Tranexamic acid to prevent bleeding in patients with hematologic malignancies and hypoproliferative thrombocytopenia: a systematic review and meta-analysis of randomized controlled trials with trial sequential analysis.

[BACKGROUND] Tranexamic acid (TXA) is commonly used off-label as an adjunct to prophylactic platelet transfusion to prevent bleeding in patients with hematological malignancies undergoing intensive chemotherapy or hematopoietic stem cell transplantation.

[OBJECTIVES] We conducted a meta-analysis of randomized controlled trials (RCTs) to assess the efficacy and safety of TXA vs placebo for the primary prevention of bleeding in patients with hematological malignancies experiencing hypoproliferative thrombocytopenia.

[METHODS] Embase, MEDLINE, and CENTRAL were searched from inception until June 30, 2025. The primary efficacy outcome was the incidence of World Health Organization grade of ≥2 bleeding. The secondary efficacy outcome was the mean number of platelet transfusions per participant in each group. Safety outcomes included the incidence of thrombotic events, veno-occlusive disease, and all-cause mortality. Random-effects meta-analyses were performed for the outcomes of interest.

[RESULTS] Two RCTs enrolling 946 patients were included. Compared with placebo, TXA did not reduce the risk of World Health Organization grade of ≥2 bleeding (relative risk [RR], 0.91; 95% CI, 0.77-1.07) or the mean number of platelet transfusions per participant (standardized mean difference, 0.00; 95% CI, -0.12 to 0.13). The risk of thrombotic events (RR, 1.00; 95% CI, 0.43-2.32), veno-occlusive disease (RR, 1.49; 95% CI, 0.42-5.25), and all-cause mortality (RR, 1.26; 95% CI, 0.78-2.02) was similar in the 2 groups. Trial sequential analysis for the primary outcome indicated that the current evidence is conclusive, suggesting the futility of additional RCTs.

[CONCLUSIONS] TXA did not provide sufficient benefits to justify its routine use for preventing bleeding in this patient population.