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

Health Professional Version
Last Modified: 04/02/2014

Changes to This Summary (04/02/2014)

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

Added text to state that nonsyndromic genetic susceptibility to AML is also being studied; for example, homozygosity for a specifc IKZF1 polymorphism has been associated with an increased risk of infant acute myeloid leukemia (AML) (cited Ross et al. as reference 16).

Classification of Pediatric Myeloid Malignancies

Revised text to state that the French-American-British classification system, which has been replaced by the World Health Organization (WHO) system, categorized AML into major subtypes primarily based on morphology and immunohistochemical detection of lineage markers.

Added Johnston et al. as reference 63, Sung et al. as reference 82, and Shiba et al. as reference 99.

Treatment Overview for Acute Myeloid Leukemia (AML)

Added Pession et al. as reference 29.

Treatment of Newly Diagnosed AML

Added Pession et al. as reference 11.

Postremission Therapy for AML

Added text to state that the optimal preparative regimen and source of donor cells for transplant in first complete remission has not been determined (cited Liu et al. and Bredeson et al. as references 23 and 24, respectively).

Acute Promyelocytic Leukemia (APL)

Added Lehmann et al. and Park et al. as references 8 and 9, respectively.

Added text to state that initiation of all-trans retinoic acid (ATRA) therapy is strongly recommended as soon as APL is suspected based on morphological and clinical presentation, because ATRA has been shown to ameliorate bleeding risk for patients with APL (cited Breen et al. and Visani et al. as references 10 and 11, respectively). Also added text to state that retrospective analysis identified an increase in early death due to hemorrhage in patients with APL in whom ATRA introduction was delayed (cited Altman et al. as reference 12).

Juvenile Myelomonocytic Leukemia (JMML)

Added text to state that there are only a small number of mutations in the leukemia cells of patients with JMML, with exome sequencing identifying approximately one nonsilent mutation per case. Also revised text to state that approximately 75% of JMML cases harbor one of three mutually exclusive mutations leading to activated RAS signaling, including direct oncogenic RAS mutations (approximately 20%–30%), NF1 inactivating mutations (approximately 10%–25%), or protein tyrosine phosphatase, nonreceptor type 11 (PTPN11) (SHP-2) mutations (approximately 35%–40%) (cited Sakaguchi et al. as reference 9).

Added text to state that cord blood transplantation results in a 5-year disease-free survival of 44%, with improved outcome in children younger than 1.4 years at diagnosis, those with non-monosomy 7 karyotype, and those receiving 5/6 to 6/6 HLA matched cord units; this suggests that cord blood can provide an additional donor pool for this group of children (cited Locatelli et al. as reference 30 and level of evidence 3iiDii).

Added Treatment Options Under Clinical Evaluation as a new subsection.

Recurrent Childhood AML and Other Myeloid Malignancies

Added text to state that a number of studies, including a large, prospective Center for International Blood and Marrow Transplant Research cohort study of children and adults with myeloid diseases, have shown similar or superior survival with busulfan-based regimens compared with total-body irradiation (cited Bredeson et al. as reference 19).

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.