LAUSR

Recent clinical data have shown a synergistic effect of venetoclax in combination with the hypomethylating agents (HMA) azacitidine and decitabine (complete remission [CR]/CR with incomplete cell count recovery [CRi]: 73%, median overall survival [OS] of 17.5 months) as well as low-dose cytarabine (LDAC; CR/CRi: 54%; median OS of 10.1 months) in the frontline setting in older patients with acute myeloid leukemia (AML) and those ineligible for intensive chemotherapy leading to approval of both combinations in the US. However, data on relapsed/refractory (R/R)-AML are scarce and heterogenous. Outcomes of patients with R/R-AML are dismal with a median OS of 3-7 months and there is no approved standard of care. Multiple clinical trials combining either venetoclax alone, venetoclax + HMA or venetoclax + LDAC as the backbone of therapy with other novel agents in R/R-AML are ongoing. As many of these trials are not randomized, it is vital to understand the response rate to venetoclax alone and venetoclax + HMA/LDAC in R/R-AML to use it as a benchmark for comparison. Therefore, we performed a systematic literature review and meta-analysis to objectively assess overall response rates (ORR), and rates of CR/CRi for R/R-AML patients treated with venetoclax or venetoclax + HMA/LDAC. MEDLINE via PubMed, Ovid EMBASE, the COCHRANE registry of clinical trials (CENTRAL), Scopus and the Web of Science electronic databases were