Systematic review and meta-analysis of ATRA plus arsenic trioxide versus ATRA plus chemotherapy in newly diagnosed acute promyelocytic leukemia

Acute promyelocytic leukemia (APL) is an aggressive malignancy. The optimal choice between all-trans retinoic acid plus arsenic trioxide (ATRA + ATO) and ATRA plus chemotherapy for newly diagnosed APL is not clear. A systematic review and meta-analysis of published studies compared these two options. Twelve studies met the inclusion criteria. In randomized controlled trials, ATRA + ATO significantly improved complete remission rates (RR 1.04; 95% CI 1.02-1.06), disease-free survival (1.22; 1.11-1.34). event-free survival (1.25; 1.20-1.29), and overall survival (1.07; 1.03-1.12) compared with ATRA + chemotherapy. Observational studies showed similar findings, supporting real-world generalizability. A trend toward lower gastrointestinal toxicity was observed with ATRA + ATO (RR 0.28; 95% CI 0.07-1.18). ATRA + ATO was associated with an increased risk of QTc prolongation (RR 3.79; p=0.05). The authors conclude that these findings support the preferential clinical use of ATRA + ATO with close cardiac monitoring.