Bone Marrow Transplantation for Diseases of Childhood

Abstract

Bone marrow transplantation in childhood is an established treatment modality for aplastic anemia, the acute and chronic leukemias, and severe combined immune deficiency. Recently, experience with this treatment has also been favorable with small numbers of children who have Wiskott-Aldrich syndrome, several types of inherited storage diseases, Fanconi's anemia, thalassemia, infantile malignant osteopetrosis, and selected cases of lymphoma and other solid tumors. The psychosocial impact and financial costs of bone marrow transplantation can be substantial. Multi-institutional, prospective, randomized trials that would compare transplantation and conventional therapy are necessary to establish the indications and precise timing for this procedure. Further development of monoclonal antibodies, a better understanding of the histocompatibility antigen systems, and improvement in pretransplantation conditioning regimens should increase the spectrum of effectiveness for bone marrow transplantation in the coming years.

Treating Genetic Diseases: Lessons from Three Children

“…Although time does not permit me to catalog efficacy of transplantation at this time, it is fair to say that it holds great promise (21)(22)(23). A large fraction of transplant recipients, 80% for kidney and bone marrow, are disease-free survivors 1 year or more after transplantation.…”

Pediatric Bone Marrow Transplantation: Intensive Care Management

“…Although chemotherapy is curative in many cases of childhood leukemia and solid tumors, BMT is an important secondary treatment for children whose disease relapses or progresses despite therapy [3][4][5][6]21]. In children who may ultimately be referred for BMT the number of transfusions given should be minimized because exposure may result in sensitization against antigens shared by the donor, and in graft rejection [22].…”

“…Leukemia and other malignancies require more aggressive pretransplantation therapy both to kill residual tumor cells and ablate the host marrow. Nevertheless, leukemic relapse after transplantation remains a major problem [3][4][5][6]21]. Even higher doses of chemotherapy are given to condition patients with solid tumors such as neuroblastoma [7].…”

Family Factors Affecting Bone Marrow Transplantation

“…Kamani and August [1] note that clinical outcomes vary as a func tion of type of underlying disease and type of transplant. Overall, survival is estimated at 50% [4].…”

“…As pointed out by Stuber [9], marital strain is common as are regression and behav ioral disturbances in the siblings. If a sibling is a donor, s/he may be particularly vulnerable for psychological difficulties [10], While the medical aspects of pediatric BMT have been the main focus thus far in the literature [4], attention is beginning to be paid to psychological sequelae [11,12] and psycho social interventions which may be offered to BMT patients' families [13]. Notably, the pos sible links between psychosocial factors and medical outcomes have not been studied em pirically, using a prospective design.…”