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Myelodysplastic SyndromesSMD-transfuNCT03643042

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

A study testing Transfusion in people with Myelodysplastic Syndromes and Transfusion-dependent Anemia, run by Lille Catholic University at 19 sites in France.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Transfusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
11 participants
Where
19 sitesFrance
Study dates
24 Mar 2021to 22 Mar 2023
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

  • 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

Who cannot join

  • 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

Trial locations and contacts

19 sites in 1 country.

19 sites in 1 country

France19 sites

  • Abbeville CH

    Abbeville, 80142

  • Amiens CHU

    Amiens, 80054

  • Arras CH

    Arras, 62000

  • Henri Duffaut CH

    Avignon, 84000

  • Besançon CHU

    Besançon, 25030

  • Bordeaux CHU

    Bordeaux, 33604

  • Cote de Nacre CHU

    Caen, 14033

  • Clermont-Ferrand CHU

    Clermont-Ferrand, 63000

  • Dunkerque CH

    Dunkirk, 59140

  • Grenoble CHU

    Grenoble, 38043

  • Le Mans CH

    Le Mans, 72037

  • Lens CH

    Lens, 62307

  • St-Vincent Hospital

    Lille, 59020

  • Limoges CHRU

    Limoges, 87042

  • Meaux CH

    Meaux, 77104

  • Archet 1 Hospital

    Nice, 06202

  • Saint-Louis Hospital, APHP

    Paris, 75475

  • Pontchaillou Hospital

    Rennes, 35033

  • Roubaix CH

    Roubaix, 59100

Other trials in France

About this study

Official title: 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.

Read more

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
Read the 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
Being tested
Transfusion
Keywords
Myelodysplastic Syndromes, Transfusion, Quality of life, Physical performance
Medical subject headings
Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TransfusionOther

    Transfusion with Hb maintain between 80 and 100g/L or Hb maintain between 100 and 120g/L

Study groups in NCT03643042
Study groupRoleWhat they receive
Restrictive groupExperimentalTransfusion with: Hb < 80g/L and Hb maintain between 80 and 100g/L
Receives: Other: Transfusion
Liberal groupExperimentalTransfusion with: Hb < 100g/L and Hb maintain between 100 and 120g/L
Receives: Other: Transfusion

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Quality of Life by Myelodysplasia Scale (QUALMS) score

    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.

    Measured: six months post-randomization

Secondary outcomes (5)

  1. Quality of Life by Myelodysplasia Scale (QUALMS) score over the twelve months of follow-up

    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.

    Measured: 3, 6, 12 Months

  2. Timed up and go test over the twelve months of follow-up

    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

    Measured: 3, 6, 12 Months

  3. Transfusion incidents rate over the twelve months of follow-up

    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)

    Measured: 3, 6, 12 Months

  4. Transfusion costs over the twelve months of follow-up

    Transfusion costs (number of Packed red blood cells (PRBC) used) during the twelve months of follow-up

    Measured: 3, 6, 12 Months

  5. Time of occurrence of diagnosis of heart and liver damage due to transfusional iron overload over twelve months of follow-up

    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.

    Measured: 3, 6, 12 Months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Aug 2018
  2. Study start24 Mar 2021
  3. Primary completion21 Sep 2022
  4. Study completion22 Mar 2023

Why it stopped: The SMD SEUIL TRANSFU study was stopped prematurely because we encountered enrolment difficulties and new treatments are available on the market for patients with RARS or SFR3B1 mutation (Luspatercept).

Resources

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
Prevention
Allocation
Randomised
Intervention model
Parallel groups
Model description
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
Masking
None (open label)
Enrolment
11 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03643042
Sponsor's protocol ID
RC-P0071
Acronym
SMD-transfu
Status last verified
Jun 2023
First submitted
21 Aug 2018
Last updated
23 Jun 2023
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A study testing Transfusion in people with Myelodysplastic Syndromes and Transfusion-dependent Anemia, run by Lille Catholic University at 19 sites in France.

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 7 inclusion and 8 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 23 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 sites in France. 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: The SMD SEUIL TRANSFU study was stopped prematurely because we encountered enrolment difficulties and new treatments are available on the market for patients with RARS or SFR3B1 mutation (Luspatercept).). 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?

Quality of Life by Myelodysplasia Scale (QUALMS) score (time frame: six months post-randomization). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03643042.

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 23 Jun 2023.

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