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    GENE:

    NPM1 (Nucleophosmin 1)

    i
    Other names: Nucleophosmin (Nucleolar Phosphoprotein B23, Numatrin), Testicular Tissue Protein Li 128, Nucleolar Protein NO38, Numatrin, NPM1, Nucleophosmin 1, Nucleophosmin/Nucleoplasmin Family, Member 1, Nucleolar Phosphoprotein B23
    2d
    PEVOLAM: Study to Compare Azacitidine Plus Pevonedistat Versus Azacitidine in Patients With Acute Myeloid Leukemia Not Eligible for Standard Chemotherapy (clinicaltrials.gov)
    P3, N=302, Active, not recruiting, PETHEMA Foundation | Trial completion date: Jun 2025 --> Jul 2026
    Trial completion date
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    IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • NPM1 (Nucleophosmin 1) • CD4 (CD4 Molecule)
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    azacitidine • pevonedistat (MLN4924)
    2d
    MT2015-29: Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders (clinicaltrials.gov)
    P2, N=300, Recruiting, Masonic Cancer Center, University of Minnesota | Trial completion date: Jun 2027 --> Jun 2028 | Trial primary completion date: Jun 2026 --> Jun 2027
    Trial completion date • Trial primary completion date
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    NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A) • IKZF1 (IKAROS Family Zinc Finger 1) • CEBPA (CCAAT Enhancer Binding Protein Alpha)
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    NPM1 mutation • MLL rearrangement
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    cyclophosphamide
    3d
    Study on the Efficacy and Safety of VA Regimen Compared to "3+7" Regimen in Newly Diagnosed AML With NPM1 or IDH1/IDH2 Mutations (clinicaltrials.gov)
    P2, N=148, Not yet recruiting, Shen yang
    New P2 trial • Head-to-Head • IO biomarker
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    IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • NPM1 (Nucleophosmin 1)
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    IDH2 mutation • NPM1 mutation
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    Venclexta (venetoclax) • cytarabine • azacitidine • daunorubicin
    3d
    CD244 modulates bone marrow infiltrating CD8+ T cells and serves as a prognostic and immunotherapeutic target in acute myeloid leukemia. (PubMed, Leuk Res)
    CD244 drives immunomodulation via dysregulated CD8+T cell differentiation and cytokine secretion, positioning it as a prognostic biomarker and a promising target for immunotherapy in AML.
    Journal • IO biomarker
    |
    NPM1 (Nucleophosmin 1) • CD8 (cluster of differentiation 8) • CEBPA (CCAAT Enhancer Binding Protein Alpha) • CD27 (CD27 Molecule) • B3GAT1 (Beta-1,3-Glucuronyltransferase 1) • KLRB1 (Killer Cell Lectin Like Receptor B1) • SELL (Selectin L)
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    NPM1 mutation
    5d
    ACTIVATE: Observational Study on APL-like aCute Myeloid Leukemia: disTInct Phenotype and Early VAscular complicaTions (clinicaltrials.gov)
    P=N/A, N=220, Recruiting, Gruppo Italiano Malattie EMatologiche dell'Adulto | Not yet recruiting --> Recruiting | Initiation date: Nov 2025 --> Jul 2026
    Enrollment open • Trial initiation date
    |
    NPM1 (Nucleophosmin 1)
    |
    NPM1 mutation
    8d
    Evaluation of Prognostic Factors and Outcomes in Primary versus Secondary Myeloid Sarcoma. (PubMed, Hum Pathol)
    Outcomes appear to be influenced by an interplay of disease context, clonal architecture, and therapeutic strategy rather than individual mutations alone, underscoring the need for integrated molecular profiling and prospective studies to guide management. This study highlights that MS with MR mutations may follow different cellular pathways to evolution.
    Journal • Tumor mutational burden
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    TMB (Tumor Mutational Burden) • FLT3 (Fms-related tyrosine kinase 3) • NRAS (Neuroblastoma RAS viral oncogene homolog) • NPM1 (Nucleophosmin 1) • ASXL1 (ASXL Transcriptional Regulator 1) • TET2 (Tet Methylcytosine Dioxygenase 2)
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    TMB-H • NRAS mutation • NPM1 mutation • TMB-L • ASXL1 mutation • TET2 mutation
    8d
    Clinical research progress of menin inhibitors for acute myeloid leukemia: latest updates from the 2025 ASH Annual Meeting. (PubMed, Exp Hematol Oncol)
    Novel menin inhibitors, including revumenib, bleximenib, ziftomenib, and enzomenib, are currently under clinical evaluation, and selected updated clinical results were presented at the 2025 American Society of Hematology (ASH) Annual Meeting. This brief review summarizes the key findings and discusses the emerging clinical questions regarding combination strategies, treatment sequencing, and molecularly defined use of menin inhibitors.
    Journal
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    NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A)
    |
    NPM1 mutation • KMT2A rearrangement
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    Revuforj (revumenib) • Komzifti (ziftomenib) • bleximenib (JNJ-6617) • enzomenib (DSP-5336)
    10d
    Mutation-specific NPM1 immunohistochemistry is a sensitive method for detecting residual disease in mid- and postinduction bone marrows: comparison of NPM1 immunohistochemistry, NPM1 real-time polymerase chain reaction, and flow cytometric studies. (PubMed, Am J Clin Pathol)
    Mutation-specific NPM1 IHC is a valuable tool for a rapid assessment of residual morphologic and molecular disease in mid- and postinduction NPM1mut AML.
    Clinical • Journal • Polymerase Chain Reaction
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    KIT (KIT proto-oncogene, receptor tyrosine kinase) • NPM1 (Nucleophosmin 1) • CD34 (CD34 molecule)
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    NPM1 mutation • KIT expression
    10d
    Fine particulate matter and tobacco product exposure exacerbates metabolic syndrome-related colon cancer via regulating oxidative stress and tumor-associated macrophage interactions. (PubMed, Cancer Metab)
    Our results provided new insights into the mechanisms of intestinal cancer progresses by paracrine IR-PD-L1 signaling, which is aided by the deregulation of oxidative stress and macrophage communication brought on by smoking carcinogen-induced the metabolic syndrome.
    Journal • PD(L)-1 Biomarker • IO biomarker
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    NPM1 (Nucleophosmin 1) • IGF2 (Insulin-like growth factor 2) • IR (Insulin receptor) • SLC2A1 (Solute Carrier Family 2 Member 1)
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    PD-L1 expression
    11d
    Optimization of Analytical Performances and Routine Laboratory Implementation of NPM1 Mutation Detection by Digital PCR in the Diagnosis and Monitoring of NPM1-Mutated Acute Myeloid Leukemias. (PubMed, Int J Lab Hematol)
    The approach enabled early relapse prediction in selected patients and improved molecular follow-up, even for rare NPM1 mutations not covered by standard qPCR panels. It simplifies laboratory routine and improves MRD assessment, according to current ELN guidelines and the growing need for personalized molecular monitoring in AML.
    Journal
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    NPM1 (Nucleophosmin 1)
    |
    NPM1 mutation
    11d
    Shogaol triggers apoptosis and ferroptosis in Ewing sarcoma by targeting the polo-like kinase 1/nucleophosmin 1 axis. (PubMed, Free Radic Biol Med)
    Key findings were validated by CETSA, isothermal dose-response fingerprinting, co-immunoprecipitation, and ubiquitination assays. These results demonstrate that shogaol exerts dual cytotoxic effects through the PLK1/NPM1 axis, presenting a potential natural therapeutic approach for Ewing sarcoma.
    Journal • IO biomarker
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    BCL2 (B-cell CLL/lymphoma 2) • NPM1 (Nucleophosmin 1) • SLC3A2 (Solute Carrier Family 3 Member 2) • PLK1 (Polo Like Kinase 1) • BAX (BCL2-associated X protein) • GPX4 (Glutathione Peroxidase 4) • TRIM28 (Tripartite Motif Containing 28)