Figure 1.

Illustration of the experimental set up. First, VPA effects were investigated in vitro using myeloid cell lines (n = 5) and in vivo using primary AML samples from patients receiving intensive chemotherapy in combination with VPA (n = 12) or without VPA (n = 6). Based on global gene expression and miRNA profiling, a VPA response signature was generated. This data was intersected with a VPA response predictor generated in 88 diagnostic AML cases who have been also intensively treated in combination with VPA (n = 40) or without VPA (n = 48). Finally, the impact of the refined VPA response predictor genes was validated in an independent cohort of 114 primary AML samples treated intensively in combination with VPA.