For patients with intermediate-risk acute myeloid leukemia (AML) without FLT3-ITD, biallelic CEBPA, and NPM1 mutations (triple-negative AML) who achieve complete remission, hematopoietic cell transplantation (HCT) significantly improved treatment outcomes.
Researchers analyzed data from two prospective randomized controlled trials on intensive induction- and risk-stratified post-remission AML therapy, performing a donor versus no-donor analysis on 497 adults age 18-60 years with triple-negative AML.
Patients with a matched related donor experienced a significantly longer relapse-free survival (RFS) (Hazard ratio [HR] 0.5; 95% CI 0.3-0.9, p=0.02) compared to patients receiving post-remission chemotherapy. The researchers noted that only 58% of patients in the donor group were transplanted in first complete remission (CR1). At 5 years after achieving CR1, RFS for matched related donor HCT recipients and patients without a donor was 49% and 26%, respectively.
Researchers then performed a multivariate analysis with allogeneic HCT as a time-dependent variable, which found that overall survival and RFS were both extended for patients who received allogeneic HCT compared with chemotherapy in CR1: (HR 0.58, 95% CI 0.37-0.9, p=0.02) and (HR 0.51, 95% CI 0.34-0.76; p=0.001), respectively.
The researchers concluded that “outside of clinical trials, allogeneic HCT should be the preferred post-remission treatment of patients with intermediate-risk NPM1-negative, CEBPA-negative, and FLT3-ITD-negative AML in CR1.”