Blood and marrow transplant, or HSCT, is a procedure that involves infusion of hematopoietic stem cells (along with hematopoietic progenitor cells) to reconstitute the hematopoietic system of a patient. The infusion of hematopoietic stem cells generally follows a preparative regimen consisting of agents designed to do the following:
HSCT is currently used in the:
This summary focuses on the use of HSCT in the treatment of childhood malignancies.
The two major HSCT approaches currently in use are the following:
An autologous transplant treats cancer by exposing patients to high-dose therapy with the intent of overcoming chemotherapy resistance in tumor cells, followed by infusion of the patient’s previously stored hematopoietic stem cells. The transplant can be performed in a single procedure or tandem sequential procedures.
Allogeneic 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. 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. Therefore, patients may require allogeneic approaches for optimal outcomes.
Because 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. 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.[1,2] 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.[3,4]
In general, HSCT typically benefits only children at high risk of relapse with standard chemotherapy approaches. Accordingly, treatment schemas that accurately identify these high-risk patients and offer HSCT if appropriate allogeneic donors are available are the preferred approach for many diseases.[5] Less well-established, higher-risk approaches to HSCT, such as haploidentical transplant, are sometimes reserved for only the very highest-risk patients. However, these higher-risk approaches are becoming safer and more efficacious and are increasingly used interchangeably with fully matched allogeneic approaches.[6-9] For more information, see the Haploidentical HSCT section in Pediatric Allogeneic Hematopoietic Stem Cell Transplant.
When comparisons of similar patients treated with HSCT or chemotherapy are made in the setting where randomized or intent-to-treat studies are not feasible, the following issues should be considered:
To account for time-to-transplant bias, the chemotherapy comparator arm should include only patients who maintained remission until the median time to HSCT. The HSCT comparator arm should also include only patients who achieved the initial remission mentioned above and maintained that remission until the time of HSCT.[10]
High-risk and intermediate-risk patient groups should not be combined because benefit or lack of benefit of HSCT in the high-risk group can be masked by different levels of benefit in the intermediate-risk group.[10]
Physician bias, for or against HSCT, is difficult to control for or detect. The effects of access to HSCT and therapeutic bias on outcomes of pediatric malignancies for which HSCT may be indicated have been poorly studied.
For more information about pediatric HSCT, see the following summaries:
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This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the use of hematopoietic stem cell transplant and cellular therapy in treating pediatric cancer. It is intended as a resource to inform and assist clinicians in the care of their patients. It does not provide formal guidelines or recommendations for making health care decisions.
This summary is reviewed regularly and updated as necessary by the PDQ Pediatric Treatment Editorial Board, which is editorially independent of the National Cancer Institute (NCI). The summary reflects an independent review of the literature and does not represent a policy statement of NCI or the National Institutes of Health (NIH).
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PDQ® Pediatric Treatment Editorial Board. PDQ Pediatric Hematopoietic Stem Cell Transplant and Cellular Therapy for Cancer. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/childhood-cancers/hp-stem-cell-transplant. Accessed <MM/DD/YYYY>. [PMID: 26389503]
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