Venetoclax in Relapsed/Refractory Acute Myeloid Leukemia: Are Supporting Evidences Enough?

Simple Summary Venetoclax (VEN) is a potent oral inhibitor of the anti-apoptotic molecule BCL2, approved for adults with chronic lymphocytic leukemia (CLL), and recently for naïve acute myeloid leukemia (AML) unfit for intensive chemotherapy. Therefore, VEN is not approved for relapsed/refractory (R/R) AML patients; consequently, this use falls within the so-called off-label use. This review provides evidence of the role of VEN-based therapy in R/R AML patients, including data from clinical trials and from retrospective studies. Abstract Despite the progress in the development of new therapeutic strategies, relapsed/refractory (R/R) acute myeloid leukemia (AML) still represents a high unmet medical need. Treatment options in this setting include enrollment into clinical trials, allogeneic stem cell transplantation and/or targeted therapy. Nevertheless, it is associated with poor outcomes. Thus, the development of new treatments, which could ameliorate the prognosis of these patients with a good safety profile are highly demanded. Recently, venetoclax (VEN) has been approved for naïve AML patients unfit for intensive chemotherapy. In this regard, regimens including VEN could represent a valuable treatment option even in those with R/R disease and several studies have been conducted to demonstrate its role in this clinical setting. This review aims to summarize the current evidence on the use of VEN regimens in the treatment of R/R AML.