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In general, HSCT typically benefits only children at high risk of relapse with standard chemotherapy approaches. However, for very high-risk patients, such as those with early relapse of acute lymphoblastic leukemia, randomized trials have not been feasible because of investigator bias.

For some diseases, randomized trials or intent-to-treat trials using an HLA-matched sibling donor have established the benefit of HSCT by direct comparison. Determining When HSCT Is Indicated: Comparison of HSCT and Chemotherapy Outcomesīecause the outcomes using chemotherapy and HSCT treatments have been changing over time, these approaches should be compared regularly to continually redefine optimal therapy for a given patient. Although autologous approaches are associated with less short-term mortality, many malignancies are resistant to even high doses of chemotherapy and/or involve the bone marrow, thus requiring allogeneic approaches for optimal outcome. Autologous transplant techniques are also being used to enable engraftment of genetically modified autologous hematopoietic stem cell progenitors to correct or ameliorate inherited disorders (e.g., immunodeficiencies, metabolic disorders, and hemoglobinopathies).Īllogeneic transplant approaches to cancer treatment also may involve high-dose therapy, but because of immunologic differences between the donor and recipient, an additional graft-versus-tumor or graft-versus-leukemia treatment effect can occur.

Meaningful percentages of cure or long-term remission from the disease must occur without significant nonhematopoietic toxicities that would otherwise limit the therapeutic benefit achieved.Īutologous transplants have also been used to reset the immune system in patients with severe autoimmune disorders.Ĭurrent pediatric indications for autologous transplant include patients with certain lymphomas, neuroblastoma, and brain tumors.

This approach may include increased tumor kill in areas where standard-dose chemotherapy has less penetration (central nervous system).

A dose-intensified chemotherapy regimen (with or without radiation therapy) with hematopoietic stem cell support is used to achieve a significantly higher cell kill than could be achieved without the use of hematopoietic stem cell support.