Investigational Clinical Trial Agents versus Conventional Second Salvage Therapies in Patients with...

Outcomes in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) remain discouraging despite the rapid expansion of treatment paradigms over the past decade. Clinicians must often decide between conventional salvage treatment regimens or enrollment in clinical trials.

We retrospectively identified 657 patients with AML in second treatment salvage and compared the outcomes of those who received investigational agents (n = 354) with conventional salvage regimens (n = 303).

Conventional salvage was associated with higher response rates [complete response (CR) or CR with incomplete count recovery rate: 22% vs. 12%, P = 0.0008] and higher early mortality rates (60-day mortality: 17% vs. 7%, P = 0.0002). There was no difference in rates of bridging to allogeneic stem cell transplant (7% vs. 10%, P = 0.2), event-free survival (EFS; median: 1.5 vs. 2 months, P = 0.9), or overall survival (OS; median: 4.1 vs. 4.5 months, P = 0.8). A subgroup analysis of conventional regimens showed improved EFS (median EFS: 3.6 vs. 1.4 months, P = 0.004) and a trend toward improved OS (median OS: 7.5 vs. 3.8 months, P = 0.06) with the addition of venetoclax in venetoclax-naïve patients.

Enrollment of R/R patients in clinical trials is a suitable alternative to conventional salvage regimens, offering novel therapeutic approaches to improve upon the historically dismal outcomes in this setting.