Treatments for recurrent or refractory childhood AML

Recurrent (relapsed) childhood acute myelogenous leukemia (AML) means that the cancer has come back after treatment and first remission. Primary refractory disease means that the cancer was resistant to or didn’t respond to the first treatment and the disease didn’t go into remission.

Treatment for recurrent or refractory childhood AML depends on:

  • the treatment that the child received before
  • how much time has passed since treatment
  • the subtype of AML
  • if there is a matched stem cell donor

Treatment for recurrent childhood AML usually includes giving drugs similar to those used in initial induction chemotherapy. Once remission is achieved with these drugs, a stem cell transplant may be used.

Treatment for refractory childhood AML often involves trying a different combination of chemotherapy drugs.


The most common combination chemotherapy drugs used for recurrent childhood AML are high-dose cytarabine (Cytosar) and one or more of the following:

  • mitoxantrone
  • fludarabine (Fludara) and idarubicin (Idamycin)
  • asparaginase (Kidrolase), pegaspargase (Oncaspar) or asparaginase erwinia (Erwinase)
  • etoposide (Vepesid, VP-16) and clofarabine (Clolar)

Find out more about chemotherapy for childhood leukemia.

Stem cell transplant

A stem cell transplant may be used after the child achieves remission from chemotherapy. Some doctors may recommend a stem cell transplant even if a second remission cannot be achieved with chemotherapy.

Find out more about stem cell transplants for childhood leukemia.

Targeted therapy

Targeted therapy uses drugs to target specific molecules (such as proteins) on cancer cells or inside them. These molecules help send signals that tell cells to grow or divide. By targeting these molecules, the drugs stop the growth and spread of cancer cells and limit harm to normal cells.

Children that have recurrent or refractory AML may be treated with monoclonal antibodies, such as gemtuzumab ozogamicin. This drug is given through a needle in a vein (called intravenous infusion).

Another targeted therapy drug that may be used is sorafenib (Nexavar) for children with a FLT3 mutation.

Find out more about targeted therapy for childhood leukemia.

Clinical trials

Children with cancer may be treated in a clinical trial. Clinical trials look at new ways to prevent, find and treat cancer. Find out more about clinical trials.

Expert review and references

  • American Cancer Society. Childhood leukemia. Atlanta, GA: American Cancer Society; 2012.
  • Montoya, L . Myeloid diseases. Baggott C, Fochtman D, Foley GV & Patterson Kelly, K (eds.). Nursing Care of Children and Adolsecents with Cancer and Blood Disorders. 4th ed. APHON; 2011: 26: pp.967-986.
  • National Cancer Institute. Childhood Acute Myeloid Leukemia/Other Myeloid MalignanciesTreatment (PDQ®) Health Professional Version. Bethesda, MD: National Cancer Institute; 2012.
  • National Cancer Institute. Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®) Patient Version. 2018.
  • Weinblatt, M. E. et al . Pediatric acute myelocytic leukemia. WebMD LLC; 2011.

Medical disclaimer

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