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

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2013 by Ottawa Hospital Research Institute
Sponsor:
Collaborators:
Canadian Blood Services
Canadian Institutes of Health Research (CIHR)
Information provided by (Responsible Party):
Ottawa Hospital Research Institute
ClinicalTrials.gov Identifier:
NCT01615146
First received: June 6, 2012
Last updated: January 22, 2013
Last verified: January 2013
  Purpose

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.


Condition Intervention
Myelodysplastic Syndrome
Leukemia
Other: Platelet Transfusion

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Outpatient Platelet Transfusions in Myelodysplastic Syndromes and Leukemia: The OPTIMAL Pilot

Resource links provided by NLM:


Further study details as provided by Ottawa Hospital Research Institute:

Primary Outcome Measures:
  • Feasibility [ Time Frame: 18 months ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Bleeding events between therapeutic and Prophylactic transfusion groups [ Time Frame: 6 month follow up period ] [ Designated as safety issue: Yes ]

    Assessments will include:

    1. Non-cutaneous Grade 2 bleeding or higher by the World Heath Organization (WHO) bleeding assessment scale 28(Appendix B)
    2. Grade 3 bleeding or higher
    3. Time from randomization to first bleeding event of grade 3 of higher
    4. Total number of red cell transfusion per group
    5. Total number of hospital days per group
    6. Number of completed daily bleeding assessments per group
    7. Quality of life
    8. Mortality


Estimated Enrollment: 60
Study Start Date: June 2012
Estimated Study Completion Date: June 2014
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Therapeutic Platelet Transfusion Arm
Patients allocated to the therapeutic platelet transfusion group will not receive routine prophylactic platelet transfusions.
Other: Platelet Transfusion

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.

Active Comparator: Prophylactic Platelet Transfusion Group
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

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.


  Show Detailed Description

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01615146

Contacts
Contact: Elizabeth Chatelain, BScN 613 737-8899 ext 71264 echatelain@ohri.ca

Locations
Canada, Ontario
the Ottawa Hospital Recruiting
Ottawa, Ontario, Canada, k1h8l6
Contact: Elizabeth Chatelain    613 737 8899 ext 71264    echatelain@ohri.ca   
Principal Investigator: Alan Tinmouth         
Principal Investigator: Dawn Sheppard         
Sponsors and Collaborators
Ottawa Hospital Research Institute
Canadian Blood Services
Canadian Institutes of Health Research (CIHR)
Investigators
Principal Investigator: Alan Tinmouth, MD, MSc Ottawa Hospital Research Institute
  More Information

No publications provided

Responsible Party: Ottawa Hospital Research Institute
ClinicalTrials.gov Identifier: NCT01615146     History of Changes
Other Study ID Numbers: OHREB 2011500
Study First Received: June 6, 2012
Last Updated: January 22, 2013
Health Authority: Canada: Ethics Review Committee

Keywords provided by Ottawa Hospital Research Institute:
Myelodysplasia
Leukemia
Thrombocytopenia
Platelet
transfusions
Outpatient

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Preleukemia
Leukemia
Syndrome
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Neoplasms by Histologic Type
Disease
Pathologic Processes

ClinicalTrials.gov processed this record on October 19, 2014