WT1 and BCORL1 Identified By Computational Biology Modeling Analysis of Patient Genomics Are Novel Predictors of Response to Azacitidine (AZA) and Lenalidomide (LEN) Treatment in Acute Myeloid Leukemia (AML)

AML patients with relapsed/refractory (R/R) disease have few effective treatment options. LEN+AZA may be an active and better tolerated regimen compared with conventional chemotherapy. This combination has been tested in a phase 2 pilot study of LEN+AZA in 37 R/R older patients with a 49% overall response rate (4 complete remission (CR) / CR with incomplete recovery (CRi) and 14 with morphologic leukemia free state (MLFS). The objective of this study was to utilize the genomics of these patients to retrospectively predict response to AZA+LEN treatment using CBM workflow and identify key genomic characteristics of the responder profiles.

Blood Journal 2018 Acute Myeloid Leukemia