- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02099669
Red Blood Cell Transfusion Thresholds and QOL in MDS (EnhanceRBC)
April 24, 2018 updated by: Sunnybrook Health Sciences Centre
Red Blood Cell Transfusion Thresholds and QOL in MDS (EnhanceRBC): a Pilot, Feasibility Study
Myelodysplastic syndromes (MDS) are bone marrow malignancies characterized by poor bone marrow function that may progress to acute myeloid leukemia.
Many patients become red blood cell transfusion-dependent.
Transfusion dependence is associated with inferior quality of life (QOL).
However, the relationship between the degree of anemia and QOL is less clear.
A commonly used transfusion strategy is to target the hemoglobin (Hb) in the range of 80-90 g/L (normal hemoglobin > 120-130g/L).
The question is: would a higher hemoglobin target lead to improvement in QOL despite the negative impact transfusion dependence may have on QOL (due to associated time commitments, expense, transfusion reactions etc).
Several groups have prospectively shown that targeting hemoglobin levels of greater than 120 g/L (with hematopoietic growth factors (HGFs) and/or blood transfusions) or incremental increases of 15-20 g/L (with HGFs alone) were associated with improved QOL.
The investigators MDS program has been conducting prospective assessments of QOL since 2007 in all registered and consented patients using a variety of validated questionnaires.
Preliminary analysis (in 236 patients) revealed that, compared with an age-matched healthy general population, MDS patients have inferior QOL.
Transfusion dependence and anemia were independently predictive of poor functioning, fatigue and decreased health utility.
Furthermore, a hemoglobin level of ≥100 g/L seemed to be the key threshold for improvement in function and symptom scores.
The investigators hypothesize that the target hemoglobins in transfusion dependent MDS patients are too low and this may account for their inferior quality of life.
Our goal is to compare the effect on QOL of a restrictive strategy (which is the current standard of care), with a liberal transfusion strategy in a large randomized controlled trial of transfusion dependent MDS outpatients.
Before embarking on such an endeavor, the investigators must first prove feasibility in a smaller pilot randomized controlled trial.
Study Overview
Status
Unknown
Intervention / Treatment
Detailed Description
Myelodysplastic syndromes (MDS) are bone marrow malignancies characterized by poor bone marrow function that may progress to acute myeloid leukemia.
Many patients become red blood cell transfusion-dependent.
Transfusion dependence is associated with inferior quality of life (QOL).
However, the relationship between the degree of anemia and QOL is less clear.
A commonly used transfusion strategy is to target the hemoglobin (Hb) in the range of 80-90 g/L (normal hemoglobin > 120-130g/L).
The question is: would a higher hemoglobin target lead to improvement in QOL despite the negative impact transfusion dependence may have on QOL (due to associated time commitments, expense, transfusion reactions etc).
Several groups have prospectively shown that targeting hemoglobin levels of greater than 120 g/L (with hematopoietic growth factors (HGFs) and/or blood transfusions) or incremental increases of 15-20 g/L (with HGFs alone) were associated with improved QOL.
The investigators MDS program has been conducting prospective assessments of QOL since 2007 in all registered and consented patients using a variety of validated questionnaires.
Preliminary analysis (in 236 patients) revealed that, compared with an age-matched healthy general population, MDS patients have inferior QOL.
Transfusion dependence and anemia were independently predictive of poor functioning, fatigue and decreased health utility.
Furthermore, a hemoglobin level of ≥100 g/L seemed to be the key threshold for improvement in function and symptom scores.
The investigators hypothesize that the target hemoglobins in transfusion dependent MDS patients are too low and this may account for their inferior quality of life.
Our goal is to compare the effect on QOL of a restrictive strategy (which is the current standard of care), with a liberal transfusion strategy in a large randomized controlled trial of transfusion dependent MDS outpatients.
Before embarking on such an endeavor, the investigators must first prove feasibility in a smaller pilot randomized controlled trial.
Study Type
Interventional
Enrollment (Anticipated)
30
Phase
- Not Applicable
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Jennifer Gallagher
- Email: jennifer.gallagher@sunnybrook.ca
Study Contact Backup
- Name: Geetha Yogendron
Study Locations
-
-
-
Toronto, Canada
- Recruiting
- Princess Margaret Hospital
-
Contact:
- Karen Yee, MD
-
Sub-Investigator:
- Karen Yee, MD
-
Toronto, Canada
- Recruiting
- Sunnybrook Health Sciences Centre/Odette Cancer Centre
-
Contact:
- Jennifer Gallagher
- Email: jennifer.gallagher@sunnybrook.ca
-
Principal Investigator:
- Rena Buckstein
-
-
Ontario
-
Hamilton, Ontario, Canada
- Recruiting
- Juravinski Cancer Centre
-
Contact:
- Erin Jamula, MSc
- Phone Number: 76461 905-521-2100
- Email: jamulae@mcmaster.ca
-
Principal Investigator:
- Brian Leber, MD
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- All patients with MDS ≥18 years of age
- Transfusion dependent: at least 1 transfusion per month in the last 8 weeks
- Hb <100g/L pre transfusion
- Life expectancy > 6 months
Exclusion Criteria:
- Unstable cardiac disease (Canadian Cardiovascular Society (CCS) III/IV angina or New York Heart Association (NYHA) III/IV congestive heart failure) requiring the transfusion target range to remain >85-100 g/L at all times
- ECOG ≥3
- Patients with red cell antibodies against high frequency antigens or multiple antibodies (would potentially delay finding blood)
- Patients on ESA's or disease modifying agents (like azacitidine) for their MDS
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Liberal transfusion strategy
Intervention: Red Blood Cell transfusions. Transfuse pRBC at a higher threshold- maintain Hb level between 110 and 120 g/L: to achieve this, 2 units of pRBCs are transfused when Hb level is < 105 g/L and 1 unit of RBCs when Hb level is 105-110 g/L. Transfusions administered more frequently. |
|
Active Comparator: Restrictive transfusion strategy
Intervention: Red Blood Cell transfusions.
Transfuse pRBC at standard of care thresholds- maintain Hb level between 85 and 100 g/L: to achieve this, 2 units of packed red blood cells (pRBCs) will be transfused when the Hb level is < 80 g/L and 1 unit of pRBCs when Hb level is 80-85 g/L: standard administration
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Percentage compliance of q2weekly hemoglobins
Time Frame: 3 months
|
The percentage compliance of q2weekly hemoglobins being within or above the target range of the RBC transfusion threshold assigned (after the 4 week run-in at study start as defined above).
We will consider this study feasible and worthy of future development into a larger randomized trial (powered for QOL difference) if compliance is ≥70%.
A compliance rate of 50-70%, would not exclude going forward with such an RCT but only after careful discussion and statistical planning
|
3 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Measures of feasibility
Time Frame: 3 months
|
|
3 months
|
Quality of life
Time Frame: 3 months
|
• The magnitude of change in physical functioning, fatigue, dyspnea and global health scores on the EORTC QLQ-C30, calculated health utility on the EQ-5D and fatigue score on FACT-F comparing the 2 RBC transfusion thresholds above.
|
3 months
|
Adverse events
Time Frame: 3 months
|
|
3 months
|
Alloimmunization rates
Time Frame: 3 months
|
Rate of alloimmunization to significant blood group antigens
|
3 months
|
Hemosiderosis
Time Frame: 3 months
|
• The impact on transfusion associated hemosiderosis rates and burdens (as measured by changes in ferritin levels and iron chelating medications)
|
3 months
|
Overall utilization of blood
Time Frame: 3 months
|
How many units of blood are required to maintain the hemoglobin threshold chosen
|
3 months
|
Time commitment
Time Frame: 3 months
|
• The overall time commitment per group, measured as the time spent in transfusion medicine clinic
|
3 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Investigators
- Principal Investigator: Rena Buckstein, MD, Sunnybrook Health Sciences Centre/Odette Cancer Centre
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
March 1, 2014
Primary Completion (Anticipated)
December 1, 2018
Study Completion (Anticipated)
January 1, 2019
Study Registration Dates
First Submitted
March 26, 2014
First Submitted That Met QC Criteria
March 28, 2014
First Posted (Estimate)
March 31, 2014
Study Record Updates
Last Update Posted (Actual)
April 26, 2018
Last Update Submitted That Met QC Criteria
April 24, 2018
Last Verified
April 1, 2018
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- OCCMDS-005
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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