Isocitrate Dehydrogenase (IDH) 1 and 2 Mutations Predicts Better Outcome in Patients with Acute Myeloid Leukemia Undergoing Allogeneic Hematopoietic Cell Transplantation: a study of the ALWP of the EBMT

Abstract Isocitrate dehydrogenase 1 and 2 (IDH1 and IDH2) mutations have uncertain prognostic implications in AML. We investigate the impact IDH1 and IDH2 mutations in AML patients undergoing allogeneic hematopoietic cell transplantation (allo-HCT) in first complete remission (CR1). In total, 1515 adult patients were included, 15.91% (n = 241) carried IDH1 mutation (mIDH1), and 26.27% (n = 398) IDH2 mutation (mIDH2) and 57.82% (n = 876) had no-IDH mutation. NPM1 was frequently encountered with IDH1 mutation (no-IDH group, n = 217, 24.8%, mIDH1, n = 103, 42.7%, mIDH2, n = 111, 27.9%, p IDH1 and mIDH2 compared to no-IDH groups (Hazard ratio HR = 0.66 (95%CI 0.47–0.91), p = 0.011; HR = 0.73 (95%CI 0.56–0.96), p = 0.025, respectively). In the mIDH1 group, overall survival (OS) was improved compared to no-IDH (HR = 0.68 (95%CI 0.48–0.94), p = 0.021), whereas mIDH2 was associated with lower incidence of relapse (HR = 0.49 (95%CI 0.34–0.7), p p = 0.004) and OS (HR = 0.74 (95%CI 0.56–0.97), p = 0.027). In the subgroup of NPM1 wild type, only IDH2 was associated with improved outcomes. In conclusion, our data suggest that IDH1 and IDH2 mutations are associated with improved outcomes in patients with AML undergoing allo-HCT in CR1.