Impact of 2 Transfusion Strategies on Quality of Life of Multitransfused Patients With Low-risk Myelodysplastic Syndrome (SMD-transfu)

June 21, 2023 updated by: Lille Catholic University

Impact of 2 Transfusion Strategies on the Quality of Life of Multi-transfused Patients With Low Risk Myelodysplastic Syndrome: Multicenter Randomized Trial Comparing a Liberal vs. Restrictive Transfusion Regimen

Myelodysplastic syndromes (MDS) are heterogeneous malignant bone marrow disorders characterized by ineffective haematopoiesis, peripheral blood cytopenias and variable risk of leukaemia transformation.

Anemia is the most common manifestation of bone marrow failure in MDS. After failure with first-line treatment by Erythropoietin, patients survive in average 5 years under long term blood transfusion. Modalities of blood transfusion are not clearly defined.

Then, the objective of this randomized comparative multicentric study is to compare two modalities of threshold for transfusion:

  • Restrictive group: Hb < 80g/L and Hb maintain between 80 and 100g/L
  • Liberal group: Hb < 100g/L and Hb maintain between 100 and 120g/L

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Myelodysplastic syndromes (MDS) are heterogeneous malignant bone marrow disorders characterized by ineffective haematopoiesis, peripheral blood cytopenias and variable risk of leukaemia transformation. The median age at diagnosis is 75 years. The incidence is about 30 per 100,000, over 70 years. Etiology is unknown in more than 85% of cases, chemo-induced causes and family cases are well individualized.

Diagnosis, prognosis, and classification (WHO) are based on joint cytologic analysis of peripheral blood, bone marrow, and spinal cytogenetic analysis. The main therapeutic objectives in low-risk MDS are to correct cytopenias, improve quality of life and prevent aggravation of co-morbidities.

Anemia is the most common manifestation of bone marrow failure in MDS. It is encountered in 80% of cases at diagnosis and almost always occurs in the progression of the disease. Its presence and importance have a pejorative prognostic value, but it is not clear whether this anemia is indicative of a more serious clonal disease or whether it is the repercussions of anemia that lead to a more severe prognosis. After failure with first-line treatment by Erythropoietin (EPO), patients survive in average 5 years under long term blood transfusion. Modalities of blood transfusion are not clearly defined.

Studies in the general geriatric population and in cases of acute anemia are in favor of a restrictive transfusion regimen (threshold around 70 g/L), while experience during MDS with EPO suggest that maintaining a higher hemoglobin count could have a favorable impact on quality of life, physical performance, or even survival of patients with MDS.

Then, the objective of this randomized comparative multicentric study is to compare two modalities of threshold for transfusion:

  • Restrictive group: Hb < 80g/L and Hb maintain between 80 and 100g/L
  • Liberal group: Hb < 100g/L and Hb maintain between 100 and 120g/L

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
11

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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 Locations

  • France
      • Abbeville, France, 80142
        • Abbeville CH
      • Amiens, France, 80054
        • Amiens Chu
      • Arras, France, 62000
        • Arras CH
      • Avignon, France, 84000
        • Henri Duffaut CH
      • Besançon, France, 25030
        • Besançon CHU
      • Bordeaux, France, 33604
        • Bordeaux CHU
      • Caen, France, 14033
        • Cote de Nacre CHU
      • Clermont-Ferrand, France, 63000
        • Clermont-Ferrand CHU
      • Dunkerque, France, 59140
        • Dunkerque CH
      • Grenoble, France, 38043
        • Grenoble Chu
      • Le Mans, France, 72037
        • Le Mans CH
      • Lens, France, 62307
        • Lens CH
      • Lille, France, 59020
        • St-Vincent Hospital
      • Limoges, France, 87042
        • Limoges CHRU
      • Meaux, France, 77104
        • Meaux CH
      • Nice, France, 06202
        • Archet 1 Hospital
      • Paris, France, 75475
        • Saint-Louis Hospital, APHP
      • Rennes, France, 35033
        • Pontchaillou Hospital
      • Roubaix, France, 59100
        • roubaix CH

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Patients with low risk or intermediate risk MDS: Revised International Prognostic Scoring System (IPSS-R) less than or equal to 4.5
  • Relapse or failure after Erythropoiesis-Stimulating Agent (ESA) therapy or others treatments (Lenalidomide, Thalidomide, 5-Azacytidine, antithymocyte globulin (ATG), Luspatercept, Decitabine, allograft)
  • Transfusion dependent: in average at least 3 transfusion episodes in the last 6 months and total of packed red blood cells (PRBC): more than 8 in the last 12 months and less than 150 in total.
  • ≥ 18 years of age
  • The Eastern Cooperative Oncology Group (ECOG) score < 4
  • Life expectancy > 12 Months
  • Patients willing to participate in the study and who have signed the informed consent form

Exclusion Criteria:

  • Patients with disease modifying agents for their MDS such as: ESA therapy, Lenalidomide, Thalidomide revlimid, Vidaza, Allograft, antithymocyte globulin (ATG), Luspatercept, Decitabine, experimental agents, other clinical trial, taken within 3 months prior to inclusion (chelators are accepted)
  • According to physician: unable to tolerate restrictive or liberal red cell transfusion thresholds (e.g. clinically significant cardio-respiratory failure)
  • Cognitive alteration (inability to complete QUALMS)
  • Inability to perform the physical performance test Timed up and go test
  • Splenomegaly > 3 cm below the costal margin
  • Severe renal failure with creatinine clearance < 30ml / min
  • Patients presenting with active bleeding or evidence of significant haemolysis
  • Patient under guardianship or curatorship

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: Prevention
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Restrictive group
Transfusion with: Hb < 80g/L and Hb maintain between 80 and 100g/L
Other: Transfusion
Transfusion with Hb maintain between 80 and 100g/L or Hb maintain between 100 and 120g/L
Experimental: Liberal group
Transfusion with: Hb < 100g/L and Hb maintain between 100 and 120g/L
Other: Transfusion
Transfusion with Hb maintain between 80 and 100g/L or Hb maintain between 100 and 120g/L

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Quality of Life by Myelodysplasia Scale (QUALMS) score
Time Frame: six months post-randomization
Quality of Life assessed by a specific validated and adapted disease scale :Quality of Life by Myelodysplasia Scale (QUALMS) score at six months post-randomization. The QUALMS consists of 38 items, and takes less than 10 minutes to complete. Scored on a scale of 0 to 100 higher score is correlated with better MDS-specific quality of life.
six months post-randomization

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Quality of Life by Myelodysplasia Scale (QUALMS) score over the twelve months of follow-up
Time Frame: 3, 6, 12 Months
Evolution of the QUALMS score over the twelve months of follow-up. Scored on a scale of 0 to 100 higher score is correlated with better MDS-specific quality of life.
3, 6, 12 Months
Timed up and go test over the twelve months of follow-up
Time Frame: 3, 6, 12 Months
Evolution of physical performance (the time required for the Timed up and go test) at six months post-randomization and over the twelve months of follow-up
3, 6, 12 Months
Transfusion incidents rate over the twelve months of follow-up
Time Frame: 3, 6, 12 Months
Transfusion incidents rate during the twelve months of follow-up among allo-immunization, hospitalization for pulmonary overload, iron overload (ferritin, transferrin saturation), TRALI (Transfusion-Related Acute Lung Injury)
3, 6, 12 Months
Transfusion costs over the twelve months of follow-up
Time Frame: 3, 6, 12 Months
Transfusion costs (number of Packed red blood cells (PRBC) used) during the twelve months of follow-up
3, 6, 12 Months
Time of occurrence of diagnosis of heart and liver damage due to transfusional iron overload over twelve months of follow-up
Time Frame: 3, 6, 12 Months
Time of occurrence of diagnosis of heart and liver damage due to transfusional iron overload over twelve months of follow-up. The diagnosis will be established according to the standard procedure based on annual MRI, in particular by measuring the LIC (liver iron concentration), the MIC (myocardial iron concentration) and the cardiac T2* value.
3, 6, 12 Months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Lille Catholic University

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Laurent Pascal, MD, GHICL

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 (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 24, 2021

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 21, 2022

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 22, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 21, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 21, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 22, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 23, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 21, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • RC-P0071

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

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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