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Trial record 4 of 12 for:    QUALMS

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

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ClinicalTrials.gov Identifier: NCT03643042
Recruitment Status : Recruiting
First Posted : August 22, 2018
Last Update Posted : April 12, 2022
Sponsor:
Information provided by (Responsible Party):
Lille Catholic University

Brief 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. 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

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndromes Transfusion-dependent Anemia Other: Transfusion Not Applicable

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 174 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention 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)
Primary Purpose: Prevention
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
Actual Study Start Date : March 24, 2021
Estimated Primary Completion Date : November 2022
Estimated Study Completion Date : May 2025


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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. 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)

  4. 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

  5. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03643042


Contacts
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Contact: Amélie Lansiaux, Md, PhD +33 3 20 22 52 69 ext +33 lansiaux.amelie@ghicl.net
Contact: Anne-Sophie Blain, CRA +33 3 20 22 57 32 ext +33 blain.anne-sophie@ghicl.net

Locations
Show Show 19 study locations
Sponsors and Collaborators
Lille Catholic University
Investigators
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Study Director: Laurent Pascal, MD GHICL
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Responsible Party: Lille Catholic University
ClinicalTrials.gov Identifier: NCT03643042    
Other Study ID Numbers: RC-P0071
First Posted: August 22, 2018    Key Record Dates
Last Update Posted: April 12, 2022
Last Verified: April 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Lille Catholic University:
Myelodysplastic Syndromes
Transfusion
Quality of life
Physical performance
Additional relevant MeSH terms:
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Preleukemia
Myelodysplastic Syndromes
Syndrome
Disease
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms