AML, Adult, Anemia, MDS
Conditions
Keywords
palliative care, Chronic Transfusion-Dependent Anemia, Transfusion-dependent anemia, MDS, AML
Brief summary
Patients with refractory myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) in exclusive palliative care frequently receive red blood cell transfusions based on hemoglobin thresholds, despite limited evidence of clinical benefit in this setting. This prospective randomized study compares a standard hemoglobin-based transfusion strategy to a quality-of-life-guided strategy using the EQ-5D-5L questionnaire, with the aim of reducing transfusion burden while maintaining patient safety and quality of life.
Detailed description
This is a prospective, single-center, randomized interventional study conducted in patients with transfusion-dependent refractory MDS or AML receiving exclusive palliative care. Palliative Care Patients are randomized to either a standard transfusion strategy based on hemoglobin thresholds or a quality-of-life-guided strategy in which red blood cell transfusions are triggered by a clinically significant deterioration in EQ-5D-5L score. The study aims to evaluate whether a quality-of-life-guided transfusion strategy can safely reduce the number of red blood cell transfusions without negatively impacting patient-reported outcomes.
Interventions
OTHERRed blood cell transfusions based on EQ-5D-5L questionnaire
Red blood cell transfusions are administered based on patient-reported quality-of-life assessments using the EQ-5D-5L questionnaire, collected weekly. A transfusion is performed when a deterioration of at least one level in any EQ-5D-5L dimension is observed.
Sponsors
Centre Hospitalier Universitaire de Nice
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
SUPPORTIVE_CARE
Masking
NONE
Intervention model description
Participants are randomly assigned in a 1:1 ratio to one of two parallel groups: a standard hemoglobin-based red blood cell transfusion strategy or a quality-of-life-guided transfusion strategy based on EQ-5D-5L assessments.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adults aged 18 years and older. * Diagnosis of refractory MDS or AML, receiving palliative care only. * Anaemia with haemoglobin ≤ 8 g/dl at inclusion (or ≤ 9 g/dl if associated with cardiovascular disease). * Transfusion dependency, defined as requiring more than 2 GR transfusions every 8 weeks. * No vitamin B9, vitamin B12 or iron deficiency. * Ability to understand and sign the informed consent form. * Ability to comply with the schedule of visits and other protocol requirements. Non inclusion Criteria: * Previous malignant disease other than MDS or AML (except basal cell or squamous cell carcinoma or carcinoma in situ of the cervix or breast). * Active uncontrolled infection (hepatitis B or C, HIV). * Use of G-CSF. * Confirmed neurocognitive disorders (based on prior diagnosis or clinical assessment by the investigator) impairing comprehension, consent or the ability to reliably complete the EQ-5D-5L questionnaire. * Active uncontrolled heart disease. * Active haemolytic anaemia. * Recent major surgery. * Life-threatening complications of MDS/AML. * Presence of another serious or unstable disease which, in the investigators' opinion, would make participation in the study inappropriate or risky for the patient's safety. * Vulnerable individuals.
Exclusion criteria
* Immediate severe complications related to MDS/AML, such as uncontrolled bleeding, pneumonia with hypoxia or shock, or severe disseminated intravascular coagulopathy. * Withdrawal of the patient's voluntary informed consent.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Red Blood Cell Transfusions | 2 months | Total number of red blood cell units transfused during the 2-month follow-up period. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Quality of Life (EQ-5D-5L) | from baseline to 1 month and 2 months. | Change in quality-of-life scores |
| Overall Survival | from inclusion to 2 months | Survival status |
Countries
France
Contacts
CONTACTThomas Cluzeau, Professor
CONTACTLydia Cherfaoui
PRINCIPAL_INVESTIGATORThomas Cluzeau
Centre Hospitalier Universitaire de Nice
Outcome results
None listed