Clinical trial NCT06614595: Transfusion in Lower Risk MDS Patients: Predictors of Adequacy of Transfusion and Quality of Life in Lower Risk MDS
Official title: Transfusion in Lower Risk MDS Patients: Predictors of Adequacy of Transfusion and Quality of Life Changes
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Low Risk Myelodysplastic Syndromes
Keywords: Myelodysplastic Syndromes
Red blood cell transfusion
Quality of life
Lead sponsor: Fundación para la Investigación Biosanitaria del Principado de Asturias
Sponsor class: OTHER
Collaborators: name: Hospital Universitario Central de Asturias
First posted: 2024-09-26
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Lower-risk Myelodysplastic syndrome patients, red blood cell transfusion dependent

Brief summary: Observational study to evaluate changes in quality of life (QoL) in patients diagnosed with low risk MDS who are red blood cell (abc) transfusion dependent.

Changes in QoL will be correlated with patient cardiorespiratory function. Cardiac biomarkers and plasma cytokines will also be evaluated.

Detailed description: At inclusion and before red blood cell (rbc) transfusion, patients cardiorespiratory function will be evaluated with an ergospirometer

Likewise, before and after rbc transfusion, patients will be asked to complete the QUALMS, a QoL instrument which will be supplied by Dr. Gregory Abel at Dana-Farber in the U.S., who led its development team.

Before and after rbc transfusion, hemoglobin, pretrasfusional hemoglobin, pre and post cardiac biomarkers troponin and NTproBNP will be collect.Pre and post serum and plasma will also be collected for cytokine analysis.

Eligibility criteria: Subjects must meet all of the following inclusion criteria:

1. They must be able to understand the study procedures, comply with them, and consent in writing prior to any specific study procedure.
2. Must be able to understand the study procedures, comply with them, and give written informed consent prior to any study-specific procedure.
3. Adult subjects ≥ 18 years of age with a diagnosis of myelodysplastic syndrome.
4. Very low, low or intermediate IPSS-R risk category.
5. Subject requires are red blood cell transfusion dependent with the following criteria: requires an average of 2 to 6 units of CH on average over an 8-week period.
6. ECOG 0-3.

Exclusion criteria

1. Subject undergoing any of the following treatments: azacitidine, decitabine, venetoclax, cytotoxic chemotherapy, radiotherapy, arsenic trioxide, interferon or interleukin.
2. High or very high IPSS-R risk category.
3. Subject diagnosed with any active neoplasm except:

   * epidermoid or basal cell carcinoma,
   * carcinoma in situ of the uterine cervix
   * Carcinoma in situ of the breast
4. Subjects with a score on the New York Heart Association Scale IV.
5. Subjects with major surgery within 8 weeks prior to study inclusion. Subjects must have fully recovered from any previous surgery prior to inclusion in the study.
6. Subject with known clinically significant anaemia due to iron, vitamin B12 or folate deficiencies or autoimmune or hereditary haemolytic anaemia or uncontrolled hypothyroidism or known clinically significant haemorrhage or sequestration or subject with drug induced anaemia.
7. New onset or uncontrolled seizures.
8. Subjects with uncontrolled systemic fungal, bacterial or viral infections (defined as ongoing signs or symptoms related to the infection without improvement despite appropriate antibiotics or antimicrobial therapy).
9. Pregnant or breastfeeding women.
10. The subject has any condition, including the presence of laboratory abnormalities that would place the subject at unacceptable risk if he/she were to participate in the study.
11. The subject has any condition or receives concomitant medication that confounds the ability to interpret the study data.

Primary outcomes:
measure: Prediction of Quality of Life (QoL) improvement, assessed by an increase in 5 points or more in the QUALMs scale
timeFrame: We expect to obtain results in one year from the beginning of the study
Secondary outcomes:
measure: Changes in QoL before and after transfusion
timeFrame: QoL will be asses the day of transfusion and seven days after
Other outcomes:
measure: Troponin and NTproBNP as predictors for QoL improvement
timeFrame: Troponin and NTproBNP will be analyzed the day of transfusion and seven days after
measure: Cardiorespiratory function
timeFrame: Ergospirometry will be performed at the inclusion in the study
measure: Cytokines
timeFrame: Cytokines will be analyzed before abc transfusion and seven days after it
Locations:
city: Oviedo
country: Spain
facility: Hospital Universitario Central de Asturias
state: Asturias
Study officials:
References:
citation: Abel GA, Efficace F, Buckstein RJ, Tinsley S, Jurcic JG, Martins Y, Steensma DP, Watts CD, Raza A, Lee SJ, List AF, Klaassen RJ. Prospective international validation of the Quality of Life in Myelodysplasia Scale (QUALMS). Haematologica. 2016 Jun;101(6):781-8. doi: 10.3324/haematol.2015.140335. Epub 2016 Mar 4.
citation: Abel GA, Klepin HD, Magnavita ES, Jaung T, Lu W, Shallis RM, Hantel A, Bahl NE, Dellinger-Johnson R, Winer ES, Zeidan AM. Peri-transfusion quality-of-life assessment for patients with myelodysplastic syndromes. Transfusion. 2021 Oct;61(10):2830-2836. doi: 10.1111/trf.16584. Epub 2021 Jul 12.