Molecular Profile Detail

Profile Name NPM1 mutant
Gene Variant Detail

NPM1 mutant (unknown)

Relevant Treatment Approaches

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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
NPM1 mutant acute myeloid leukemia not applicable N/A Clinical Study Prognostic In multiple clinical studies, an NPM1 mutation without FLT3 internal tandem duplication (ITD) mutation was associated with favorable prognosis in acute myeloid leukemia (AML) patients, compared to AML patients harboring an NPM1 mutation and FLT3 ITD mutation (PMID: 19047294, PMID: 24573385, PMID: 25713434). 25713434 19047294 24573385
NPM1 mutant acute myeloid leukemia not applicable N/A Clinical Study Prognostic In clinical analyses, mutations in NPM1, in the absence of a FLT3 ITD mutation, were associated with favorable prognosis in acute myeloid leukemia patients with normal karyotype (PMID: 26239249, PMID: 26586702, PMID: 26676635, PMID: 16109776). 26239249 26676635 16109776 26586702
NPM1 mutant acute myeloid leukemia predicted - sensitive Cytarabine + Venetoclax Phase Ib/II Actionable In a Phase I/II trial, Venclexta (venetoclax) in combination with low-dose cytarabine resulted in complete remission or complete remission with incomplete count recovery in 89% (8/9) of patients with acute myeloid leukemia harboring NPM1 mutations who were ineligible for intensive chemotherapy (ASH Annual Meeting, Dec 2018, Abstract 284; NCT02287233). detail...
NPM1 mutant acute myeloid leukemia predicted - sensitive Entospletinib Preclinical - Patient cell culture Actionable In a preclinical study, NPM1 mutations correlated with sensitivity to Entospletinib in patient-derived acute myeloid leukemia samples in an ex vivo assay (PMID: 30333627). 30333627
NPM1 mutant leukemia predicted - sensitive EPZ004777 Preclinical Actionable In preclinical study, treatment with EPZ004777 resulted in reduced proliferation and increased differentiation of murine leukemia cells expressing an NPM1 mutation, and improved survival in NPM1-mutant murine leukemia models (PMID: 27535106). 27535106
NPM1 mutant acute myeloid leukemia predicted - sensitive MI-503 Preclinical - Cell line xenograft Actionable In a preclinical study, MI-503 inhibited growth of an acute myeloid leukemia cell line and primary AML samples harboring NPM1 mutations in culture, and reduced tumor burden and improved survival in an NPM1-mutant AML cell line xenograft model (PMID: 27535106). 27535106
NPM1 mutant acute myeloid leukemia predicted - sensitive Ibrutinib Preclinical - Patient cell culture Actionable In a preclinical study, NPM1 mutations correlated with sensitivity to Imbruvica (ibrutinib) in patient-derived acute myeloid leukemia samples in an ex vivo assay (PMID: 30333627). 30333627
NPM1 mutant acute myeloid leukemia predicted - sensitive EPZ004777 Preclinical - Patient cell culture Actionable In a preclinical study, EPZ004777 decreased HOX gene expression, and reduced proliferation and increased differentiation of a human acute myeloid leukemia (AML) cell line and primary AML samples harboring NPM1 mutations in culture (PMID: 27535106). 27535106
NPM1 mutant acute myeloid leukemia sensitive NSC348884 Preclinical Actionable In a preclinical study, acute myeloid leukemia cells harboring a NPM1 mutation were sensitive to NSC348884 as demonstrated by induced apoptosis (PMID: 21719597). 21719597
NPM1 mutant acute myeloid leukemia sensitive ENMD-2076 Phase I Actionable In a Phase I trial, three acute myeloid leukemia patient harboring an NPM1 mutation demonstrated anti-leukemia activity when treated with ENMD-2076 (PMID: 27406088). 27406088
Clinical Trial Phase Therapies Title Recruitment Status
NCT01266083 Phase II WT1 vaccine Trial of a WT-1 Analog Peptide Vaccine in Patients With Acute Myeloid Leukemia (AML) or Acute Lymphoblastic Leukemia (ALL) Completed
NCT03375307 Phase II Olaparib Olaparib in Treating Patients With Metastatic or Advanced Urothelial Cancer With DNA-Repair Defects Recruiting
NCT02381548 Phase I Adavosertib + Belinostat Phase I Trial of AZD1775 and Belinostat in Treating Patients With Relapsed or Refractory Myeloid Malignancies or Untreated Acute Myeloid Leukemia Terminated
NCT03862157 Phase Ib/II Azacitidine + MLN4924 + Venetoclax Azacitidine, Venetoclax, and Pevonedistat in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia Recruiting