Myelodysplastic SyndromeOPTIMALNCT01615146
Outpatient Platelet Transfusions in Myelodysplastic Syndromes and Leukemia: The OPTIMAL Pilot
A study testing Platelet Transfusion in people with Myelodysplastic Syndrome and Leukemia, run by Ottawa Hospital Research Institute at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome, Leukemia
- What is being tested?
- Platelet Transfusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteCanada
- Study dates
- Jun 2012to Jun 2015
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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)
Show all 11Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read more
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.
Read the detailed description
Randomization:
A local study nurse will use a web-based randomization system (permuted random blocks of two or four patients) to allocate consenting patients. Medical and research staff and investigators will be blinded to randomization scheme.
Study Duration and Follow-up:
All patients will be assigned to either a therapeutic or prophylactic platelet transfusion for a 6-month period. This period of time will allow us to assess our primary feasibility outcomes of enrollment, compliance with transfusion protocols, and completion of bleeding evaluations and quality of life questionnaires by patients.
Patients in both groups will have their CBC measured at least weekly while on study. More frequent monitoring of the platelet count may be performed at the discretion of the treating physician. The research team will clinically assess patients within 1 week (+/- 3 days) of randomization, and monthly thereafter. Patients will be asked to report any non-cutaneous grade 2 or greater bleeding immediately to their treating physicians and the study team. All the self-assessments of bleeding will be reviewed at each patient visit to ensure that all clinically relevant bleeding episodes have been captured.
Patients whose platelet count recovers to greater than 20 x 109/L for at least six weeks will be taken off the weekly platelet count monitoring as reflects clinical practice. They will continue to be monitored for the duration of the study. If their platelet count falls to 10 x 109/L or below then they will be restarted on the monitoring and previous transfusion protocol.
Data Collection:
Baseline Data - The following clinical and laboratory data will be collected at the time of enrolment: (1) demographic data, (2) diagnosis including date and disease stage, (3) prior chemotherapy, (4) ECOG performance status, (5) comorbidites, (6) previous platelet and red cell transfusions, (7) red cell transfusion history, (8) prior bleeding events, (9) quality of life (EQ5D) (10) routine bloodwork
Transfusions:
All platelet and red cell transfusions will be recorded including date and number of units. For platelet transfusions, the type of platelet product (e.g. apheresis or buffy coat) and ABO compatibility will recorded. Additionally, the indication for all transfusions (prophylactic or to treat bleeding) will be obtained from the attending physician.
Bleeding Assessment:
Participants will be asked to complete a simple bleeding questionnaire on a daily basis which has been previously tested and used in outpatients with thrombocytopenia. Patients will receive a short training session and written material on how to complete the form. A bleeding assessment will be performed at all study follow-up visits (interview, physical exam and review of daily bleeding forms). Bleeding events will be assigned a bleeding grade by the study personnel. A final bleeding grade will be assigned by an adjudication panel of 2 blinded physicians who will independently assign bleeding scores. Any discrepancies in bleeding scores will be resolved by consensus.
Quality of Life:
Quality of life will be measured using the EQ-5D. The EQ-5D is a validated tool for measurement of health-related quality of life there is a precedent for its use in transfusion medicine trials. Quality of life assessments will be performed at baseline and at all subsequent patient visits.
- Conditions
- Myelodysplastic Syndrome, Leukemia
- Being tested
- Platelet Transfusion
- Keywords
- Myelodysplasia, Leukemia, Thrombocytopenia, Platelet, transfusions, Outpatient
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Anemia, Refractory, with Excess of Blasts, Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Platelet TransfusionOther
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.
| Study group | Role | What they receive |
|---|---|---|
| Therapeutic Platelet Transfusion Arm | Experimental | Patients allocated to the therapeutic platelet transfusion group will not receive routine prophylactic platelet transfusions. Receives: 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. Receives: Other: Platelet Transfusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 18 months
Secondary outcomes (1)
Bleeding events between therapeutic and Prophylactic transfusion groups
Assessments will include:
- Non-cutaneous Grade 2 bleeding or higher by the World Heath Organization (WHO) bleeding assessment scale 28(Appendix B)
- Grade 3 bleeding or higher
- Time from randomization to first bleeding event of grade 3 of higher
- Total number of red cell transfusion per group
- Total number of hospital days per group
- Number of completed daily bleeding assessments per group
- Quality of life
- Mortality
Measured: 6 month follow up period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2012 (estimated)
- Study startJun 2012
- Primary completionJun 2015
- Study completionJun 2015
Why it stopped: Poor recruitment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromeStudies of the same condition, open and under way.
- More trials run by Ottawa Hospital Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ottawa Hospital Research Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Canadian Blood Services, Canadian Institutes of Health Research (CIHR)
- Responsible party
- Sponsor
- Investigators
- Alan Tinmouth, MD, MSc, Principal investigator, Ottawa Hospital Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01615146
- Sponsor's protocol ID
- OHREB 2011500
- Acronym
- OPTIMAL
- Status last verified
- Sep 2015
- First submitted
- 6 Jun 2012
- Last updated
- 2 Sep 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Outpatient Platelet Transfusions in Myelodysplastic Syndromes and Leukemia: The OPTIMAL Pilot
- ClinicalTrials.gov ID
- NCT01615146
- Acronym / sponsor ID
- OPTIMAL / OHREB 2011500
- Conditions
- Myelodysplastic Syndrome, Leukemia
- Keywords
- Myelodysplasia, Leukemia, Thrombocytopenia, Platelet, transfusions, Outpatient
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Poor recruitment
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Ottawa Hospital Research InstituteUniversity, hospital or other
- Collaborators
- Canadian Blood Services, Canadian Institutes of Health Research (CIHR)
- Study start
- Jun 2012
- Primary completion
- Jun 2015 (actual)
- Study completion
- Jun 2015 (actual)
- First posted
- 8 Jun 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Randomization:
A local study nurse will use a web-based randomization system (permuted random blocks of two or four patients) to allocate consenting patients. Medical and research staff and investigators will be blinded to randomization scheme.
Study Duration and Follow-up:
All patients will be assigned to either a therapeutic or prophylactic platelet transfusion for a 6-month period. This period of time will allow us to assess our primary feasibility outcomes of enrollment, compliance with transfusion protocols, and completion of bleeding evaluations and quality of life questionnaires by patients.
Patients in both groups will have their CBC measured at least weekly while on study. More frequent monitoring of the platelet count may be performed at the discretion of the treating physician. The research team will clinically assess patients within 1 week (+/- 3 days) of randomization, and monthly thereafter. Patients will be asked to report any non-cutaneous grade 2 or greater bleeding immediately to their treating physicians and the study team. All the self-assessments of bleeding will be reviewed at each patient visit to ensure that all clinically relevant bleeding episodes have been captured.
Patients whose platelet count recovers to greater than 20 x 109/L for at least six weeks will be taken off the weekly platelet count monitoring as reflects clinical practice. They will continue to be monitored for the duration of the study. If their platelet count falls to 10 x 109/L or below then they will be restarted on the monitoring and previous transfusion protocol.
Data Collection:
Baseline Data - The following clinical and laboratory data will be collected at the time of enrolment: (1) demographic data, (2) diagnosis including date and disease stage, (3) prior chemotherapy, (4) ECOG performance status, (5) comorbidites, (6) previous platelet and red cell transfusions, (7) red cell transfusion history, (8) prior bleeding events, (9) quality of life (EQ5D) (10) routine bloodwork
Transfusions:
All platelet and red cell transfusions will be recorded including date and number of units. For platelet transfusions, the type of platelet product (e.g. apheresis or buffy coat) and ABO compatibility will recorded. Additionally, the indication for all transfusions (prophylactic or to treat bleeding) will be obtained from the attending physician.
Bleeding Assessment:
Participants will be asked to complete a simple bleeding questionnaire on a daily basis which has been previously tested and used in outpatients with thrombocytopenia. Patients will receive a short training session and written material on how to complete the form. A bleeding assessment will be performed at all study follow-up visits (interview, physical exam and review of daily bleeding forms). Bleeding events will be assigned a bleeding grade by the study personnel. A final bleeding grade will be assigned by an adjudication panel of 2 blinded physicians who will independently assign bleeding scores. Any discrepancies in bleeding scores will be resolved by consensus.
Quality of Life:
Quality of life will be measured using the EQ-5D. The EQ-5D is a validated tool for measurement of health-related quality of life there is a precedent for its use in transfusion medicine trials. Quality of life assessments will be performed at baseline and at all subsequent patient visits.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Therapeutic Platelet Transfusion Arm | Experimental | Other: Platelet TransfusionPatients allocated to the therapeutic platelet transfusion group will not receive routine prophylactic platelet transfusions. |
| Prophylactic Platelet Transfusion Group | Active comparator | Other: Platelet TransfusionPatients 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Platelet TransfusionPatients 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. | Other | Prophylactic Platelet Transfusion Group, Therapeutic Platelet Transfusion Arm |
Primary outcome
Feasibility
Time frame: 18 months
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.
1 secondary outcome
Bleeding events between therapeutic and Prophylactic transfusion groups
Time frame: 6 month follow up period
Results and safety
The study was stopped early (reason given: Poor recruitment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alan Tinmouth, MD, MSc principal investigator
Ottawa Hospital Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Platelet Transfusion in people with Myelodysplastic Syndrome and Leukemia, run by Ottawa Hospital Research Institute at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 2 Sep 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Poor recruitment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Feasibility (time frame: 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Sep 2015.