Combination Chemotherapy, Biological Therapy, and Bone Marrow Transplantation in Treating Patients With Acute Myeloid Leukemia
Leukemia, Neutropenia
ClinicalTrials.gov recordTREATMENT READING
Source sections for this topic, with the original link and date. Patient and professional reading are labelled separately.
Not medical advice · Not a complete treatment or trial directory
Made by Triangle.
Selected text from the National Cancer Institute, with the original link and date. Made by Triangle. This page is not an NCI PDQ summary. Source topics may be broader than an individual diagnosis.
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. Types of targeted therapy include the following:
Sources: Targeted therapy
Tyrosine kinase inhibitor therapy: This treatment blocks the enzyme, tyrosine kinase, that causes stem cells to become more white blood cells (blasts) than the body needs. Tyrosine kinase inhibitors may be used with chemotherapy drugs as adjuvant therapy (treatment given after the initial treatment, to lower the risk that the cancer will come back).
Sources: Tyrosine kinase inhibitor · enzyme · white blood cells · blasts · adjuvant therapy
Tyrosine kinase inhibitor therapy: This treatment blocks the enzyme, tyrosine kinase, that causes stem cells to become more white blood cells (blasts) than the body needs. Tyrosine kinase inhibitors may be used with chemotherapy drugs as adjuvant therapy (treatment given after the initial treatment, to lower the risk that the cancer will come back).
Imatinib, dasatinib, and nilotinib are used to treat childhood chronic myelogenous leukemia.
Sorafenib, midostaurin, and gilteritinib, which target a mutated (changed) form of a gene called FLT3, are being studied to treat childhood AML.
Sources: Sorafenib · midostaurin · gilteritinib · mutated · gene · FLT3
Trametinib is being studied in children with recurrent or refractory juvenile myelomonocytic leukemia.
Sources: Trametinib · recurrent · refractory
Monoclonal antibodies: Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells.
Sources: Monoclonal antibodies · immune system · proteins · toxins · radioactive
Monoclonal antibodies: Monoclonal antibodies are immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells.
Gemtuzumab ozogamicin is a type of monoclonal antibody that is attached to a chemotherapy drug. It is used in the treatment of AML.
Sources: Gemtuzumab ozogamicin
See Drugs Approved for Leukemia for more information.
Sources: Drugs Approved for Leukemia
This page is made by Triangle. The reading above is selected source text, not a treatment recommendation, and it is not medical advice.
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndromes caused by a specific chromosome change.
Sources: Lenalidomide · transfusions · myelodysplastic syndromes · chromosome
Arsenic trioxide and tretinoin are drugs that kill certain types of leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of acute promyelocytic leukemia.
Sources: Arsenic trioxide · tretinoin
See Drugs Approved for Acute Myeloid Leukemia for more information.
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change. It is sometimes used to treat transient abnormal myelopoiesis (TAM).
Sources: Watchful waiting · monitoring · condition · signs · symptoms
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
Supportive care is given to lessen the problems caused by the disease or its treatment. All patients with leukemia receive supportive care treatments. Supportive care may include the following:
Sources: Supportive care
Transfusion therapy: A way of giving red blood cells or platelets to replace blood cells destroyed by disease or cancer treatment. The blood may be donated from another person or it may have been taken from the patient earlier and stored until needed.
Sources: red blood cells · platelets · donated
Antifungal agents: Drugs, such as Caspofungin or fluconazole, used to prevent or treat infections caused by a fungus (a type of microorganism). This is important in the care of patients with AML.
Sources: Caspofungin · fluconazole · infections · fungus · microorganism
Drug therapy, such as antibiotics.
Sources: Drug therapy · antibiotics
Leukapheresis: A procedure in which a special machine is used to remove white blood cells from the blood. Blood is taken from the patient and put through a blood cell separator where the white blood cells are removed. The rest of the blood is then returned to the patient's bloodstream. Leukapheresis is used to treat patients with very high white blood cell counts.
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website.
Sources: NCI website
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
To learn more about side effects that begin during treatment for cancer, visit Side Effects.
Sources: side effects · Side Effects
Regular follow-up exams are very important. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following:
Sources: follow-up · late effects
Physical problems that affect the following:
Physical problems that affect the following:
Heart, lung, or endocrine function.
Bone development.
Fertility (ability to have children).
Sources: Fertility
Energy level.
Changes in mood, feelings, thinking, learning, or memory.
Second cancers (new types of cancer), such as breast cancer.
Sources: Second cancers · breast cancer
Some late effects may be treated or controlled. It is important that parents of children who are treated for AML or other blood diseases talk with their child's doctors about the effects cancer treatment can have on their child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information).
Sources: blood · Late Effects of Treatment for Childhood Cancer
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.
Sources: clinical trial · standard treatment
Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.
Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.
Sources: side effects
Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI’s clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.
Sources: clinical trials search · ClinicalTrials.gov
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
As your child goes through treatment, they will have follow-up tests or check-ups. Some tests that were done to diagnose or stage the cancer may be repeated to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.
Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your child's condition has changed or if the cancer has recurred (come back).
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
For information about the treatments listed below, see the Treatment Option Overview section.
Sources: Treatment Option Overview
Treatment of newly diagnosed childhood acute myeloid leukemia (AML) during the induction phase may include the following:
Sources: diagnosed · acute myeloid leukemia · induction
Combination chemotherapy.
Sources: Combination chemotherapy
Central nervous system prophylaxis therapy with intrathecal chemotherapy.
Sources: Central nervous system prophylaxis · therapy · intrathecal chemotherapy
Targeted therapy with a monoclonal antibody (gemtuzumab ozogamicin).
Sources: Targeted therapy · monoclonal antibody · gemtuzumab ozogamicin
A clinical trial of targeted therapy (sorafenib).
Sources: clinical trial · sorafenib
A clinical trial of chemotherapy and targeted therapy (gilteritinib).
Sources: chemotherapy · gilteritinib
Supportive care.
Sources: Supportive care
Treatment of childhood AML during the remission phase (consolidation/intensification therapy) depends on the subtype of AML and may include the following:
Sources: remission · consolidation · intensification therapy
Combination chemotherapy.
High-dose chemotherapy followed by using blood stem cells from a donor.
Sources: High-dose chemotherapy · Source reference · blood stem cells · donor
Targeted therapy (such as FLT3 inhibitors).
Sources: FLT3
Treatment of refractory childhood AML may include the following:
Sources: refractory
Chemotherapy.
Targeted therapy with a monoclonal antibody (gemtuzumab ozogamicin).
Radiation therapy to treat a myeloid sarcoma that does not completely respond to chemotherapy.
Sources: Radiation therapy · myeloid sarcoma
Treatment of recurrent childhood AML may include the following:
Sources: recurrent
Combination chemotherapy.
Targeted therapy with a monoclonal antibody (gemtuzumab ozogamicin).
Radiation therapy to treat locally recurring myeloid sarcoma.
A clinical trial of targeted therapy (midostaurin, sorafenib, or gilteritinib).
Sources: midostaurin
Combination chemotherapy and stem cell transplant, for patients who have had a second complete remission.
A second stem cell transplant, for patients whose disease came back after the first stem cell transplant.
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
For information about the treatments listed below, see the Treatment Option Overview section.
Sources: Treatment Option Overview
Transient abnormal myelopoiesis (TAM) usually goes away on its own. For newly diagnosed TAM that does not go away on its own or causes other health problems, treatment may include the following:
Sources: Transient abnormal myelopoiesis · diagnosed
Supportive care, including transfusion therapy or leukapheresis.
Sources: Supportive care · transfusion · leukapheresis
Chemotherapy.
Sources: Chemotherapy
Treatment of newly diagnosed acute myeloid leukemia (AML) in children aged 4 years or younger who have Down syndrome may include the following:
Sources: acute myeloid leukemia · Down syndrome
Combination chemotherapy plus central nervous system prophylaxis therapy with intrathecal chemotherapy.
Sources: Combination chemotherapy · central nervous system prophylaxis · intrathecal chemotherapy
A clinical trial of a new chemotherapy regimen that depends on how the child responds to initial chemotherapy.
Sources: clinical trial · regimen · responds
Treatment of newly diagnosed AML in children older than 4 years who have Down syndrome may be the same as treatment for children without Down syndrome.
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
For information about the treatments listed below, see the Treatment Option Overview section.
Sources: Treatment Option Overview
Treatment of newly diagnosed childhood acute promyelocytic leukemia (APL) may include the following:
Sources: diagnosed · acute promyelocytic leukemia
Tretinoin with arsenic trioxide.
Sources: Tretinoin · arsenic trioxide
Tretinoin plus chemotherapy with or without arsenic trioxide.
Sources: chemotherapy
Supportive care.
Sources: Supportive care
Treatment of childhood APL during the remission phase (consolidation/intensification therapy) may include the following:
Sources: remission · consolidation · intensification therapy
Tretinoin with arsenic trioxide.
Tretinoin plus chemotherapy with or without arsenic trioxide.
Treatment of childhood APL during the remission phase (maintenance therapy) may include the following:
Sources: maintenance therapy
Tretinoin with combination chemotherapy.
Sources: combination chemotherapy
Treatment of recurrent childhood APL may include the following:
Sources: recurrent
Arsenic trioxide therapy with or without tretinoin.
Targeted therapy with a monoclonal antibody (gemtuzumab ozogamicin).
Sources: Targeted therapy · monoclonal antibody · gemtuzumab ozogamicin
Stem cell transplant using blood stem cells from the patient or a donor.
Sources: Stem cell transplant · blood stem cells · donor
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
For information about the treatments listed below, see the Treatment Option Overview section.
Sources: Treatment Option Overview
Treatment of newly diagnosed juvenile myelomonocytic leukemia (JMML) may include the following:
Sources: diagnosed · juvenile myelomonocytic leukemia
Combination chemotherapy followed by stem cell transplant.
Sources: Combination chemotherapy · stem cell transplant
Treatment of refractory or recurrent childhood JMML may include the following:
Sources: refractory · recurrent
A second stem cell transplant, for patients whose disease came back after the first stem cell transplant.
A clinical trial of targeted therapy with a tyrosine kinase inhibitor (trametinib).
Sources: clinical trial · targeted therapy · tyrosine kinase inhibitor · trametinib
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
For information about the treatments listed below, see the Treatment Option Overview section.
Sources: Treatment Option Overview
Treatment of newly diagnosed chronic myelogenous leukemia (CML) may include the following:
Sources: diagnosed · chronic myelogenous leukemia
Targeted therapy with a tyrosine kinase inhibitor (imatinib, dasatinib, or nilotinib).
Sources: Targeted therapy · tyrosine kinase inhibitor · imatinib · dasatinib · nilotinib
A clinical trial to study the rate of CML recurrence after stopping tyrosine kinase inhibitor therapy, and the ability to achieve remission again if CML recurs in children with CML who are in remission for at least 2 years after receiving treatment with the same tyrosine kinase inhibitor for at least 3 years.
Sources: clinical trial · recurrence · remission
Treatment of refractory or recurrent childhood CML may include the following:
Sources: refractory · recurrent
Targeted therapy with a tyrosine kinase inhibitor (dasatinib or nilotinib).
Stem cell transplant using blood stem cells from a donor.
Sources: Stem cell transplant · blood stem cells · donor
Source: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Patient Version, National Cancer Institute.
Source updated: June 5, 2024. Captured 2026-09-09.
For information about the treatments listed below, see the Treatment Option Overview section.
Sources: Treatment Option Overview
Treatment of newly diagnosed childhood myelodysplastic syndromes (MDS) may include the following:
Sources: diagnosed · myelodysplastic syndromes
Stem cell transplant using blood stem cells from a donor.
Sources: Stem cell transplant · blood stem cells · donor
Supportive care, including transfusion therapy and antibiotics.
Sources: Supportive care · transfusion · therapy · antibiotics
Steroid therapy.
Sources: Steroid therapy
Lenalidomide therapy, for patients with certain gene changes.
Sources: Lenalidomide · therapy · gene
If the MDS becomes acute myeloid leukemia (AML), treatment will be the same as treatment for newly diagnosed AML.
Sources: acute myeloid leukemia · diagnosed
Leukemia, Neutropenia
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ClinicalTrials.gov recordLeukemia, Myelodysplastic Syndromes
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ClinicalTrials.gov recordLeukemia
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ClinicalTrials.gov recordAcute Myeloid Leukemia in children and adolescents
Infant Acute Biphenotypic Leukemia
Infant Acute Undifferentiated Leukemia
Treatment of Recurrent or Refractory Childhood AML
Refractory Childhood AML (Induction Failure)