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.