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A Rational Appraisal of Bone Marrow Transplantation and Immunosuppressive Therapy for Severe Aplastic Anemia
1Bone Marrow Transplant Program, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida 33612, USA.
With significant improvements in the rates of graft rejection and acute graft-vs-host disease, bone marrow transplantation remains the therapy of choice for the young aplastic anemia patient with an HLA-matched related donor. While immunosuppressive therapy can induce responses in a significant proportion of patients, long-term follow-up studies have demonstrated a high rate of relapses and evolution to malignant clonal hematopoietic disorders. Therefore, this therapeutic option should be considered for patients who are not transplant candidates. The potential roles for hematopoietic growth factors and alternative marrow donors needs to be further defined.
With significant improvements in the rates of graft rejection and acute graft-vs-host disease, bone marrow transplantation remains the therapy of choice for the young aplastic anemia patient with an HLA-matched related donor. While immunosuppressive therapy can induce responses in a significant proportion of patients, long-term follow-up studies have demonstrated a high rate of relapses and evolution to malignant clonal hematopoietic disorders. Therefore, this therapeutic option should be considered for patients who are not transplant candidates. The potential roles for hematopoietic growth factors and alternative marrow donors needs to be further defined.