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Autologous Bone Marrow Transplant

Also called: blood and marrow transplant, hematopoietic stem cell transplant (HSCT), hematopoietic cell transplant (HCT)

What is an autologous bone marrow transplant?

An autologous bone marrow transplant replaces damaged or destroyed blood-making cells with healthy ones donated in advance by the patient. The “auto” prefix means “self” or “oneself.” Blood-making 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 bone marrow transplant, a patient first receives high-doses of cancer-directed therapy. This therapy may injure or destroy the child’s normal blood cells. Patients are given their own cells after this high-dose therapy, to replace the ones that were just destroyed. Learn about transplants using donor cells from other people: allogeneic stem cell / bone marrow transplant.

What are autologous bone marrow transplants used to treat?

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

  • Hodgkin lymphoma
  • Non-Hodgkin lymphoma
  • Acute promyelocytic leukemia
  • Some solid tumors such as neuroblastoma

Autologous transplants can also be used as part of gene therapy for disorders of the immune system.

What happens before an autologous bone marrow transplant?

To decide whether the patient can have the transplant, clinicians do the following:

  • Test the patients for infectious diseases.
  • Conduct a medical exam to see if the patient will be able to tolerate the collection process.
  • Collect blood-making cells:
    • These cells from the blood and/or bone marrow are taken from the patient and frozen. They are then stored until the patient is ready for the transplant.
    • If marrow will be used, doctors usually insert a needle into a bone (often the pelvis) to pull out the marrow.
    • Another method uses blood-making cells that circulate throughout the body. This method takes cells from the patient’s vein in a method similar to a blood donation.

Before the transplant, the patient receives cancer-directed treatment to destroy as many abnormal cells as possible. This is called the preparative or conditioning regimen. This may include chemotherapy, radiation therapy or both. At the same time 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.
  • Radiation therapy - uses high-energy X-rays or other types of radiation to kill cancer cells or stop them from growing.

How are autologous bone marrow transplants done?

After the chemo and/or radiation treatment, your child's stored healthy cells will be thawed. These cells are given back to your child through 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 bone marrow transplants?

Patients receive their own cells, so there is no risk that the immune system will view the transplanted cells as foreign and attack or reject them.

Before the bone marrow can begin producing new, healthy cells, your child could have: 

  • Side effects from the conditioning regimen received before the cell infusion
  • Infection because fewer white blood cells (or immune cells) are present to protect the patient
  • Anemia caused by fewer red blood cells
  • Blood-clotting and bleeding problems caused by low platelet levels
  • Graft failure if the cells do not grow well (this is very rare)

After the new cells have grown, patients could:

  • Have side effects from the therapy used to treat their underlying disease or as part of the transplant
  • Undergo disease relapse or reoccurrence

What are the survival rates for autologous bone marrow transplants?

Survival in children following autologous bone marrow transplantation depends on:

  • The 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

Why come to St. Jude for autologous 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 3,200 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 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

CATCHAML: CAR T-Cell Therapy for Acute Myelogenous Leukemia (AML)

CATCHAML: CD123-Directed Autologous T-Cell Therapy for Acute Myelogenous Leukemia

Diseases Treated:

Acute Myelogenous Leukemia

Eligibility:

  • 21 years old or younger
  • Relapsed/refractory CD123+ AML, B-ALL, T-ALL or blastic plasmacytoid dendritic cell neoplasm
  • Has a suitable bone marrow transplant donor for allogeneic bone marrow transplant
View Trial

CN160: Ruxolitinib Therapy in Children after Bone Marrow Transplant

A Phase II Pediatric Study of a Graft-vs.-host disease (GVHD) prophylaxis regimen with no calcineurin inhibitors after day +60 post first allogeneic hematopoietic cell transplant for hematological malignancies

Diseases Treated:

Eligibility:

  • Ages 12–21 years
  • Lymphoid or myeloid-based cancer that requires a bone marrow transplant

 

View Trial

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.
View Trial

HAP2HCT: Partially Matched Family Donor Bone Marrow Transplant in Children and Young Adults with High Risk Cancer

TCRαβ-depleted Progenitor Cell Graft with Additional Memory T-Cell DLI, plus Selected Use of Blinatumomab, in Naïve T-Cell depleted Haploidentical Donor Hematopoietic Cell Transplantation for Hematologic Malignancies

Diseases Treated:

Eligibility:

For transplant recipient:

  • 21 years or younger
  • Does not have a suitable sibling donor or volunteer unrelated donor
  • Has a suitable single haplotype matched family member donor
  • Diagnosed with high risk hematologic malignancy
  • No prior allogeneic hematopoietic cell transplant
View Trial

HAPSAA: Partially Matched Related Donor Bone Marrow Transplant for Patients with Aplastic Anemia

Haploidentical Donor Hematopoietic Cell Transplantation for Patients with Severe Aplastic Anemia

Diseases Treated:

Aplastic Anemia

Eligibility:

  • 21 years old and younger
  • Diagnosis of severe aplastic anemia
  • No available matched donor
View Trial

LVXSCID-ND: Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) in Newly Diagnosed Infants

A Pilot Feasibility Study of Gene Transfer For X-Linked Severe Combined Immunodeficiency (SCID-X1) in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector To Transduce Autologous CD34 + Hematopoietic Stem Cells

Diseases Treated:

X-linked Severe Combined Immunodeficiency (SCID-X1)

Eligibility:

  • Diagnosis of Severe Combined Immunodeficiency, X-linked (SCID-X1)
  • Newborn to 2 years of age
  • No prior therapy with allogeneic stem cell transplantation
  • No HIV infection
View Trial

MEMCAR19: Allogeneic CAR T-Cell Therapy for Relapsed/Refractory CD19-Positive Leukemia

Phase I Study Evaluating Allogeneic Memory T Cells Engineered to Express Chimeric Antigen Receptors Specific for CD19 for the Treatment of Pediatric and Young Adult Patients with Relapsed or Refractory CD19-positive Leukemia

Diseases Treated:

Acute lymphoblastic leukemia

Eligibility:

Donor eligibility includes:

  • At least 18 years old
  • At least single haplotype matched family member
  • HIV negative
  • Not pregnant or breastfeeding
  • Completed the process of donor eligibility determination as defined in the study

 

Recipient eligibility includes:

  • 21 years old or younger*
  • Diagnosed with relapsed and/or refractory CD19-positive leukemia (as defined in the study)
  • Cohort A only – Relapsed and/or refractory CD19-positive leukemia AND previously received a hematopoietic cell transplant from the selected CAR-T donor
  • Cohort B only: Can not receive autologous CD19-CAR T-cell therapy (as defined in the study)
  • Detectable CD19+ leukemia in the bone marrow
  • Adequate organ function (as defined in the study)

* Initial 3 participants must be at least 12 years old.

View Trial

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

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
View Trial

SCDHCT: Reduced Intensity Related Donor HCT for Patients with Severe Sickle Cell Disease

Reduced Intensity Related Donor Peripheral Blood Derived Hematopoietic Progenitor Cell Transplantation for Patients with Severe Sickle Cell Disease

Diseases Treated:

Sickle Cell Disease

Eligibility:

  • 25 years old or younger
  • Diagnosed with severe sickle cell disease
  • Has a suitable HLA-matched sibling donor or single haplotype matched (half matched) family member donor (parents or siblings)
View Trial

SJCAR19: CAR T-Cell Therapy for Children and Young Adults with Acute Lymphoblastic Leukemia

A Phase I/II Study Evaluating CD19-Specific CAR Engineered Autologous T-Cells in Pediatric and Young Adult Patients with Relapsed or Refractory CD19+ Acute Lymphoblastic Leukemia

Diseases Treated:

Acute lymphoblastic leukemia

Eligibility:

 

 

View Trial

TransIT3: Study of Immune Therapy vs. Unrelated Donor Transplant for Severe Aplastic Anemia in Children

A Phase 3 Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation with Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients with Severe Aplastic Anemia

Diseases Treated:

Aplastic Anemia

Eligibility:

  • Severe aplastic anemia
  • Up to 25 years old
  • Does not have a fully matched sibling donor
  • Has at least 2 matched, unrelated donors
View Trial
 
 

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