Insight into the Pathophysiology of Aplastic Anemia Provided Through the Results of Marrow Transplantation
The patient with aplastic anemia who has a normal identical twin to serve as marrow donor provides a unique opportunity for therapy by marrow transplantation which in turn offers insight into the basic nature of the disease. In 1964, in describing the second successful such case transplanted in Seattle , we argued that restoration of marrow function by a simple infusion of normal marrow, without any conditioning regimen, argued strongly against the disease being due to lack of a nutrient or hormone or a persisting toxic factor. Rather, correction of the defect by an infusion of “stem cells” indicated that the probable mechanism involved an acquired intrinsic stem cell defect. In addition, the etiological agent responsible for the original marrow failure must have been transient in nature.
KeywordsMarrow Transplantation Aplastic Anemia Graft Rejection Paroxysmal Nocturnal Hemoglobinuria Severe Aplastic Anemia
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