Skip to content

Cancer Health Center

Font Size

Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Myelodysplastic Syndromes

The myelodysplastic syndromes (MDS) and myeloproliferative syndromes (MPS), which represent between 5% and 10% of all myeloid malignancies in children, are a heterogeneous group of disorders with the former usually presenting with cytopenias and the latter with increased peripheral white blood cell (WBC), red blood cell, or platelet counts. MDS is characterized by ineffective hematopoiesis and increased cell death, while MPS is associated with increased progenitor proliferation and survival. Because they both represent disorders of very primitive, multipotential hematopoietic stem cells, curative therapeutic approaches nearly always require allogeneic stem cell transplantation.

Patients usually present with signs of cytopenias, including pallor, infection, or bruising. The bone marrow is usually characterized by hypercellularity and dysplastic changes in myeloid precursors. Clonal evolution eventually can lead to the development of acute myeloid leukemia (AML). The percentage of abnormal blasts is less than 20%. The less common, hypocellular MDS, can be distinguished from aplastic anemia in part by its marked dysplasia, clonal nature, and higher percentage of CD34-positive precursors.[1,2]

Recommended Related to Cancer

General Information

Cannabis, also known as marijuana, originated in Central Asia but is grown worldwide today. In the United States, it is a controlled substance and is classified as a Schedule I agent (a drug with increased potential for abuse and no known medical use). The Cannabis plant produces a resin containing psychoactive compounds called cannabinoids. The highest concentration of cannabinoids is found in the female flowers of the plant.[1]Clinical trials conducted on medicinal Cannabis are limited. The...

Read the General Information article > >

Although the etiology of MDS has not been elucidated, clues have begun to be defined. For instance, approximately 20% of malignant myeloid disorders, including MDS, in adults have been shown to have mutations in the TET2 gene.[3] Other genes shown to be mutated in MDS include EZH2, DNMT3A, ASXL1, IDH1/2, RUNX1, ETV6/TEL, and TP53. Most of these genes are key elements of epigenetic regulation of the genome and affect DNA methylation and/or histone modification.[3,4,5] Mutations in proteins involved in RNA splicing have been described in 45% to 85% of MDS.[6] MDS in both adults and children has been shown to have aberrant DNA methylation patterns and approximately one-half of cases are characterized by hypermethylation of the promoters for the CDKN2B and CALC genes, both of which play roles in cell cycle regulation.[7,8] Inherited disorders, such as Fanconi anemia, due to germline mutations in DNA repair genes, or in dyskeratosis congenita, due to mutations in genes regulating telomere length, have significantly increased risk of developing MDS.[9] Additional bone marrow failure syndromes may also evolve into MDS, including those due to mutations in genes encoding ribosome associated proteins, such as Shwachman-Diamond syndrome and Diamond-Blackfan anemia.[9] The 15-year cumulative risk of MDS in patients with severe congenital neutropenia, also know as Kostmann syndrome, which is due to mutations in the gene encoding elastase, has been estimated to be 15% with an annual risk of MDS/AML of 2% to 3%; how mutations affecting this protein as well as what role the chronic exposure of granulocyte-colony stimulating factor (G-CSF) contribute to the development of MDS is unclear.[10,11] Inherited mutations in the RUNX1 or CEPBA genes have also been shown to be associated with familial MDS/AML.[12,13]

    1|2|3|4|5
    Next Article:

    Today on WebMD

    Colorectal cancer cells
    A common one in both men and women.
    Lung cancer xray
    See it in pictures, plus read the facts.
     
    sauteed cherry tomatoes
    Fight cancer one plate at a time.
    Ovarian cancer illustration
    Do you know the symptoms?
     
    Jennifer Goodman Linn self-portrait
    Blog
    what is your cancer risk
    HEALTH CHECK
     
    colorectal cancer treatment advances
    Video
    breast cancer overview slideshow
    SLIDESHOW
     
    prostate cancer overview
    SLIDESHOW
    lung cancer overview slideshow
    SLIDESHOW
     
    ovarian cancer overview slideshow
    SLIDESHOW
    Actor Michael Douglas
    Article