Allogeneic stem cell transplantation for the treatment of diseases associated with a deficiency in bone marrow products

Our understanding of the pathophysiology of hematopoietic failure associated syndromes led to the developmental of potentially curative procedures for the treatment of many diseases including Severe aplastic anemia, Fanconi's anemia, Primary immunodeficiency, Osteopetrosis, and Metabolic diseases. Although the number of patients that were transplanted for bone marrow deficiency diseases is relatively low as compared to patients with hematological malignancies, the impact on the knowledge of hematopoiesis and transplantation biology is tremendous. Moreover, the patient's average young age suffering from these diseases further encourage searching for curative approaches. Lucking a fully MHC matched donor, remained a significant obstacle in stem cell transplantation for non-malignant hematological disorders. Lessons from attempts to cure aplasic anemia with bone marrow transplantation guided us to the improvement of pretransplant conditioning regimens and prevention of graft versus host reactions after transplantation. Furthermore, in recent years optimization of disease specific protocol have been successfully designed and clinically applied.