R.O'Donnell, Tom Chauncey,
Stephen J.Forman, H.Joachim Deeg, Wendy W.Hu, Frederick
R.Appelbaum, Kris Doney, Mary E.D.Flowers, Jean Sanders, Wendy
Leisenring
Biology of Blood and Marrow Transplantation 7:39-44 (2001)
© 2001 American Society for Blood and Marrow Transplantation
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.
Download a PDF version of the full article using the link below:
 7.1.Storb.pdf
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