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

Официальное название

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

Обзор

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.

Подробное описание

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.

Первичные конечные точки

• Prediction of Quality of Life (QoL) improvement, assessed by an increase in 5 points or more in the QUALMs scale [Срок оценки: We expect to obtain results in one year from the beginning of the study]

Вторичные конечные точки (1)

• Changes in QoL before and after transfusion [Срок оценки: QoL will be asses the day of transfusion and seven days after]

Критерии участия

Subjects must meet all of the following inclusion criteria:

• 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.

Критерии исключения

• 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.
• 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.
• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Испания · 1 центр

• Hospital Universitario Central de Asturias — Oviedo

Публикации

• 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
• 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

Идентификаторы

NCT: NCT06614595 · 2024.244

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗