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    Association details:
    Biomarker:PTPN11 mutation
    Cancer:Juvenile Myelomonocytic Leukemia
    Drug:Mekinist (trametinib) (MEK inhibitor)
    Direction:Sensitive
    Evidence:
    Evidence Level:
    Sensitive: C2 – Inclusion Criteria
    Source:
    clinicaltrials.gov
    Title:

    Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

    Excerpt:
    ......
    Trial ID:
    ADVL1521
    Evidence Level:
    Sensitive: C4 – Case Studies
    Source:
    Mol Ther
    Title:

    Potential clinical use of azacitidine and MEK inhibitor combination therapy in PTPN11-mutated juvenile myelomonocytic leukemia

    Published date:
    02/03/2023
    Excerpt:
    Finally, we report two patients with JMML and PTPN11 mutations treated with 5-Aza, trametinib, and chemotherapy who experienced a clinical response because of the combination treatment.
    Secondary therapy:
    Chemotherapy + azacitidine
    DOI:
    10.1016/j.ymthe.2023.01.030