Quality of Life-Guided Transfusion in Refractory MDS or AML (Q-TRANS)

Centre Hospitalier Universitaire de Nice

Status: Not yet enrolling

Conditions: Anemia; MDS; AML, Adult

Treatments: Other: Red blood cell transfusions based on EQ-5D-5L questionnaire

Study type: Interventional

Funder types: Other

Identifiers

NCT07328191

25-AOI-02

Details and patient eligibility

About

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.

Full 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.

Enrollment: 52 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Supportive Care

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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52 participants in 2 patient groups

Hemoglobin-Based Transfusion Strategy

No Intervention group

Description:

Red blood cell transfusions are performed according to standard hemoglobin thresholds (Hb \<7 g/dL, or \<8 g/dL in patients with cardiovascular comorbidities). Standard of care arm

Quality of Life-Guided Transfusion Strategy

Experimental group

Description:

Red blood cell transfusions are performed when a clinically significant deterioration (≥1 level decrease in at least one EQ-5D-5L domain compared to baseline) is observed

Treatment:

Other: Red blood cell transfusions based on EQ-5D-5L questionnaire