Outpatient Platelet Transfusions in Myelodysplastic Syndromes and Leukemia: The OPTIMAL Pilot

As a result of the underlying disease or its therapy, it is common for patients with blood cancers to have low platelet counts. While platelet transfusions may be beneficial in preventing or treating bleeding symptoms, in circumstances where the risk of bleeding is low they may be unnecessary or even harmful. As a blood product, transfusion of platelets may be associated with infectious or allergic complications, and frequent hospital visits for transfusion may adversely affect quality of life. Additionally, the potentially overuse of platelet products places a burden on health care resources. The benefit of the current practice of prophylactic platelet transfusions to prevent hemorrhage is unknown. The randomized data that exists is more than 25 years old and not informative given methodological limitations and the changing standards of supportive care. An alternative, therapeutic, strategy involves only administering platelets to control active bleeding. The standard of practice in inpatients receiving high dose chemotherapy (either for acute leukemia or as part of stem cell transplantation) is prophylactic platelet transfusions. In outpatients not receiving high dose chemotherapy, the risk of bleeding is significantly lower. No randomized trials have examined the optimal platelet transfusion strategy in outpatients with blood cancers undergoing supportive or palliative therapy. Thus the potential benefit of prophylactic transfusions in the outpatient setting is unknown. The investigators propose to perform a pilot randomized controlled trial to determine if a larger trial is possible. The ultimate goal is to determine if a strategy of therapeutic platelet transfusions is safe and effective in outpatients with blood cancers and low platelet counts.

Sex

All

Minimum age

18 Years

Inclusion Criteria:

1. Adults 18 years or older with documented MDS (including MDS-subtype, CMML) or AML (as defined by WHO criteria)
2. Severe thrombocytopenia defined as a platelet count of ≤ 10 x 109/L documented on two consecutive samples at least 7 days apart.
3. Receiving outpatient-based supportive or palliative care including palliative cytoreductive, immunomodulatory or hypomethylating therapy, e.g. hydroxyurea or low dose cytarabine, lenalidomide, azacytidine, or decitabine.
4. Eastern Cooperative Oncology Group (ECOG) performance status of 0 - 2.

Exclusion Criteria:

1. High-dose therapy in past 2 months, e.g. AML-type induction or consolidation therapy
2. Thrombocytopenia suspected to be due to immune or peripheral destruction
3. Splenomegaly, palpated at greater than 5 cm below the costal margin or greater than 20 cm on imaging
4. Alloimmune platelet refractoriness
5. Clinically relevant bleed (grade 3 or higher) within the past 3 months
6. Coagulopathy (prothrombin time or activated partial thromboplastin more than 1.5 times the upper limit of normal or fibrinogen less than 2 g/L)
7. Require anticoagulant therapy, e.g. heparin, or antiplatelet therapy, e.g. aspirin
8. Significant renal impairment (Creatinine more than 1.5 times the upper limit of normal)
9. Geographic inaccessibility resulting in the inability to comply with follow-up visits
10. Pregnant or breast-feeding
11. Unwilling or unable to provide informed consent