What is a major limitation of hematopoietic cell transplantation (HCT) for treatment of aplastic anemia?

Updated: Jan 29, 2021
  • Author: Sameer Bakhshi, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

One of the major problems of HCT in aplastic anemia is the high rate of rejection (10%; range, 5-50%). The rejection rate correlates positively with the number of transfusions the patient received and the duration of his or her disease prior to transplantation.

Past attempts to reduce rejection rates included use of a higher stem cell dose, as well as the addition of total body irradiation to cyclophosphamide conditioning. Although that was associated with a reduced incidence of graft rejection, the benefit was negated by high transplant-related mortality (TRM) due to an increase in GVHD.


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