Transfusion in Lower Risk MDS Patients: Predictors of Adequacy of Transfusion and Quality of Life in Lower Risk MDS

NCT06614595 | Recruiting

• 1 other identifier: 2024.244
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 1

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.

Conditions

Low Risk Myelodysplastic Syndromes

Keywords

Myelodysplastic Syndromes; Red blood cell transfusion; Quality of life

Outcome Measures

Primary Outcomes (1)

• Prediction of Quality of Life (QoL) improvement, assessed by an increase in 5 points or more in the QUALMs scale

  To design a score that help to identify which patients benefit from red blood cell Transfusion in terms of QoL improvement

  We expect to obtain results in one year from the beginning of the study

Secondary Outcomes (1)

• Changes in QoL before and after transfusion

  QoL will be asses the day of transfusion and seven days after

Other Outcomes (3)

• Troponin and NTproBNP as predictors for QoL improvement

  Troponin and NTproBNP will be analyzed the day of transfusion and seven days after

• Cardiorespiratory function

  Ergospirometry will be performed at the inclusion in the study

• Cytokines

  Cytokines will be analyzed before abc transfusion and seven days after it

Eligibility Criteria

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

Study Population

Lower-risk Myelodysplastic syndrome patients, red blood cell transfusion dependent

You may qualify if:

• They must be able to understand the study procedures, comply with them, and consent in writing prior to any specific study procedure.
• Must be able to understand the study procedures, comply with them, and give written informed consent prior to any study-specific procedure.
• Adult subjects ≥ 18 years of age with a diagnosis of myelodysplastic syndrome.
• Very low, low or intermediate IPSS-R risk category.
• 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.
• ECOG 0-3.

You may not qualify if:

• Subject undergoing any of the following treatments: azacitidine, decitabine, venetoclax, cytotoxic chemotherapy, radiotherapy, arsenic trioxide, interferon or interleukin.
• High or very high IPSS-R risk category.
• 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
• Subjects with a score on the New York Heart Association Scale IV.
• 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.
• New onset or uncontrolled seizures.
• 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).
• Pregnant or breastfeeding women.
• 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.
• The subject has any condition or receives concomitant medication that confounds the ability to interpret the study data.

Sponsors & Collaborators

• Fundación para la Investigación Biosanitaria del Principado de Asturias: lead
• Hospital Universitario Central de Asturias: collaborator

Study Sites (1)

Hospital Universitario Central de Asturias

Oviedo, Principality of Asturias, 33011, Spain

RECRUITING

Related Publications (2)

• 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.

  PMID: 26944474 BACKGROUND

• 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.

  PMID: 34251040 BACKGROUND

Biospecimen

Retention: SAMPLES WITHOUT DNA

serum and plasma for cytokine analysis

MeSH Terms

Conditions

Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Teresa Bernal del Castillo

CONTACT

985108000; bernaldelcastillo@gmail.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator, MD, PHD

Study Record Dates

• First Submitted: September 24, 2024
• First Posted: September 26, 2024
• Study Start: September 20, 2024
• Primary Completion: December 20, 2025
• Study Completion: December 21, 2025
• Last Updated: September 27, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1)