Prophylactic platelet transfusions are common for patients with hematological malignancies and severe hypoproliferative thrombocytopenia. Tranexamic acid, an antifibrinolytic drug, has been shown to reduce bleeding in surgery patients, patients requiring cesarean sections, and those with acquired bleeding disorders. Results from the Trial to Evaluate Tranexamic Acid Therapy in Thrombocytopenia (TREATT), however, do not support the routine use of tranexamic acid for patients with hematological malignancies. In this double blind, international phase 3 superiority trial, 616 patients with hematological malignancies (median age 58 years; 62% male; 87% white; and 42% with acute myeloid leukemia) and severe thrombocytopenia were randomized to receive either tranexamic acid or saline placebo every 8 hours when platelets dropped below 30 x 109 platelets/L for 30 days or until platelet recovery. After 30 days, 32% (30/298) of the patients in tranexamic acid arm had died or had WHO grade 2 bleeding or higher compared to 34% (98/295) of patients in the placebo arm (HR 0.92; 95% C.I., 0.67-1.27, p=0.62). In addition, no differences were observed in the number of platelet or RBC transfusions or the odds of bleeding between the two arms. Importantly, tranexamic acid therapy was safe and did not increase thrombotic or veno-occlusive events, but other treatments to reduce platelet transfusions and bleeding in patients with hematological malignancies are needed.
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