Integration of Functional Ex Vivo Drug Testing and Molecular Profiling Reveals Novel Vulnerabilities in CMML

摘要: Chronic myelomonocytic leukemia (CMML) denotes a group of rare hematologic neoplasms characterized by peripheral blood monocytosis. Moreover, they have overlapping features with myelodysplastic syndrome (MDS) and myeloproliferative neoplasms (MPN). The genomic landscapes of CMML have been recently described and uncovered few somatic events involving a subset of recurrent gene mutations as TET2, SRSF2, ASXL1, and RAS . Despite the increased knowledge in disease biology, this has not led to deeper understanding of the pathophysiology of the disease mechanisms nor translated in improved outcomes or novel therapeutic modalities. Patients with CMML exhibit significant inter and intra patient heterogeneity with regard to genetic and/or clinical features, which makes identification of disease-specific therapeutic vulnerabilities challenging. Hence, there is need to identify novel treatment …