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

Lille Catholic University

Status: Terminated

Conditions: Transfusion-dependent Anemia; Myelodysplastic Syndromes

Treatments: Other: Transfusion

Study type: Interventional

Funder types: Other

Identifiers

NCT03643042

RC-P0071

Details and patient eligibility

About

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

Full 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

Enrollment: 11 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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11 participants in 2 patient groups

Restrictive group

Experimental group

Description:

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

Treatment:

Other: Transfusion

Liberal group

Experimental group

Description:

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

Treatment:

Other: Transfusion