1st successfully treated multi-transfused patient with severe aplastic anemia using reduced intensity non-myeloablative stem cell transplantation using total lymphoid irradiation (TLI) for prevention of graft rejection
This young woman presented with multiply-transfused severe aplastic anemia. Aplastic anemia is an acquired disease characterized by deficiency of hematopoietic stem cells. This results in severe anemia and a constant need of blood transfusions. Furthermore, the lack of production of white blood cells increases the risk of life-threatening infections. Poor platelet production causes a serious risk of spontaneous bleeding.
Allogeneic stem cell transplantation is the only possible approach for cure of severe aplastic anemia, depending on durable engraftment of normal donor’s hematopoietic stem cells. Due to multiple transfusions received prior to admission for transplant, there is an increased risk of graft rejection following a standard transplant procedure.
Low dose total lymphoid irradiation (TLI) was applied for the first time in order to prevent rejection of donor’s stem cells, in addition to a well-tolerated and safe conditioning. Fast engraftment was accomplished, and the patient recovered without any major complications.
This young woman got married several months later and gave birth to 3 children. Before Slavin’s time, conventional transplant procedures resulted in engraftment failure due to allograft rejection and using more aggressive conditioning for prevention of graft rejection resulted in an increased risk of loss of ovarian function in women and loss of sperm production in men. This former patient is currently over 38 years out with no need for any additional treatment since the transplant procedure.
Avoiding the use of aggressive myeloablative conditioning also minimizes the risk of graft-vs-host disease (GVHD) following allogeneic stem cell transplantation, the most serious unavoidable complication following conventional allogeneic stem cell transplantation.