Pediatric bone marrow transplantation

Abstract

Bone marrow transplantation has been used to treat children with a variety of malignant, hematologic, immunologic, and genetic disorders. It is currently the treatment of choice for patients with certain types of leukemia (eg, acute myelogenous leukemia, chronic myelogenous leukemia, juvenile chronic myelogenous leukemia), severe aplastic anemia, severe combined immunodeficiency syndrome, and select genetic diseases (eg, Hurler's syndrome, osteopetro-sis). However, most children who would benefit from a transplant do not have an HLA-identical sibling to serve as the marrow donor. Several strategies have been developed to circumvent this problem including the utilization of unrelated volunteers or partially matched family members as donors, and autologous bone marrow or peripheral stem cell transplants. Progress has also been made in reducing the occurrence of transplant-related complications. The incidence of cytomegalovirus disease has diminished with the use of screened blood products and antiviral drug therapy. The risk of bacterial infections has also been reduced by the prophylactic administration of antibiotics and intravenous gammaglobulin. In addition, cytokines such as recombinant human granulocyte-macrophage colony-stimulating factor may promote early neutrophil recovery after transplantation, providing additional protection against infection. Cytokines may also prolong the survival of patients with graft failure. New methods have been reported for the prevention and treatment of graft-versus-host disease by the selective elimination of T lymphocytes from the marrow before transplantation or from the blood after transplantation. For children who undergo transplantation because of an oncologic disorder, relapse remains a major cause of treatment failure. The use of marrow purging techniques and biologic response modifiers may further improve the outlook for these patients.