What is the role of chemotherapy in pediatric acute myelocytic leukemia (AML) treatment?

Updated: Sep 12, 2017
  • Author: Mark E Weinblatt, MD; Chief Editor: Jennifer Reikes Willert, MD  more...
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Virtually all chemotherapeutic drug regimens include some combination of an anthracycline (most often daunorubicin [daunomycin]) with cytosine arabinoside (cytarabine). Other drugs that have been administered include fludarabine, etoposide, amsacrine, dexamethasone, 6-thioguanine, cyclophosphamide, and mitoxantrone.

For many years, most children in the United States were treated with chemotherapy protocols developed by the Children’s Cancer Group and the Pediatric Oncology Group. These protocols, which used different multiagent chemotherapies, were associated with improved results as therapy was intensified. Although these treatments prolonged pancytopenia, they decreased induction failures and substantially improved disease-free survival.

After all of the pediatric national groups merged to form the Children's Oncology Group (COG), the recommended regimen, [9] based on the Medical Research Council acute myeloid leukemia trials, was adapted; this consisted of 2 cycles of induction therapy with infusions of daunomycin, cytosine arabinoside, etoposide (ADE therapy). 

In September 2017, the FDA approved gemtuzumab ozogamicin (Mylotarg) for the treatment of relapsed or refractory CD33-positive AML in patients aged 2 years and older.

Gemtuzumab ozogamicin originally received accelerated approval in May 2000 as a stand-alone treatment for relapsed CD33-positive AML in older patients, but was voluntarily withdrawn from the market after subsequent confirmatory trials failed to verify clinical benefit and demonstrated safety concerns, including a high number of early deaths. The September 2017 approval includes a lower recommended dose, a different treatment schedule and a new patient population. [10]

The International Berlin-Frankfurt-Münster (BFM) Study Group reported that children with relapsed AML who received liposomal daunorubicin (DNX) in conjunction with the FLAG regimen (fludarabine, cytarabine, and granulocyte colony-stimulating factor [G-CSF]) had improved early treatment response. [11, 12] Although overall long-term survival was similar in the 2 treatment groups, children with core-binding factor (CBF) AML who received FLAG/DNX had a 24% higher 4-year probability of survival than those who received the FLAG regimen alone. [11, 12]

Patients who are FLT3 positive can benefit from targeted agents, such as sorafenib.

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