Evaluation of Tranexamic Acid in Myelodysplastic Syndromes and Acute Myeloid Leukemia

RATIONALE: Myelodysplastic syndromes (MDS), myelodysplastic/myeloproliferative neoplasm (MDS/MPN) and acute myeloid leukemia (AML) are serious, life-changing blood cancers. Despite the best efforts of their care team, patients with MDS and AML commonly experience complications related to bleeding. These complications affect patient quality-of-life and can sometimes lead to hospitalization or death. Evaluation of affordable and widely available treatments to minimize bleeding complications among patients with MDS and AML is needed.

STUDY OBJECTIVES: To evaluate the feasibility of tranexamic acid (TXA) that will evaluate the efficacy and safety of treatments to minimize bleeding in patients with MDS and AML treated in the outpatient setting.

METHODOLOGY: The investigators will conduct a multicenter pilot randomized control trial (RCT) for outpatients ≥18 years of age with MDS and AML. Patients with MDS and AML with low platelet counts will receive TXA (a medication that prevents clots from dissolving). TXA is commonly used in other clinical settings but have not been studied in patients with MDS or AML receiving outpatient chemotherapy (ie, chemotherapy that can be given from clinic, rather than a hospital). In this study, 50% of patients will be randomized (like the flip of a coin) to receive the medication the investigators are studying. The other 50% of patients will receive a matching placebo.

OUTCOMES: The primary feasibility outcome is the ability to enroll a mean of 1 patient per site per month.

SITES AND DURATION: The investigators will initially enroll patients from 10-15 sites across Canada. The expected duration of enrollment is 2 years.

SIGNIFICANCE: With a broad range of stakeholders, including patient partners, the trial will address a broadly applicable patient-prioritized question. Tranexamic acid is readily available, inexpensive, and has an established side effect profile. Results of this trial are highly generalizable and will broadly impact the care of patients with MDS and AML.

Inclusion criteria

Master platform inclusion criteria:

• Age ≥ 18 years
• Diagnosis of myelodysplastic syndromes, myelodysplastic/myeloproliferative neoplasm or acute myeloid leukemia

MYELO-CAN TXA inclusion criteria:

• Receipt of less-intensive chemotherapy (includes both frontline and relapsed/refractory setting)
• Severe thrombocytopenia (platelets ≤ 30x10^9/L or platelets ≤ 50x10^9/L prior to chemotherapy initiation)

Exclusion criteria

Master platform exclusion criteria:

• Participant is deemed unlikely to survive >30 days (as determined by clinical team)
• Participant unable to provide informed consent

MYELO-CAN TXA exclusion criteria:

• Known allergy to tranexamic acid
• Active thromboembolic disease
• Active ischemic heart disease
• Gross hematuria
• Stage V chronic kidney disease
• Clinically suspected disseminated intravascular coagulation (DIC)
• Pregnancy and/or breastfeeding