Hematopoietic stem cell transplantation (HSCT)

Hematopoietic stem cell transplantation (HSCT), also known as bone marrow transplantation, is a special form of therapy that involves taking cells that are normally found in the bone marrow (stem cells) from the donor or from cord blood and giving them back, by intravenous infusion, to the recipient, either the same person or to another person.

The sources of these stem cells can be:

from the patient (autologous)
from a relative (allogeneic)
from a matched unrelated donor (MUD)

Rationale for use of HSCT

Many chemotherapeutic agents show a steep dose response curve - small changes in dose produce significant changes in response. But the bone marrow limits the amount of chemotherapy and radiation that can be given. The use of HSCT allows use of higher doses of drugs and obviates concern about marrow toxicity.

Indications for HSCT for pediatric malignancies

Allogeneic (hematopoietic cell source is not obtained from the patient)

ALL (very high risk or in 2nd remission)
AML, JMML, CML, MDS
lymphoma (2nd remission)

Autologous (hematopoietic cell source is obtained from the patient):

neuroblastoma (advanced stage)
relapsed Hodgkins disease
stage 4 solid tumours (possible indication)
relapsed solid tumours (possible indication)
brain tumours (possible indication)
relapsed AML, ALL, CML (possible indication)

Types of HSCT

Allogeneic

Syngeneic, i.e. from an identical twin
Matched related: usually from an HLA matched sibling, usually first choice of donor for leukemia.
Matched unrelated (MUD): the donor is usually identified through a donor registry, used if there is no matched related donor available.
Partially matched related donor: may be used if there is no matched donor available. May be T cell depleted or selected for CD34+ stem cells.
Cord blood: The stem cells from cord blood can be used for a matched or partially matched allogeneic HSCT.

Autologous

Autologous purged transplant (i.e. treated ex vivo to remove malignant cells): this type of transplant may be used for leukemia (AML) or neuroblastoma.
Autologous non-purged: used for diseases that do not infiltrate the bone marrow to allow ultra high doses of chemotherapy and radiotherapy, e.g. Hodgkin's Lymphoma.

Common complications of HSCT

Toxicity from the chemotherapy and/or radiotherapy, e.g. GI inflammation, liver, renal and lung toxicity.
Complications of pancytopenia - bleeding, infection, and anemia.
Immunological problems:

Graft versus host disease: In this disorder the bone marrow graft recognizes the host as being foreign and attacks the host cells. This results in an autoimmune disease primarily affecting the skin, liver, GI tract and joints.
Rejection of the graft.
Immunodeficiency.

Long-term toxicity: includes endocrine and growth abnormalities, cataracts, decreased fertility, learning disabilities and second malignancy.

For a more comprehensive discussion on HSCT in children, please see the Spring 2005 Pediatric Oncology/Hematology Network newsletter.

For information for families with children undergoing transplantation, see:

FAQs about HSCT - updated April 2007
Going Home Package – Allogeneic SCT - updated June 2008
Going Home Package – Autologous SCT - updated July 2008
Peripheral Blood Stem Cell Collection - PBSC - updated November 2007

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The information on this site, i.e. guideline/educational material/policy or procedure, has been developed for use only within BC Children's Hospital (BC Children’s). There are support systems at BC Children’s that may not exist in other clinical settings, and therefore, any adoption of these materials cannot be the responsibility of BC Children’s. Agencies other than BC Children’s should use this information as a guideline for reference purposes only. All materials are the property of BC Children’s and may only be reprinted in whole or in part with our expressed permission.

Page updated July 2008

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PHSA improves the health of British Columbians by seeking province-wide solutions to specialized health care needs in collaboration with BC health authorities and other partners. For more information, visit http://www.phsa.ca

PHSA agencies include: BC Cancer Agency, BC Centre for Disease Control, BC Children’s Hospital and Sunny Hill Health Centre for Children, BC Mental Health & Addiction Services, BC Provincial Renal Agency, BC Transplant, BC Women’s Hospital & Health Centre, and Cardiac Services BC.