Molecular Profile Detail

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Profile Name KMT2A - AFF1
Gene Variant Detail

KMT2A - AFF1 (gain of function)

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
KMT2A - AFF1 leukemia sensitive I-BET151 + SGC0946 Preclinical - Cell culture Actionable In a preclinical study, I-BET151 and SGC0946 synergistically inhibited proliferation of leukemia cells harboring KMT2A-AFF1 in culture (PMID: 27294782). 27294782
KMT2A - AFF1 acute myeloid leukemia sensitive Dinaciclib Preclinical Actionable In a preclinical study, treatment with Dinaciclib resulted in induced cell death of acute myeloid leukemia cells harboring KMT2A-AFF1 in culture (PMID: 26627013). 26627013
KMT2A - AFF1 acute myeloid leukemia predicted - sensitive AZD5153 Preclinical - Cell line xenograft Actionable In a preclinical study, AZD5153 inhibited proliferation of acute myeloid leukemia cells harboring KMT2A-AFF1 (MLL-AF4) in culture, resulted in tumor growth inhibition in cell line xenograft models (PMID: 27573426). 27573426
KMT2A - AFF1 acute myeloid leukemia sensitive MI-503 Preclinical - Cell line xenograft Actionable In a preclinical study, MI-503 reduced expression of KMT2A (MLL) fusion target genes, inhibited growth of an acute myeloid leukemia (AML) cell lines harboring KMT2A-AFF1 (MLL-AF4) in culture, and inhibited tumor growth and progression in an AML cell line xenograft model harboring KMT2A-AFF1 (MLL-AF4) (PMID: 25817203). 25817203
KMT2A - AFF1 leukemia sensitive IRAK1/4 Preclinical - Cell culture Actionable In a preclinical study, leukemia cells harboring KMT2A-AFF1 demonstrated sensitivity to IRAK1/4 in culture, resulting in decreased cell proliferation (PMID: 28065413). 28065413
KMT2A - AFF1 B-cell acute lymphoblastic leukemia predicted - resistant Blinatumomab Case Reports/Case Series Actionable In a clinical case study, Blincyto (blinatumomab) treatment in a patient with B-lymphoblastic leukemia harboring KMT2A-AFF1 resulted in lineage switch as demonstrated by identification of myeloid sarcoma of the breast and acute myeloid leukemia within one month of treatment onset, along with mixed phenotype acute leukemia observed following cessation of treatment (PMID: 31885955). 31885955
KMT2A - AFF1 acute lymphoblastic leukemia predicted - sensitive AZD5153 Preclinical - Cell line xenograft Actionable In a preclinical study, AZD5153 inhibited proliferation of acute lymphocytic leukemia cells harboring KMT2A-AFF1 (MLL-AF4) in culture, resulted in tumor growth inhibition in cell line xenograft models (PMID: 27573426). 27573426
Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT02727803 Phase II anti-thymocyte globulin + Fludarabine + haNK cells + Melphalan anti-thymocyte globulin + Cyclophosphamide + Fludarabine + haNK cells + Rituximab anti-thymocyte globulin + Busulfan + Clofarabine + Fludarabine + haNK cells Personalized NK Cell Therapy After Chemotherapy and Cord Blood Transplant in Treating Patients With Myelodysplastic Syndrome, Leukemia, Lymphoma or Multiple Myeloma Recruiting