What is the prognosis of pediatric acute myelocytic leukemia (AML)?

Updated: Sep 12, 2017
  • Author: Mark E Weinblatt, MD; Chief Editor: Jennifer Reikes Willert, MD  more...
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With an overall survival rate of 45-60%, the prognosis for children with acute myeloid leukemia has improved significantly since the late 20th century.

A Japanese consortium reported an overall 5-year survival rate of 62%. [4] The long-term, disease-free survival rate is approximately 65% for patients receiving human leukocyte antigen (HLA)–matched stem cell transplants from family donors, but, as with chemotherapy, this rate is lower in high-risk patients. When patients die during treatment or after relapse, the cause is most commonly infection, bleeding, or refractory disease.

A 2012 study from Japan confirmed the results of the AML99 trial for newly diagnosed pediatric patients with AML with a 5-year overall survival (OS) of 75.6% and event-free survival (EFS) of 61.6%. This group compared their results to another cohort of newly diagnosed AML patients and found their results to be the same as the original AML99 trial with 5-year OS of 77.7% and EFS of 66.7%. Interestingly, the 5-year EFS in patients with a normal karyotype was lower compared to the original AML99 trial. [5]

For children with Down syndrome, current outcomes favor younger children, with a survival rate of 84-86% for children younger than age 2 years, 79% for children aged 2-4 years, and only 33% for children older than age 4 years. [6]

Acute promyelocytic leukemia prognosis has an event-free survival rate of 70-80%, with overall survival close to 90%. [7]

A study by Klco et al looked to determine whether genomic approaches can provide novel prognostic information for adult patients with de novo AML. The study found that although comprehensive genomic data from the patients did not improve outcome assessment, the detection of persistent leukemia-associated mutations in at least 5% of bone marrow cells in day 30 remission samples was associated with a significantly increased risk of relapse, and reduced overall survival. [8]

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