Trial to evaluate tranexamic acid therapy in Thrombocytopenia

Chief investigators: Dr Simon Stanworth and Dr Lise Estcourt
Primary sponsor: NHSBT
Funder: NHSBT and the National Health and Medical Research Council of Australia (NHMRC)


TREATT is a double blind, randomised controlled trial evaluating the safety and efficacy of tranexamic acid in patients with haematological malignancies with severe thrombocytopenia.

Patients with cancers of the blood often develop low blood cell counts either as a consequence of the disease or the treatment by chemotherapy or stem cell transplantation. Platelet transfusions are commonly given to raise low platelet counts and reduce the risk of clinical bleeding (prophylaxis) or to stop active bleeding (therapy).

However, recent studies have indicated that many patients continue to experience bleeding despite the use of platelet transfusions. Tranexamic acid is a class of drug called antifibrinolytic. These drugs act to reduce the breakdown of clots formed in response to bleeding. Antiofibrinolytics have been used widely in both elective and emergency surgery and have been shown to decrease blood loss and the use of red cell transfusions.

The purpose of this study is to test whether giving tranexamic acid to patients receiving treatment for blood cancers reduces the risk of bleeding or death, and the need for platelet transfusions. Patients will be randomised to receive tranexamic acid or a placebo (given intravenously, or orally). We will measure the rates of bleeding daily using a short structured assessment of bleeding, and we will record the number of transfusions given to patients.

Study hypothesis

The hypothesis is that in patients with haematological malignancies, during a period of severe thrombocytopenia, prophylactic use of antifibrinolytics will decrease bleeding or death and the demand for platelet transfusions.

Study design

A Phase III, randomised, double-blind, placebo-controlled, parallel, superiority trial.

Patients will be randomised to receive tranexamic acid or a matching placebo in a 1:1 ratio, stratified by site. Randomisation will further be balanced within blocks of varying undisclosed sizes.

Study contact

Gillian Powter
Tel: 07590 353091 / 01865 387903


C. Dyer, C. R. Alquist, M. Cole-Sinclair, E. Curnow, N. M. Dunbar, L. J. Estcourt, R. Kaufman, J. M. Kutner, J. McCullough, Z. McQuilten, L. Potiphar, B. Rioux-Masse, S. Slichter, A.Tinmouth, K. Webert, A. P. Yokoyama, S. J. Stanworth, & on behalf of the BEST Collaborative – A multicentred study to validate a consensus bleeding assessment tool developed by the biomedical excellence for safer transfusion collaborative for use in patients with haematological malignancy. Vox Sanguinis 2018. DOI: 10.1111/vox.12627