Successful allogeneic stem cell transplantation with fludarabine-based conditioning regimen in severe aplastic anemia.

ABSTRACT

Graft failure is a problem in HLA-identical sibling transplants for patients with refractory severe aplastic anaemia (SAA). Intensification efforts includes addition of radiation or biologic agents such as antithymocyte globulin (ATG), procarbazine or cyclophosphamide has often been advocated to combat this problem. With this approach engraftment rate has improved. However the incidence of transplant related complications are also increased, resulting in little change in the overall outcome. We therefore investigated the use of combination of fludarabine and cyclophosphamide as a non-myeloablative conditioning regimen in a patient who was refractory to multiple immunosuppressive agents and transfusions. He received peripheral blood stem cells from his HLA-identical sibling donor. With a follow up of eighteen months, the patient is alive with complete and durable hematopoietic engraftment. Fludarabine-based conditioning regimen therefore has the potential to be successfully and safely used in patients with SAA undergoing transplant.