Bone marrow transplantation in children with hereditary disorders

Hereditary disorders represent a very heterogenous group of diseases affecting the hematopoietic system. Bone marrow transplantation is the treatment of choice for immunodeficiencies, mucopolysaccharidosis, thalassemia, Fancoli's anemia, osteopetrosis, and other rare disorders. Indications for bone marrow transplantation are well known when an HLA-identical sibling is identified. Results of alternative donor transplantations are still under investigation. Recently, progress has been made in the analysis of the mechanism of the disease at the molecular level and in the identification of gene mutations. Clinical implications of these discoveries might modify the future indications for bone marrow transplantation.