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    Association details:
    Biomarker:SETBP1 mutation + NRAS mutation
    Cancer:Juvenile Myelomonocytic Leukemia
    Drug:Mekinist (trametinib) (MEK inhibitor)
    Direction:Sensitive
    Evidence:
    Evidence Level:
    Sensitive: D – Preclinical
    Source:
    Leukemia
    Title:

    Mutant SETBP1 enhances NRAS-driven MAPK pathway activation to promote aggressive leukemia

    Published date:
    05/17/2021
    Excerpt:
    In this study, we found that when co-expressed with Ras pathway mutations, SETBP1 promoted oncogenic transformation of murine bone marrow in vitro and aggressive myeloid leukemia in vivo...Cells expressing NRAS and SETBP1 are sensitive to inhibitors of the MAPK pathway, and treatment with the MEK inhibitor trametinib conferred a survival benefit in a mouse model of NRAS/SETBP1-mutant disease.
    DOI:
    10.1038/s41375-021-01278-2
    Evidence Level:
    Sensitive: D – Preclinical
    Source:
    ASH 2019
    Title:

    SETBP1 Mutations Accelerate NRAS-Mutant Leukemia

    Published date:
    11/06/2019
    Excerpt:
    Trametinib, an inhibitor of this pathway, doubles overall survival in our murine model of NRAS/SETBP1-mutant leukemia, thus providing encouraging pre-clinical data for the use of trametinib in SETBP1-mutant disease.
    DOI:
    10.1182/blood-2019-125125