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

NCT03643042 | Terminated

• 1 other identifier: RC-P0071
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 19

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

Conditions

Myelodysplastic Syndromes; Transfusion-dependent Anemia

Keywords

Myelodysplastic Syndromes; Transfusion; Quality of life; Physical performance

Outcome Measures

Primary Outcomes (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.

  six months post-randomization

Secondary Outcomes (5)

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

  3, 6, 12 Months

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

  3, 6, 12 Months

• Transfusion incidents rate over the twelve months of follow-up

  3, 6, 12 Months

• Transfusion costs over 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

  3, 6, 12 Months

Study Arms (2)

Restrictive group

EXPERIMENTAL

Transfusion with: Hb \< 80g/L and Hb maintain between 80 and 100g/L

Other: Transfusion

Liberal group

EXPERIMENTAL

Transfusion with: Hb \< 100g/L and Hb maintain between 100 and 120g/L

Other: Transfusion

Interventions

Transfusion OTHER

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

Liberal group; Restrictive group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Lille Catholic University: lead

Study Sites (19)

Abbeville CH

Abbeville, 80142, France

Location

Amiens CHU

Amiens, 80054, France

Location

Arras CH

Arras, 62000, France

Location

Henri Duffaut CH

Avignon, 84000, France

Location

Besançon CHU

Besançon, 25030, France

Location

Bordeaux CHU

Bordeaux, 33604, France

Location

Cote de Nacre CHU

Caen, 14033, France

Location

Clermont-Ferrand CHU

Clermont-Ferrand, 63000, France

Location

Dunkerque CH

Dunkirk, 59140, France

Location

Grenoble CHU

Grenoble, 38043, France

Location

Le Mans CH

Le Mans, 72037, France

Location

Lens CH

Lens, 62307, France

Location

St-Vincent Hospital

Lille, 59020, France

Location

Limoges CHRU

Limoges, 87042, France

Location

Meaux CH

Meaux, 77104, France

Location

Archet 1 Hospital

Nice, 06202, France

Location

Saint-Louis Hospital, APHP

Paris, 75475, France

Location

Pontchaillou Hospital

Rennes, 35033, France

Location

Roubaix CH

Roubaix, 59100, France

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

Blood Transfusion

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics

Study Officials

• Laurent Pascal, MD

  GHICL

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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 Record Dates

• First Submitted: August 21, 2018
• First Posted: August 22, 2018
• Study Start: March 24, 2021
• Primary Completion: September 21, 2022
• Study Completion: March 22, 2023
• Last Updated: June 23, 2023

Record last verified: 2023-06

Data Sharing

• IPD Sharing: Will not share

Locations

FR (19)