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

作者：Daniel Nguyen, Hagop M. Kantarjian, Aram Bidikian, Carvalho Manuel, Wei Qiao, Jing Ning, Courtney D. DiNardo, Tapan M. Kadia, Naval Daver, Gautam Borthakur, Elias Jabbour, Musa Yılmaz, Abhishek Maiti, Koji Sasaki, Guillermo Montalban‐Bravo, Kelly S. Chien, Danielle Hammond, Sherry Pierce, Michael Andreeff, Farhad Ravandi, Nicholas J. Short, Guillermo Garcia‐Manero, Ghayas C. Issa · 发表于：Clinical Cancer Research · 年份：2026 · DOI：10.1158/1078-0432.ccr-26-0951 · 研究领域：Acute Myeloid Leukemia Research、Hematopoietic Stem Cell Transplantation、Acute Lymphoblastic Leukemia research

PURPOSE: Outcomes in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) remain discouraging despite rapid expansion of treatment paradigms over the past decade. Clinicians must often decide between conventional salvage treatment regimens or enrollment on 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) versus conventional salvage regimens (N=303). RESULTS: Conventional salvage was associated with higher response rates (CR/CRi 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 months vs 2.0 months, P=0.9) or overall survival (OS; median: 4.1 months vs 4.5 months, P=0.8). A subgroup analysis of conventional regimens showed improved EFS (median EFS: 3.6 months vs 1.4 months, P=0.004) and a trend towards improved OS (median OS: 7.5 months vs 3.8 months, P=0.06) with the addition of venetoclax in venetoclax-naïve patients. CONCLUSION: Enrollment of R/R patients on clinical trials is a suitable alternative to conventional salvage regimens, offering novel therapeutic approaches to improve upon the historically dismal outcomes in this setting.