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Acute Myeloid Leukemia (AML)

Also called: AML, ANLL, acute myelocytic leukemia, acute myelogenous leukemia, acute granulocytic leukemia, acute non-lymphocytic leukemia

What is acute myeloid leukemia?

In acute myeloid leukemia (AML), white blood cells, produced in bone marrow, are abnormal and do not become healthy cells. These abnormal cells crowd out the normal ones, so the patient’s body has a harder time fighting off infection.

How common is acute myeloid leukemia?

  • Only about 500 children are found to have AML in the U.S. each year. (It is much more common in adults.) However, it is the most common second cancer among children treated for other cancers.
  • The number of children with AML is about equal between boys and girls and between whites and African-Americans.
  • AML is also seen more often in people who are exposed to large amounts of radiation and some kinds of chemicals.

What are the symptoms of acute myeloid leukemia?

If your child has AML, the following symptoms may be present:

  • Fever
  • Infection
  • Easy bruising and bleeding
  • Frequent nosebleeds
  • Bleeding that is hard to stop, even from a small cut
  • Pain in bones or joints
  • Swollen glands
  • Poor appetite

How is acute myeloid leukemia treated?

Chemotherapy (“chemo”) is the primary AML treatment:

  • 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.

Doctors look at several factors to decide which medications to use and how aggressive treatment should be. Some of these factors include the child’s age and the child’s white blood cell count before treatment.

Expect your child’s AML treatment to include two phases:

  • Induction - to kill the leukemia cells in the blood and bone marrow and put the disease into remission (a return to normal blood cell counts)
    • Central nervous system (CNS) sanctuary therapy (also called CNS prophylaxis) - may also be given during this time to kill leukemia cells that remain in the brain and spinal cord after standard chemo. With this treatment, chemo medications are injected into the fluid-filled space between the thin layers of tissue that cover the brain and spinal cord.
  • Consolidation/intensification/post-induction - to rid the body of any remaining cells that could begin to grow and cause the leukemia to return (relapse). This begins after remission has been confirmed and may continue for two or three more years.

St. Jude investigators have pioneered methods to detect a very small number of leukemia cells that stay in the bone marrow after the completion of the induction therapy. These methods can detect a single AML cell among 1,000 normal cells. Children who have more than one cell in 1,000 after completing the induction phase are at the greatest risk of relapsing.

  • Stem cell transplant - may be used for a child who is at high risk of relapse or who has AML that is resistant to other treatments. A stem cell transplant includes replacing blood-forming cells in the bone marrow that have been killed by chemo and/or radiation therapy:
    • A stem cell transplant gives the patient new blood cells from a donor’s blood or bone marrow. These cells grow into healthy blood cells to replace the ones the child lost.
    • Some types of stem cell transplants may be called “bone marrow transplants” because the cells come from the donor’s bone marrow.
  • Immunotherapy - uses specific types of cells called natural killer (NK) cells. These cells are obtained from parents and are given to patients with the goal of eliminating leukemia cells.
    • NK cells are harvested from the blood of one of the parents. The cells are then infused into the child’s blood.
    • The NK cells circulate in the child’s body and recognize and attach to the leukemia cell wall.
    • NK cells damage the cell walls of the AML cells, leading to their destruction.
  • Radiation therapy - uses high-energy X-rays or other types of radiation to kill cancer cells or stop them from growing. This is rarely used in AML treatment.

What are the survival rates for acute myeloid leukemia?

  • About 90 percent of children with AML have no cancer cells in their blood after induction. However, AML later returns in about 20 percent of these cases.
  • Five-year survival rates range from 65 to 75 percent.

Why choose St. Jude for your child’s leukemia treatment?

  • 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 hospital’s leukemia studies have pioneered the way the world treats childhood leukemia.
  • St. Jude pioneered outpatient clinical trials for children with leukemia, reducing the need for inpatient stays.
  • St. Jude scientists are using new forms of treatment to improve the outcome of AML. Current clinical trials include:
    • Treatment based on the specific subtype of AML and on the response to therapy: intensive use of chemo plus stem cell transplant
    • Checking for minimal residual disease by flow-cytometric and molecular techniques
    • Clofarabine, sorafenib and other new drugs for the treatment of AML
    • Natural killer cell transplantation
    • Using new methods to look for genetic defects in AML

Associated Clinical Trials

ALTE1631: Web-based Exercise Study for Children and Adolescents with Cancer

Web-based Physical Activity Intervention among Children and Adolescents with Cancer

Diseases Treated:

leukemia, solid tumor, brain tumor, lymphoma, carcinoma


This is a non-therapeutic clinical trial open to patients receiving treatment at a Children’s Oncology Group (COG)-affiliated institution.

  • At least 8 years old and younger than 16 years old
  • Diagnosed with childhood cancer, in remission
  • Completed therapy within the past 12 months
  • Performance status corresponding to ECOG scores of 0, 1, 2
  • Fewer than 420 minutes of moderate to vigorous physical activity (MVPA) over the last week
  • Able to write and read English, Spanish or French (patient and at least one parent/guardian)
  • Not pregnant
View Trial

AML23: Clinical Trial Studying the Safety of Using Venetoclax and Chemotherapy to Treat Newly Diagnosed Childhood AML

A Collaborative Phase 2 Study of Venetoclax in Combination with Conventional Chemotherapy in Pediatric Patients with Acute Myeloid Leukemia

Diseases Treated:

Acute Myeloid Leukemia (AML)


  • Diagnosed with AML
  • Older than 28 days and younger than 22 years
  • No prior AML treatment
View Trial

APAL2020D: Venetoclax in Children with Relapsed Acute Myeloid Leukemia (AML)

A Randomized Phase 3 Trial of Fludarabine/Cytarabine/Gemtuzumab Ozogamicin With or Without Venetoclax in Children With Relapsed AML

Diseases Treated:

Acute Myeloid Leukemia (AML)


  • Diagnosis of relapsed acute myeloid leukemia (AML)
  • Ages 29 days old to 21 years old
  • Have had AML come back for the first or second time
  • May have heart problems that prevent them from taking anthracyclines
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


  • 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:


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


View Trial

CPXSMN: CPX-351 in Pediatric Patients with Secondary Myeloid Neoplasms

A Prospective, Multicenter, Single-Arm Pilot Study of CPX-351 (VYXEOS) in Individuals < 22 Years with Secondary Myeloid Neoplasms

Diseases Treated:


  • 1–22 years old
  • Treatment-related or secondary MDS/AML 
View Trial

DIRECT70: CAR T–Cell Therapy for Children with Blood Malignancies

DIRECT70: CAR T–Cell Therapy Directed to CD70 for Pediatric Patients with Hematological Malignancies (DIRECT70)

Diseases Treated:



  • Up to 21 years old
  • CD70+ acute myeloid leukemia, acute lymphoblastic leukemia (B-ALL, T-ALL), lymphoma, or myelodysplastic syndrome
  • If prior allogeneic hematopoietic cell transplant, no graft vs. host disease
  • Has an identified hematopoietic cell transplantation donor
  • Has adequate organ function
View Trial

DSCOG: Learning, Behavior and Social Skills in Survivors of Childhood Leukemia with Down syndrome

Neurocognitive and Psychosocial Outcomes in Survivors of Childhood Leukemia with Down syndrome

Diseases Treated:



This is a non-therapeutic clinical trial that is open only to patients who were treated at St. Jude Children’s Research Hospital since 1980 and are currently followed at the hospital.

  • St. Jude Children’s Research Hospital patient with acute leukemia (i.e., ALL or AML)
  • Trisomy 21 Down syndrome diagnosis
  • Completed all cancer therapy at St. Jude since 1980 and at least six months prior to study visit
  • English as the primary language
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:


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

HAPNK1: Blood and Marrow Transplant (BMT) for Children with Leukemia/Lymphoma

Haploidentical Donor Hematopoietic Progenitor Cell and Natural Killer Cell Transplantation with a TLI-Based Conditioning Regimen in Patients with Hematologic Malignancies

Diseases Treated:

Leukemia and lymphoma


  • 21 years and younger
  • Does not have a suitable HLA-matched sibling donor or volunteer HLA-matched unrelated donor or is not a candidate for conventional matched donor transplant due to refractory disease
  • Has a suitable single haplotype-matched family member donor
  • High-risk hematologic malignancy, including certain diagnoses of:
    • Acute lymphoblastic leukemia (ALL)
    • Acute myeloid leukemia (AML)
    • Chronic myelogenous leukemia (CML)
    • Myelodysplastic syndrome (MDS)
    • Hodgkin lymphoma
    • Non-Hodgkin lymphoma
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


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

PAINBDY1: Treating Pain in Children with Cancer: Pain Buddy

Treating Pain in Children with Cancer: A 21st Century Innovative Approach (Pain Buddy)

Diseases Treated:

Endocrine Tumors
Ewing Sarcoma


This is a research study open only to St. Jude patients and their caregivers.

  • 8 to 18 years old
  • Within 16 weeks of initial cancer diagnosis
  • Receiving outpatient chemotherapy treatment for cancer
  • Can speak, read and write English. Parents who can speak, read, and write in English and Spanish
  • Have Internet access
View Trial

RAVAML: Revumenib, Azacitidine, and Venetoclax in Acute Myeloid Leukemia

A Phase 1 Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients with Refractory or Relapsed Acute Myeloid Leukemia

Diseases Treated:

Acute Myeloid Leukemia (AML)
Acute Leukemia of Ambiguous Lineage (ALAL)


  • 1–30 years old
  • Acute myeloid leukemia or acute leukemia of ambiguous lineage
  • Specific genetic changes
  • Adequate organ function
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:


  • 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

SELCLAX: A Study of Venetoclax and Selinexor with Chemotherapy in Children with AML

A Phase 1 Study of Venetoclax and Selinexor in Combination with Chemotherapy in Pediatric Patients with Refractory or Relapsed Acute Myeloid Leukemia

Diseases Treated:

Acute myeloid leukemia


  • Diagnosis of one of the following:
    • Relapsed or refractory acute myeloid leukemia (AML)
    • Relapsed or refractory acute leukemia of ambiguous lineage (ALAL)
  • 30 years old or younger (24 years or younger at St. Jude)
  • Ineligible for other curative therapy
View Trial

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