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

NCT01615146 | Terminated DMC

• 1 other identifier: OHREB 2011500
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Myelodysplastic Syndrome; Leukemia

Keywords

Myelodysplasia; Leukemia; Thrombocytopenia; Platelet; transfusions; Outpatient

Outcome Measures

Primary Outcomes (1)

• Feasibility

  Overall enrollment, off protocol transfusions per each randomized group, total number of platelet transfusions per group and patient compliance with daily self assessment of bleeding will be evaluated.

  18 months

Secondary Outcomes (1)

• Bleeding events between therapeutic and Prophylactic transfusion groups

  6 month follow up period

Study Arms (2)

Therapeutic Platelet Transfusion Arm

EXPERIMENTAL

Patients allocated to the therapeutic platelet transfusion group will not receive routine prophylactic platelet transfusions.

Other: Platelet Transfusion

Prophylactic Platelet Transfusion Group

ACTIVE COMPARATOR

Patients allocated to the prophylactic platelet transfusions will receive a platelet transfusion (a single dose of random donor platelets (4 unit pool or random donor platelets or one apheresis unit) when the measured platelet count is \< 10 x 109/L.

Other: Platelet Transfusion

Interventions

Platelet Transfusion OTHER

Patients allocated to the therapeutic platelet transfusion group will not receive routine prophylactic platelet transfusions. Platelet transfusions will be given to treat documented clinically relevant bleeding defined as WHO bleeding of grade 2 or greater. Patients may be transfused at the discretion of the treating physician. The indication for all platelet transfusions will be recorded by asking the ordering physician. Patients allocated to the prophylactic platelet transfusions will receive a platelet transfusion when the measured platelet count is \< 10 x 109/L. Patients may receive additional platelet transfusions at the discretion of the treating physician. The indication for all platelet transfusions will be recorded.

Prophylactic Platelet Transfusion Group; Therapeutic Platelet Transfusion Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adults 18 years or older with documented MDS (including MDS-subtype, CMML) or AML (as defined by WHO criteria)
• Severe thrombocytopenia defined as a platelet count of ≤ 10 x 109/L documented on two consecutive samples at least 7 days apart.
• 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.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 - 2.

You may not qualify if:

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

Sponsors & Collaborators

• Ottawa Hospital Research Institute: lead
• Canadian Blood Services: collaborator
• Canadian Institutes of Health Research (CIHR): collaborator

Study Sites (1)

the Ottawa Hospital

Ottawa, Ontario, k1h8l6, Canada

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes; Leukemia; Anemia, Refractory, with Excess of Blasts; Thrombocytopenia

Interventions

Platelet Transfusion

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Neoplasms by Histologic Type; Neoplasms; Anemia, Refractory; Anemia; Blood Platelet Disorders; Cytopenia

Intervention Hierarchy (Ancestors)

Blood Component Transfusion; Blood Transfusion; Biological Therapy; Therapeutics

Study Officials

• Alan Tinmouth, MD, MSc

  Ottawa Hospital Research Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 6, 2012
• First Posted: June 8, 2012
• Study Start: June 1, 2012
• Primary Completion: June 1, 2015
• Study Completion: June 1, 2015
• Last Updated: September 2, 2015

Record last verified: 2015-09

Locations

CA (1)