Retrospective study of investigational clinical trial agents versus conventional second salvage therapies in R/R AML

Outcomes of patients with relapsed/refractory acute myeloid leukemia (R/R AML) remain discouraging despite rapid expansion of treatment paradigms over the past decade. Clinicians must often choose between conventional salvage treatment regimens and enrollment on clinical trials. A retrospective study at MD Anderson Cancer Center (Houston, TX) compared outcomes of R/R AML patients in second treatment salvage receiving investigational agents (n=354) or conventional salvage regimens (n=303). Conventional salvage was associated with higher response rates (CR/CRi 22% versus 12%; p=0.0008) and higher early mortality rates (60-day mortality 17% versus 7%; p=0.0002). There was no significant difference in rates of bridging to allogeneic stem cell transplant (7% versus 10%; p=0.2), event-free survival (median 1.5 months versus 2.0 months; p=0.9), or overall survival (median4.1 months versus 4.5 months; p=0.8). A subgroup analysis of conventional regimens showed improved EFS (median 3.6 versus 1.4 months; p=0.004) and a trend towards improved OS (median 7.5 versus 3.8 months; p=0.06) with the addition of venetoclax in venetoclax-naïve patients. The authors conclude that enrollment of R/R AML patients on clinical trials is a suitable alternative to conventional salvage regimens.