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Pediatric Hematopoietic Stem Cell Transplant and Cellular Therapy for Cancer (PDQ®)–Health Professional Version

General Information About Hematopoietic Stem Cell Transplant (HSCT)

Rationale for HSCT

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:

  • Create marrow space.
  • Suppress the patient's immune system to prevent rejection.
  • Eradicate malignant cells in patients with cancer.

HSCT is currently used in the:

  • Treatment of malignancies,
  • Replacement or modulation of an absent or poorly functioning hematopoietic or immune system, or for the
  • Treatment of certain genetic diseases. In these cases, insufficient expression of the affected gene product can be partially or completely overcome by circulating hematopoietic stem cells transplanted from a donor with normal gene expression.

This summary focuses on the use of HSCT in the treatment of childhood malignancies.

Autologous Versus Allogeneic HSCT

The two major HSCT approaches currently in use are the following:

  • Autologous (using the patient's own hematopoietic stem cells).
  • Allogeneic (using related- or unrelated-donor hematopoietic stem cells).

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.

Determining When HSCT Is Indicated: Comparison of HSCT and Chemotherapy 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:

  1. Remission/disease status: Comparisons of HSCT and chemotherapy should include only patients who obtain remission, preferably after similar approaches to salvage therapy, because patients who fail to obtain remission have poor outcomes with any therapy.[10]

    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]

  2. Therapy approaches used for comparison: Comparisons should be made with the best or most used chemotherapy/immunotherapy or HSCT approaches used during the time frame under study.
  3. HSCT approach: HSCT approaches that are very high risk or have documented lower rates of survival should not be combined for analysis with standard-risk HSCT approaches.
  4. Criteria for relapse: Risk factors for relapse should be carefully defined, and analysis should be based on the most current knowledge of risk.
  5. Selection bias: Attempts should be made to understand and eliminate or correct for selection bias. Examples include the following:
    • Higher-risk patients preferentially undergoing HSCT (i.e., patients who take several rounds to achieve remission or have disease relapse after obtaining remission and go back into a subsequent remission before HSCT).
    • Sicker patients deferred from HSCT because of comorbidities.
    • Related to the time-to-transplant bias noted above, patients who undergo HSCT after relapse or recurrence are a subset of all patients with a disease recurrence and will be selected from those who are able to obtain a remission and remain healthy enough to undergo HSCT.
    • Patient or parent refusal.
    • Lack of or inability to obtain insurance approval for HSCT.
    • Lack of access to HSCT because of distance or inability to travel.

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:

  1. Matthay KK, Villablanca JG, Seeger RC, et al.: Treatment of high-risk neuroblastoma with intensive chemotherapy, radiotherapy, autologous bone marrow transplantation, and 13-cis-retinoic acid. Children's Cancer Group. N Engl J Med 341 (16): 1165-73, 1999. [PUBMED Abstract]
  2. Woods WG, Neudorf S, Gold S, et al.: A comparison of allogeneic bone marrow transplantation, autologous bone marrow transplantation, and aggressive chemotherapy in children with acute myeloid leukemia in remission. Blood 97 (1): 56-62, 2001. [PUBMED Abstract]
  3. Lawson SE, Harrison G, Richards S, et al.: The UK experience in treating relapsed childhood acute lymphoblastic leukaemia: a report on the medical research council UKALLR1 study. Br J Haematol 108 (3): 531-43, 2000. [PUBMED Abstract]
  4. Gaynon PS, Harris RE, Altman AJ, et al.: Bone marrow transplantation versus prolonged intensive chemotherapy for children with acute lymphoblastic leukemia and an initial bone marrow relapse within 12 months of the completion of primary therapy: Children's Oncology Group study CCG-1941. J Clin Oncol 24 (19): 3150-6, 2006. [PUBMED Abstract]
  5. Merli P, Algeri M, Del Bufalo F, et al.: Hematopoietic Stem Cell Transplantation in Pediatric Acute Lymphoblastic Leukemia. Curr Hematol Malig Rep 14 (2): 94-105, 2019. [PUBMED Abstract]
  6. Bertaina A, Merli P, Rutella S, et al.: HLA-haploidentical stem cell transplantation after removal of αβ+ T and B cells in children with nonmalignant disorders. Blood 124 (5): 822-6, 2014. [PUBMED Abstract]
  7. Handgretinger R, Chen X, Pfeiffer M, et al.: Feasibility and outcome of reduced-intensity conditioning in haploidentical transplantation. Ann N Y Acad Sci 1106: 279-89, 2007. [PUBMED Abstract]
  8. Huang XJ, Liu DH, Liu KY, et al.: Haploidentical hematopoietic stem cell transplantation without in vitro T-cell depletion for the treatment of hematological malignancies. Bone Marrow Transplant 38 (4): 291-7, 2006. [PUBMED Abstract]
  9. Luznik L, Fuchs EJ: High-dose, post-transplantation cyclophosphamide to promote graft-host tolerance after allogeneic hematopoietic stem cell transplantation. Immunol Res 47 (1-3): 65-77, 2010. [PUBMED Abstract]
  10. Pulsipher MA, Peters C, Pui CH: High-risk pediatric acute lymphoblastic leukemia: to transplant or not to transplant? Biol Blood Marrow Transplant 17 (1 Suppl): S137-48, 2011. [PUBMED Abstract]

Current Clinical Trials

Use our advanced clinical trial search to find NCI-supported cancer clinical trials that are now enrolling patients. The search can be narrowed by location of the trial, type of treatment, name of the drug, and other criteria. General information about clinical trials is also available.

Latest Updates to This Summary (06/13/2024)

The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.

This summary was comprehensively reviewed.

This summary is written and maintained by the PDQ Pediatric Treatment Editorial Board, which is editorially independent of NCI. The summary reflects an independent review of the literature and does not represent a policy statement of NCI or NIH. More information about summary policies and the role of the PDQ Editorial Boards in maintaining the PDQ summaries can be found on the About This PDQ Summary and PDQ® Cancer Information for Health Professionals pages.

About This PDQ Summary

Purpose of This Summary

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.

Reviewers and Updates

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).

Board members review recently published articles each month to determine whether an article should:

  • be discussed at a meeting,
  • be cited with text, or
  • replace or update an existing article that is already cited.

Changes to the summaries are made through a consensus process in which Board members evaluate the strength of the evidence in the published articles and determine how the article should be included in the summary.

The lead reviewers for Pediatric Hematopoietic Stem Cell Transplant and Cellular Therapy for Cancer are:

  • Thomas G. Gross, MD, PhD (National Cancer Institute)
  • Michael A. Pulsipher, MD (Huntsman Cancer Institute at University of Utah)

Any comments or questions about the summary content should be submitted to through the NCI website's Email Us. Do not contact the individual Board Members with questions or comments about the summaries. Board members will not respond to individual inquiries.

Levels of Evidence

Some of the reference citations in this summary are accompanied by a level-of-evidence designation. These designations are intended to help readers assess the strength of the evidence supporting the use of specific interventions or approaches. The PDQ Pediatric Treatment Editorial Board uses a formal evidence ranking system in developing its level-of-evidence designations.

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The preferred citation for this PDQ summary is:

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: Accessed <MM/DD/YYYY>. [PMID: 26389503]

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