Skip to main navigation Skip to main content Skip to footer For Medicare For Providers For Brokers For Employers Español For Individuals & Families: For Individuals & Families Medical Dental Other Supplemental Explore coverage through work How to Buy Health Insurance Types of Dental Insurance Open Enrollment vs. Special Enrollment See all topics Shop for Medicare plans Member Guide Find a Doctor Log in to myCigna
Home Knowledge Center Wellness Library Pediatric Hematopoietic Stem Cell Transplant and Cellular Therapy for Cancer (PDQ®): Treatment - Health Professional Information [NCI]

Pediatric Hematopoietic Stem Cell Transplant and Cellular Therapy for Cancer (PDQ®): Treatment - Health Professional Information [NCI]

This information is produced and provided by the National Cancer Institute (NCI). The information in this topic may have changed since it was written. For the most current information, contact the National Cancer Institute via the Internet web site at or call 1-800-4-CANCER.

General Information About Hematopoietic Stem Cell Transplant (HSCT)

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, thus requiring allogeneic approaches for optimal outcome.

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 generally 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.[6,7,8,9] 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 do very poorly 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 a benefit of HSCT in the high-risk group can be masked when outcomes are similar to those achieved in the intermediate-risk group.[10]

  2. Therapy approaches used for comparison: Comparisons should be made with the best or most commonly used chemotherapy and 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 who 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. Pediatric Autologous Hematopoietic Stem Cell Transplant.
  2. Pediatric Allogeneic Hematopoietic Stem Cell Transplant.
  3. Pediatric Chimeric Antigen Receptor (CAR) T-Cell Therapy.
  4. Complications, Graft-Versus-Host Disease, and Late Effects after Pediatric Hematopoietic Stem Cell Transplant.


  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.
  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.
  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.
  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.
  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.
  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.
  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.
  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.
  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.
  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.
Current Clinical Trials

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 (12 / 12 / 2023)

Latest Updates to This Summary (12 / 12 / 2023)

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.

Editorial changes were made to this summary.

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

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 (Children's Hospital Los Angeles)

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.

Permission to Use This Summary

PDQ is a registered trademark. Although the content of PDQ documents can be used freely as text, it cannot be identified as an NCI PDQ cancer information summary unless it is presented in its entirety and is regularly updated. However, an author would be permitted to write a sentence such as "NCI's PDQ cancer information summary about breast cancer prevention states the risks succinctly: [include excerpt from the summary]."

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]

Images in this summary are used with permission of the author(s), artist, and/or publisher for use within the PDQ summaries only. Permission to use images outside the context of PDQ information must be obtained from the owner(s) and cannot be granted by the National Cancer Institute. Information about using the illustrations in this summary, along with many other cancer-related images, is available in Visuals Online, a collection of over 2,000 scientific images.


Based on the strength of the available evidence, treatment options may be described as either "standard" or "under clinical evaluation." These classifications should not be used as a basis for insurance reimbursement determinations. More information on insurance coverage is available on on the Managing Cancer Care page.

Contact Us

More information about contacting us or receiving help with the website can be found on our Contact Us for Help page. Questions can also be submitted to through the website's Email Us.

Last Revised: 2023-12-12

This information does not replace the advice of a doctor. Healthwise, Incorporated, disclaims any warranty or liability for your use of this information. Your use of this information means that you agree to the Terms of Use. Learn how we develop our content.

Healthwise, Healthwise for every health decision, and the Healthwise logo are trademarks of Healthwise, Incorporated.

<cipublic-spinner variant="large"><span>Loading…</span></cipublic-spinner>

Page Footer

I want to...

Get an ID card File a claim View my claims and EOBs Check coverage under my plan See prescription drug list Find an in-network doctor, dentist, or facility Find a form Find 1095-B tax form information View the Cigna Glossary Contact Cigna


Individuals and Families Medicare Employers Brokers Providers

Secure Member Sites

myCigna member portal Health Care Provider portal Cigna for Employers Client Resource Portal Cigna for Brokers

The Cigna Group Information

About Cigna Healthcare Company Profile Careers Newsroom Investors Suppliers The Cigna Group Third Party Administrators International Evernorth

 Cigna. All rights reserved.

Privacy Legal Product Disclosures Cigna Company Names Customer Rights Accessibility Non-Discrimination Notice Language Assistance [PDF] Report Fraud Sitemap Cookie Settings


Individual and family medical and dental insurance plans are insured by Cigna Health and Life Insurance Company (CHLIC), Cigna HealthCare of Arizona, Inc., Cigna HealthCare of Illinois, Inc., Cigna HealthCare of Georgia, Inc., Cigna HealthCare of North Carolina, Inc., Cigna HealthCare of South Carolina, Inc., and Cigna HealthCare of Texas, Inc. Group health insurance and health benefit plans are insured or administered by CHLIC, Connecticut General Life Insurance Company (CGLIC), or their affiliates (see a listing of the legal entities that insure or administer group HMO, dental HMO, and other products or services in your state). Accidental Injury, Critical Illness, and Hospital Care plans or insurance policies are distributed exclusively by or through operating subsidiaries of Cigna Corporation, are administered by Cigna Health and Life Insurance Company, and are insured by either (i) Cigna Health and Life Insurance Company (Bloomfield, CT); (ii) Life Insurance Company of North America (“LINA”) (Philadelphia, PA); or (iii) New York Life Group Insurance Company of NY (“NYLGICNY”) (New York, NY), formerly known as Cigna Life Insurance Company of New York. The Cigna name, logo, and other Cigna marks are owned by Cigna Intellectual Property, Inc. LINA and NYLGICNY are not affiliates of Cigna.

All insurance policies and group benefit plans contain exclusions and limitations. For availability, costs and complete details of coverage, contact a licensed agent or Cigna sales representative. This website is not intended for residents of New Mexico.

Selecting these links will take you away from to another website, which may be a non-Cigna website. Cigna may not control the content or links of non-Cigna websites. Details