Differential protein and mRNA expression in PBMNCs from nonresponder vs responder with MDS/AML after treatment with omacetaxine and VEN. (A) CyTOF analysis demonstrating myeloid and apoptosis-related proteins differentially expressed at the baseline (C1D1) in phenotypically characterized AML stem/progenitor cells from nonresponders compared to those from patients who responded. (B) mRNA gene expression was exclusively altered in PBMNCs harvested at C1D5 after treatment with omacetaxine and VEN, compared to C1D1, in the patients with MDS who responded, as determined in KEGG pathway genes via RNA-Seq and gene set enrichment analysis. Top downregulated genes include β-catenin members (LEF1, FZD3, WNT7A, and WNT1) and hedgehog signaling pathway member (PTCH1). (C) CyTOF analysis demonstrating log2-fold reduction in expression of proteins in PBMNCs after response to omacetaxine and VEN treatment (C1D5 vs C1D1), occurring only in responders (patients 8 and 10 with MDS) vs nonresponders (patients 14 and 20). Lo, low; mtRux1, mutant RUNX1; PD, patient derived; Pos, positive.