Abstract
BackgroundPatients with haematological malignancies often develop thrombocytopenia as a consequence of either their disease or its treatment. Platelet transfusions are commonly given to raise a low platelet count and reduce the risk of clinical bleeding (prophylaxis) or stop active bleeding (therapy). Recent studies have shown that many patients continue to experience bleeding despite the use of prophylactic platelet transfusions. Tranexamic acid is an anti-fibrinolytic, which reduces the breakdown of clots formed in response to bleeding. Anti-fibrinolytics have been shown to prevent bleeding, decrease blood loss and use of red cell transfusions in elective and emergency surgery, and are used widely in these settings. The aim of this trial is to test whether giving tranexamic acid to patients receiving treatment for haematological malignancies reduces the risk of bleeding or death and the need for platelet transfusions.MethodsThis is a multinational randomised, double-blind, placebo-controlled, parallel, superiority trial. Patients will be randomly assigned to receive tranexamic acid (given intravenously or orally) or a matching placebo in a 1:1 ratio, stratified by site. Patients with haematological malignancies receiving intensive chemotherapy or stem cell transplantation (or both) who are at least 18 years of age and expected to become severely thrombocytopenic for at least 5 days will be eligible for this trial. The primary outcome of the trial is the proportion of patients who died or had bleeding of World Health Organization grade 2 or above during the first 30 days of the trial. We will measure the rates of bleeding daily by using a short, structured assessment of bleeding, and we will record the number of transfusions given to patients. We will assess the risk of arterial and venous thrombosis for 120 days from the start of trial treatment.DiscussionThis trial will assess the safety and efficacy of using prophylactic tranexamic acid during a period of intensive chemotherapy and associated thrombocytopenia in people with haematological disorders.Trial registrationThis study was prospectively registered on Current Controlled Trials on 25 March 2015 (ISRCTN73545489) and is also registered on ClinicalTrials.gov (NCT03136445).
Highlights
Patients with haematological malignancies often develop thrombocytopenia as a consequence of either their disease or its treatment
Many patients experience bleeding despite the use of prophylactic platelets with World Health Organization (WHO) grade 2 or greater bleeding reported in 48% to 79% of patients receiving prophylactic platelets in clinical trials [8, 9]
The burden of bleeding varies in sub-groups of patients (e.g., higher bleeding rates in patients with acute leukaemia or receiving an allogeneic stem cell transplant (SCT) and lower rates in patients receiving an autologous Stem cell transplant (SCT)) [14]. These findings suggest that factors other than absolute platelet count may be important in prevention of bleeding in patients with haematological malignancies and that current policies for prophylactic platelet transfusions have a limited role in reducing much of the bleeding seen in haematology patients undergoing intensive chemotherapy or stem cell transplantation or both
Summary
Study design and setting This is a multinational, randomised, double-blind, placebo-controlled, parallel, superiority trial. Bleeding assessments Daily data collection and bleeding assessment will commence when the patient is randomly assigned and will stop when one of the following occurs: It has been 30 days since the first recorded platelet count was not more than 30 × 109/L The participant withdraws his or her consent to having bleeding assessments performed. 1. The proportion of participants who die or have bleeding of grade 2 or above by WHO criteria during the first 30 days from the first dose of trial treatment, or planned first dose for those participants who did not receive treatment, will be estimated by the KM method and compared by using Cox regression analysis.
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