Autologous Stem Cell / Bone Marrow Transplant

Also called: bone marrow transplant, BMT, stem cell transplant, SCT

What is an autologous stem cell / bone marrow transplant?

An autologous stem cell / bone marrow transplant replaces damaged or destroyed bone marrow stem cells with healthy ones donated in advance by the patient. The “auto” prefix means “self” or “oneself.” Stem cells from the patient’s own blood and/or bone marrow are collected and used in the transplant. (Learn about transplants using donor cells from other people: allogeneic stem cell / bone marrow transplant.)

Stem cells are produced in the spongy area of bones known as marrow. These cells develop into all types of blood cells in the body. In an autologous stem cell / bone marrow transplant, the transplanted stem cells grow and become normal blood cells that help fight disease.

What are autologous stem cell / bone marrow transplants used to treat?

These transplants are used to treat blood-related cancers and other disorders, including:

What happens before an autologous stem cell / bone marrow transplant?

Clinicians decide whether the patient can have the transplant:

  • The patient is tested for infectious diseases in the same way as people donating blood.
  • A medical exam shows whether the patient will be able to tolerate the collection process.
  • Stem cells are collected (harvested):
    • Stem cells from the blood and/or bone marrow are taken from the patient and frozen.
    • If marrow is to be used, doctors usually insert a needle into a bone (often the pelvis) to pull out the marrow.
    • Another method uses blood stem cells that circulate throughout the body. This method obtains stem cells from the patient’s vein in a method similar to a blood donation.

Before the cells are transplanted, the patient receives treatment to destroy as many abnormal cells as possible and to increase the chance of transplant success. The patient may receive chemotherapy, radiation therapy or both. At the same time that these therapies kill cancer cells they can also destroy many healthy blood cells.

  • Chemotherapy (“chemo”) - uses powerful medicines to kill cancer cells or stop them from growing (dividing) and making more cancer cells.
    • Chemo may be injected into the bloodstream, so that it can travel throughout the body.
    • Some chemo may be given by mouth.
    • Combination therapy uses more than one type of chemo at a time.
  • Ablative therapy (also called myeloablative therapy) - uses high-dose chemo to kill both healthy and cancerous bone marrow cells, leaving the transplanted cells more room to grow. The dose can be changed depending on your child’s ability to tolerate this strong treatment. A “mini-transplant” uses lower, less-toxic chemo doses and/or radiation.
  • Radiation therapy - uses high-energy X-rays or other types of radiation to kill cancer cells or stop them from growing.

How are autologous stem cell / bone marrow transplants done?

After the chemo and / or radiation treatment is complete, the patient’s healthy stem cells are transplanted into the bloodstream:

  • The procedure is similar to a blood transfusion.
  • The cells travel through the blood until they reach the marrow and start growing new, healthy cells.

What problems can occur with autologous stem cell / bone marrow transplants?

Patients receive their own stem cells, so there is no risk that the immune system will react to the transplanted cells as foreign and attack or reject them (graft-versus-host disease).

Problems that may occur include:

  • Sometimes stem cell harvesting also collects cancer cells, which may cause the cancer to return.
  • Infection is common because fewer white blood cells are present to fight it.
  • Fewer red blood cells can cause anemia.
  • Low platelet levels can cause blood clotting problems and dangerous bleeding.

What are the survival rates for autologous stem cell / bone marrow transplants?

Survival in children following autologous stem cell / bone marrow transplantation depends on:

  • What type of cancer the child has
  • How well the child responded to earlier treatment
  • How far the cancer has spread, if it has, to other areas of the body

In a recent study, children with acute promyelocytic leukemia had an overall five-year survival rate of 82 percent following autologous stem cell transplant.

Patients with solid tumors that have relapsed or spread and who have a certain protein called KIR on the cancer-fighting immune cells (natural killer cells), have a survival rate of 70 percent following autologous stem cell transplant.

Why come to St. Jude for autologous stem cell / bone marrow transplantation?

  • St. Jude is the only National Cancer Institute-designated Comprehensive Cancer Center devoted solely to children.
  • St. Jude has created more clinical trials for cancer than any other children’s hospital in the United States.
  • The nurse-to-patient ratio at St. Jude is unmatched— averaging 1:3 in hematology and oncology, and 1:1 in the Intensive Care Unit.
  • The St. Jude Transplant Program has extensive experience, having performed more than 2,500 transplants since 1982.
  • St. Jude is accredited by the Foundation for the Accreditation of Cellular Therapy (FACT) for autologous and allogeneic peripheral blood and marrow transplantation in children and adults.
  • To improve outcomes and reduce side effects associated with transplants, doctors in the St. Jude Transplant Program work closely with laboratory scientists to rapidly move discoveries from the lab to the clinic.
  • St. Jude support staff members have been specially trained to care for children receiving autologous stem cell / bone marrow transplants.
  • St. Jude researchers are studying different chemotherapies for pre-transplant conditioning to prevent relapse. Scientists are also researching how to prevent relapse by giving immunotherapy after transplantation.

Associated Clinical Trials

10-CBA: A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) for Transplantation in Pediatric and Adult Patients with Hematologic Malignancies and Other Indications

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

  • Participant is receiving an allogeneic hematopoietic stem cell transplant at St. Jude Children’s Research Hospital using an unlicensed cord blood unit (CBU).
  • The patient has a disorder affecting the hematopoietic system that is either inherited, acquired, or a result from myeloablative treatment.
View Trial

AML08: Clofarabine Plus Cytarabine Versus Conventional Induction Therapy And A Study Of NK Cell Transplantation In Newly Diagnosed Acute Myeloid Leukemia

A Randomized Trial of Clofarabine Plus Cytarabine Versus Conventional Induction Therapy and of Natural Killer Cell Transplantation Versus Conventional Consolidation Therapy in Patients With Newly Diagnosed Acute Myeloid Leukemia

Diseases Treated:

Acute myeloid leukemia (AML)

Eligibility:

  • 21 years of age or younger at the time of study entry
  • No prior therapy for AML except for one dose of intrathecal therapy and the use of hydroxyurea or low-dose cytarabine for hyperleukocytosis
  • Must not have Down syndrome, acute promyelocytic leukemia (APL), juvenile myelomonocytic leukemia (JMML), Fanconi anemia (FA), Kostmann syndrome, Shwachman syndrome and other  bone marrow failure syndromes
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ASCIST: Donor Natural Killer Cell Infusion after Autologous CD133+ Selected Stem Cell Transplant in Treating Younger Patients with High-Risk Solid Tumors or Lymphomas

A Phase I Study of Immunotherapy Including Haploidentical NK Cell Infusion Following CD133+Positively-Selected Autologous Hematopoietic Stem Cells In Children With High-Risk Solid Tumors or Lymphomas

Diseases Treated:

High-risk solid tumors and lymphomas

Eligibility:

  • Has a type of high-risk cancer including
    • Neuroblastoma
    • Solid tumors
    • Lymphomas
  • 21 years of age or younger
  • Has not received a prior hematopoietic (blood cells from bone marrow) stem cell transplant within the previous three months
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BMTFU: Protocol for Collecting Long-Term Follow-Up Data on Recipients of Hematopoietic Stem Cell Transplant

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

View Trial

DE0605: Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study

Diseases Treated:

Hepatic veno-occlusive disease

Eligibility:

  • The patient must have diagnosis of hepatic VOD
  • Patients must also provide voluntary written informed consent to the protocol to be eligible for the study.
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GENEFU: Long-Term Follow-Up of Patients who have Received Gene Therapy/Gene Marked Products

Long-Term Follow-Up of Patients who have Received Gene Therapy/Gene Marked Products

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

  • Participant received an integrating retroviral or lentiviral vector based gene transfer or gene marked product at St. Jude Children’s Research Hospital within the past 15 year time period.
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NCBP01: Safety Study of Unlicensed, Investigational Cord Blood Units Manufactured by the NCBP for Unrelated Transplantation

A Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units (CBUs) manufactured by the National Cord Blood Program (NCBP) and provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients

Diseases Treated:

Leukemia and other blood diseases

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

  • Participant is receiving an allogeneic hematopoietic stem cell (HSC) transplant at St. Jude Children's Research Hospital using an unlicensed cord blood unit (CBU).
  • Participant may be of any age and either gender.
  • Participant has a medical disorder affecting the hematopoietic system that is inherited, acquired, or a result from myeloablative treatment.
  • Participant is receiving HPC-CORD BLOOD product manufactured by NCBP (at least one, if the graft contains more than one units).
View Trial

NMDPD: Protocol for a Research Database for Hematopoietic Stem Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries

National Marrow Donor Program: A research Database for Allogeneic Unrelated Hematopoietic Stem Cell Transplantation

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

View Trial

RADIANT: Donor Hematopoietic Stem Cell Transplant in Treating Young Patients with Relapsed or Refractory Solid Tumors or Lymphomas

A Phase I Study of CD45RA+ Depleted Haploidentical Stem Cell Transplantation In Children with Relapsed or Refractory Solid Tumors

Diseases Treated:

Relapsed or refractory solid tumors or lymphomas

Eligibility:

  • Between the ages of 2 and 21 years
  • Solid tumor or lymphoma as primary cancer
  • Cancer that has come back or did not respond to previous chemotherapy
  • No heart, kidney or liver problems
View Trial

REF2HCT: Haploidentical Bone Marrow Transplant for Leukemia and Lymphoma

Provision of TCRγδ T Cells and Memory T Cells plus Selected Use of Blinatumomab in Naïve T-cell Depleted Haploidentical Donor Hematopoietic Cell Transplantation for Hematologic Malignancies Relapsed or Refractory despite Prior Transplantation

Diseases Treated:

Eligibility:

  • 21 years old and younger
  • Diagnosed with one of the following that has come back or did not improve after bone marrow transplant
    • Acute lymphoblastic leukemia (ALL)
    • Acute myeloid leukemia
    • Myeloid sarcoma
    • Chronic myeloid leukemia (CML)
    • Juvenile myelomonocytic leukemia (JMML)
    • Myelodysplastic syndrome (MDS)
    • Non-Hodgkin lymphoma (NHL)
  • Has a family member who is a suitable stem cell donor
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TWIPES: Bath Wipes in Reducing Skin Bacteria in Younger Patients Undergoing Stem Cell Transplant

A Pilot Study of the Safety Followed by a Randomized Controlled Trial of the Efficacy of Non-Antimicrobial Theraworx™ Bath Wipes in the Reduction of Skin Colonization with Vancomycin-Resistant Enterococci Compared to Standard Bath Wipes in Children Undergoing Hematopoietic Stem Cell Transplantation

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

This clinical trial has inclusion and exclusion criteria. See full trial for specifics.

View Trial

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