Data from Investigational Clinical Trial Agents versus Conventional Second Salvage Therapies in Patients with Relapsed/Refractory Acute Myeloid Leukemia

Year: 2026

Type: other

Abstract: Purpose: 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. Experimental Design: 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). Results: 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. Conclusions: 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.

DOI: 10.1158/1078-0432.c.8715341

Topic: Acute Myeloid Leukemia Research

Subfield: Hematology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

OpenAlex ID: W7213228303