Recent Advances in Bone Marrow Transplantation in Children

Abstract

The most significant recent advances in bone marrow transplantation in children include the increasing number of diseases which can be treated successfully by this therapy, methods of increasing the number of available donors and the prevention of cytomegalovirus (CMV) infections. Matched allogeneic marrow transplantation is now established therapy for selected patients with leukemia, myelodysplastic syndromes, aplastic anemia, immunodeficiency syndromes and osteopetrosis. Recently, marrow transplantation has been successfully extended to the treatment of hemoglobinopathies, particularly thalassemia, and certain metabolic storage disorders such as Hurler's syndrome and metachromatic leukodystrophy. The development of effective marrow T-cell depletion techniques and unrelated donor registries offer the promise of increasing the number of patients who can benefit from this therapy by enabling mismatched transplantation. Obstacles that have to be overcome include the increased risk of graft failure and leukemic relapse associated with T-cell depletion, and graftversus-host disease characterizing mismatched transplantation. Finally a major advance in preventing CMV pneumonia, until recently the major cause of death following bone marrow transplantation, has been use of CMV-negative blood products when the patient and marrow donor are CMV negative.