Recent clinical trial results suggest that in patients receiving certain treatments for hematologic malignancy who are at risk of thrombocytopenia, prophylactic therapy with tranexamic acid (TXA) did not significantly reduce the incidence of bleeding events of grade 2 or higher, as defined by World Health Organization (WHO) criteria. The study results were reported in the journal Blood.

The study was the randomized, controlled, double-blind A-TREAT trial ( Identifier: NCT02578901). The study enrolled patients with a hematologic malignancy or aplasia being treated with chemotherapy, immunotherapy, or hematopoietic stem cell transplant and were at risk of hypoproliferative thrombocytopenia with a platelet count at or below 10,000/mL for 5 or more days.

After a fall in platelet count to below 50,000/mL, patients were randomized to TXA or placebo study arms. With a drop in platelet count to below 30,000/mL, patients were activated to the treatment phase. TXA was given as 1.3 g orally or 1.0 g intravenously every 8 hours. The primary study outcome was the incidence of bleeding at WHO grade 2 or higher during the first 30 days after activation. Several other outcomes were also assessed.

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A total of 330 patients were activated to the treatment phase, with the primary outcome evaluable in 145 patients receiving TXA and 144 receiving placebo. WHO grade 2 or higher bleeding in the first 30 days after activation occurred in 50.3% of patients in the TXA group and in 54.2% of patients in the placebo group (adjusted odds ratio, 0.83; 95% CI, 0.50-1.34; P =.44).

Participants in either group did not show a significant difference in the mean number of platelet transfusions received (mean difference, 0.07; 95% CI, -1.90 to 2.04; P =.95). The mean days alive without grade 2 or higher bleeding was also similar between groups (mean difference, 0.79; 95% CI, -0.40 to 1.98; P =.19). No deaths were caused by serious bleeding in either group.

Thrombotic events were assessed for 120 days after activation, and they were reported in 3.7% of patients in the TXA group, compared with 5.5% in the placebo group. There were no deaths due to thrombosis observed in 120 days in either group. Rates of adverse events and serious adverse events were similar between treatment arms, although central line occlusions were reported in more patients of the TXA arm (16.6%) than in the placebo arm (6.7%).

“Among patients with hematologic malignancy undergoing chemotherapy or hematopoietic stem cell transplantation, prophylactic treatment with TXA compared with placebo did not significantly reduce the risk of WHO grade ≥2 bleeding,” the study investigators concluded in their report.

Disclosures: Some authors have declared affiliations with or received grant support from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.


Gernsheimer TB, Brown SP, Triulzi DJ, et al. Prophylactic tranexamic acid in patients with hematologic malignancy: a placebo-controlled, randomized clinical trial. Blood. 2022;140(11):1254-1262. doi:10.1182/blood.2022016308