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Complete remission in severe aplastic anemia after high-dose
cyclophosphamide without bone marrow transplantation
RA Brodsky, LL Sensenbrenner and RJ Jones
Johns Hopkins Oncology Center, Johns Hopkins Medical Institutions,
Baltimore, MD 21287-8967, USA.
Severe aplastic anemia (SAA) can be successfully treated with allogeneic
bone marrow transplantation (BMT) or immunosuppressive therapy. However,
the majority of patients with SAA are not eligible for BMT because they
lack an HLA-identical sibling. Conventional immunosuppressive therapy also
has major limitations; many of its remissions are incomplete and relapse or
secondary clonal disease is common. Cyclophosphamide is a potent
immunosuppressive agent that is used in all BMT conditioning regimens for
patients with SAA. Preliminary evidence suggested that high-dose
cyclophosphamide, even without BMT, may be beneficial to patients with SAA.
Therefore, 10 patients with SAA and lacking an HLA-identical sibling were
treated with high-dose cyclophosphamide (45 mg/kg/d) for 4 consecutive days
with or without cyclosporine. A complete response (hemoglobin level, >
13 g/dL; absolute neutrophil count, > 1.5 x 10(9)/L, and platelet count
> 125 x 10(9)/L) was achieved in 7 of the 10 patients. One of the
complete responders died from the acquired immunodeficiency syndrome 44
months after treatment with high-dose cyclophosphamide. The 6 remaining
patients are alive and in continuous complete remission, with a median
follow-up of 10.8 years (range, 7.3 to 17.8 years). The median time to last
platelet transfusion and time to 0.5 x 10(9) neutrophils/L were 85 and 95
days, respectively. None of the complete responders has relapsed or
developed a clonal disease. These results suggest that high-dose
cyclophosphamide, even without BMT, may be more effective than conventional
immunosuppressive therapy in restoring normal hematopoiesis and preventing
relapse or secondary clonal disorders. Hence, further studies confirming
the efficacy of this approach in SAA are indicated.
Volume 87,
Issue 2,
pp. 491-494,
01/15/1996
Copyright © 1996 by The American Society of Hematology
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