Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®)–Health Professional Version

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Changes to This Summary (08/28/2018)

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.

Classification of Pediatric Myeloid Malignancies

Added text to state that a large retrospective study from the international Berlin-Frankfurt-Münster group demonstrated that initial therapy with an acute lymphoblastic leukemia (ALL)-type regimen was associated with a superior outcome compared with acute myeloid leukemia (AML)-type or combined ALL/AML regimens, particularly in cases with CD19 positivity or other lymphoid antigen expression. In this study, hematopoietic stem cell transplanation (HSCT) in first complete remission was not beneficial, with the possible exception of cases with morphologic evidence of persistent marrow disease after the first month of treatment (cited Hrusak et al. as reference 19).

Added introductory text and Figures 1 and 2 about the genomics of pediatric AML.

Added text to state that for patients who have FLT3-ITD, the presence of either WT1 mutations or NUP98-NSD1 fusions is associated with poorer outcome than for patients who have FLT3-ITD without these alterations. Conversely, when FLT3-ITD is accompanied by NPM1 mutations, the outcome is relatively favorable and is similar to that of pediatric AML cases without FLT3-ITD (cited Bolouri et al. as reference 49).

Added text to state that some of the FLT3 point mutations appear to be specific to pediatric patients.

Treatment of Childhood AML

Revised text to state that for the children in second remission who underwent HSCT, the 5-year overall survival (OS) was 61%, as opposed to a 5-year OS of 18% for those who did not include HSCT in their therapy.

Acute Promyelocytic Leukemia (APL)

Revised text to state that in the Children's Oncology Group (COG) AAML0631 trial, which included treatment with chemotherapy, ATRA, and arsenic trioxide, risk classification primarily defined early death risk rather than relapse risk.

Added Testi et al. as reference 41.

Added Lo Coco et al. and Esteve et al. as references 67 and 68, respectively.

Myelodysplastic Syndromes (MDS)

Added text about the results of a retrospective analysis that used a capture assay to target mutations known to predispose to marrow failure and MDS, which was performed on genomic DNA from peripheral blood mononuclear cell samples from patients undergoing stem cell transplant for MDS and aplastic anemia (cited Keel et al. as reference 11).

Juvenile Myelomonocytic Leukemia (JMML)

Added text to state that COG conducted a randomized trial in children with JMML that compared a standard-intensity preparative regimen with a reduced-intensity regimen. The trial closed to enrollment early when an interim analysis revealed a higher frequency of relapse/disease persistence in children who received the reduced-intensity regimen than in children who received the standard-intensity regimen (cited Dvorak et al. as reference 36).

Added Treatment Options Under Clinical Evaluation as a new subsection.

Chronic Myelogenous Leukemia (CML)

Added text to state that dasatinib is approved by the U.S. Food and Drug Administration (FDA) for use in children with CML. Also added text about the results of a phase II trial that included 84 children who were newly diagnosed in chronic phase with CML, which was the basis of the FDA approval (cited Gore et al. as reference 44).

Added text to state that nilotinib was approved by the FDA for children with CML in March 2018. Also added text about the results of two sponsored trials that tested the efficacy and safety of nilotinib in pediatric patients with CML (cited Novartis Pharmaceuticals Corporation and Hijiya et al. as references 45 and 46, respectively).

Added text to state that after 1 year of administration of nilotinib, 7 of 27 children converted to a major molecular response who were either intolerant or resistant to imatinib or dasatinib and not already in major molecular response.

Special Considerations for the Treatment of Children With Cancer

Added Wolfson et al. as reference 2.

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.

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