Platelet Transfusion Requirements in Hematopoietic Transplantation Pilot Study

Official Title

Platelet Transfusion Requirements in Hematopoietic Transplantation (PATH Pilot)

Summary:

It is hypothesized that a strategy using prophylactic oral Tranexamic Acid (TXA) with therapeutic platelet transfusions is safe and effective compared to prophylactic platelet transfusions in patients undergoing an autologous hematopoietic stem cell transplantation (who are at risk for bleeding).

Trial Description

Primary Outcome:

• Enrolment, as measured by the number of patients screened per month at each site
• Number of off-protocol platelet transfusions, with a target of < 10% off-protocol transfusions in each treatment arm
• Total number of platelet transfusions/group, with a target of 25% reduction in the tranexamic acid arm
• Adherence to tranexamic acid use, defined as excellent (greater than or equal to 90% use), acceptable (75-90% use), poor (< 75% use)

Secondary Outcome:

• WHO (World Health Organization) Bleeding events of Grade 2 or higher
• Time from randomization to bleeding of WHO bleeding events Grade 2 or higher
• Number of days with bleeding of WHO bleeding events Grade 2 or higher
• Bleeding Severity Measurement Scale for bleeding events Grade 2 or higher
• Number of platelet and/or red cell transfusions
• Time to platelet recovery
• Number of days with platelet count < 10 x 10^9/L
• LOS (Length of hospital stay)
• Adverse transfusion reactions
• Bearman Toxicity Score
• Infections at Day 30
• Quality of Life measurements, as determined by a battery of QoL instruments

In Canada, over 1,500 autologous hematopoietic stem cell transplantations (ASCT) are performed annually for hematologic malignancies. It is currently standard practice to provide a prophylactic transfusion of platelets to prevent bleeding when the daily measured platelet count is less than 10 x 109/L. A patient may require up to six adult platelet doses during the post-transplant period. However, the true benefit of prophylactic platelet transfusions in the ASCT setting is unclear and has been called into question by several recent studies. Prophylactic platelet transfusions may not only be unnecessary, they may be detrimental to the patient. Among blood products, platelet transfusions are associated with the highest risk of both infectious and non-infectious complications: this would include bacterial infections and allergic /febrile reactions. Moreover, the potential overuse of platelet products places a significant burden on a scarce health care resource that is provided through volunteer donations. An alternative strategy to prevent bleeding and reduce the need for platelet transfusions involves administering Tranexamic Acid, an oral antifibrinolytic agent to stabilize blood clots and reduce bleeding. Tranexamic Acid is safe and effective in many clinical scenarios, and may be a reasonable alternative for prophylactic platelet transfusions. In the setting of ASCT, Tranexamic Acid may reduce bleeding and further enhance a strategy of therapeutic platelet transfusions where platelets are administered only in the event of active bleeding symptoms. The effect of prophylactic platelet transfusions and Tranexamic Acid on clinical, quality of life and economic outcomes in patients receiving ASCT is unknown. The primary aim of this research program is to perform a randomized controlled trial to determine whether a strategy of prophylactic Tranexamic Acid (with therapeutic platelet transfusions) is safe and effective compared to prophylactic platelet transfusions in patients undergoing ASCT. Before conducting a larger trial, the investigators first propose a pilot randomized controlled trial to determine the feasibility of such a study.

View this trial on ClinicalTrials.gov