Phase III Trial Evaluates Routinized Tranexamic Acid for Bleeding Reduction in Hematologic Malignancy

In the TREATT phase III trial, investigators assessed routinized use of tranexamic acid to reduce bleeding in patients receiving intensive therapy for hematologic malignancy and severe thrombocytopenia. They published their findings in The Lancet Haematology.

“There is insufficient evidence to support routine use of tranexamic acid to reduce bleeding in patients with haematological malignancies undergoing intensive chemotherapy,” the investigators concluded.

The international, multicenter, randomized, double-blind trial initially enrolled 616 adults who were receiving intensive chemotherapy or hematopoietic stem-cell transplantation for a hematologic malignancy. Patients also had platelet counts of 10 × 10⁹/L or lower for at least five days. The total cohort had a median age of 58 years, and 62% of participants were men.

In the modified intention-to-treat population used for analysis in the trial, 300 patients received tranexamic acid, 1 g intravenously or 1.5 g orally, every eight hours; and 297 patients received placebo when platelet counts fell below 30 × 10⁹/L.

After 30 days of treatment, bleeding of World Health Organization grade 2 or higher or death had occurred in 31.7% of the trial’s tranexamic acid group and in 34.2% of the placebo group, with a hazard ratio of 0.92 (P=.62). The investigators noted there were no differences in thrombotic events or veno-occlusive disease between the two groups.

By day 60 of the trial, in the tranexamic acid group 94 serious adverse events affecting 77 patients had occurred, and in the placebo group, 103 events affected 82 participants.

TREATT was funded by the UK National Health Service Blood and Transplant and Australian National Health and Medical Research Council.

Reference

TREATT Trial Investigators. Tranexamic acid versus placebo to prevent bleeding in patients with haematological malignancies and severe thrombocytopenia (TREATT): a randomised, double-blind, parallel, phase 3 superiority trial. Lancet Haematol. 2025;12(1):e14-e22. doi:10.1016/S2352-3026(24)00317-X