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Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)

Health Professional Version
Last Modified: 05/23/2014

Changes to This Summary (05/23/2014)

The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.

General Information

Revised text to state that nonsyndromic genetic susceptibility to AML is also being studied; for example, homozygosity for a variant IKZF1 allele has been associated with increased risk of infant AML.

Classification of Pediatric Myeloid Malignancies

Revised the list of molecular abnormalities associated with favorable prognosis to state that the t(8;21) translocation occurs in approximately 12% of children with AML. Revised text to state that in AML with t(15;17), less common translocations involving the retinoic acid receptor alpha can also result in APL and that identification of cases with the t(11;17)(q23;q21) is important because of their decreased sensitivity to all-trans retinoic acid.

Added text to the list of molecular abnormalities associated with an unfavorable prognosis to state that for APL, FLT3-ITD and point mutations occur in 30% to 40% of children and adults (cited Abu-Duhier et al. as reference 81). Also revised text to state that it remains unclear whether FLT3 mutations are associated with poorer prognosis in patients with APL who are treated with modern therapy that includes all-trans retinoic acid and arsenic trioxide (cited Schnittger et al. as reference 85).

Revised the list of other molecular abnormalities observed in pediatric AML to state that an MLL gene rearrangement occurs in approximately 20% of children with AML. Revised text to state that the t(6;9) subgroup of AML occurs infrequently in children and accounts for less than 1% of AML cases; also added text to state that the median age of children with DEK-NUP214 AML is 10 to 11 years, and approximately 40% of pediatric patients have FLT3-ITD (cited Sandahl et al. as reference 100). Also revised text to state that t(6;9) AML appears to be associated with a high risk of treatment failure in children, particularly for those not proceeding to allogeneic stem cell transplantation. Revised text to state that initial reports have demonstrated that CBFA2T3-GLIS2 is a fusion product present in approximately 2% of pediatric AML.

Revised text about the World Health Organization classification of myeloid and lymphoid neoplasms with eosinophilia and abnormalities of PDGFRA (4q12), PDGFRB (5q33.2), or FGFR1 (8p11.2).

Added text to state that recurrent mutations in SETBP1 or JAK3 have been identified in addition to RAS pathway mutations in a proportion of juvenile myelomonocytic leukemia (JMML) cases (16%); these mutations were generally subclonal and are thus thought to be secondary mutations; there was a trend towards worsened overall survival for cases with these mutations (cited Sakaguchi as reference 189).

Treatment Overview for AML

Added text to the list of prognostic factors in childhood AML to state that patients with high presenting leukocyte counts have a higher risk of developing pulmonary and central nervous system complications and a higher risk of induction death (cited Sung et al as reference 30). Tevised text to state that early response to therapy can be assessed by fluorescence in situ hybridization.

Revised text to state that standard-risk cytogenetics is defined by the absence of either low-risk or high-risk cytogenetic characteristics.

Treatment of Newly Diagnosed AML

Added text to state that approximately 3% of patients die during induction chemotherapy (cited Cooper et al. as reference 8).

JMML

Added text to state that recurrent mutations in SETBP1 or JAK3 have been identified in addition to RAS pathway mutations in a proportion of JMML cases (16%); these mutations were generally subclonal and are thus thought to be secondary mutations; there was a trend towards worsened overall survival for cases with these mutations.

Recurrent Childhood AML and Other Myeloid Malignancies

Cited Shukla et al. as reference 12 and level of evidence 2Div.

Added text to state that a large prospective cohort study that included children and adults with myeloid diseases showed comparable or superior outcome with busulfan-based regimens compared with total-body irradiation.

This summary is written and maintained by the PDQ Pediatric Treatment Editorial Board, which is editorially independent of NCI. The summary reflects an independent review of the literature and does not represent a policy statement of NCI or NIH. More information about summary policies and the role of the PDQ Editorial Boards in maintaining the PDQ summaries can be found on the About This PDQ Summary and PDQ NCI's Comprehensive Cancer Database pages.