A randomised feasibility n-of-1 trial of weekly-interval red cell transfusion in myelodysplastic syndromes

Monash University0 个研究点目标入组 30 人开始时间: 2023年9月20日最近更新: 2023年10月16日

适应症

试验速览

暂无简介。

•1. All patients aged >=18 years with WHO-defined MDS or mixed myeloproliferative/myelodysplastic neoplasm overlap. syndromes (MPN/MDS)
•2. Transfusion-dependent: on average at least 1 RBC transfusion episode per month in the last 8 weeks (at least two transfusion episodes, each with at least two units of RBCs during the 16 weeks prior to study entry).
•3. Continuing transfusion requirement expected for at least 6 months.
•4. Life expectancy >= 6 months.
•5. No new treatments likely to affect transfusion requirements during the study period.
•6. Able to attend hospital to receive weekly transfusions.
•7. Able to complete QoL questionnaires.
•8. Able to participate in physical function tests (6-minute walk test, accelerometer to measures steps taken per day.)
•9. Red cell phenotyping and/or genotyping results available.
•10. For the qualitative study, able to understand and converse in conversational English for purposes of interviews.

•1. Unable to tolerate a weekly transfusion schedule as determined by the attending clinician
•2. Poor performance/functional status (Eastern Cooperative Oncology Group system; ECOG >=3)
•3. Patients on Erythropoietic-Stimulating Agent (ESA) or disease modifying agents for their MDS (such as Enalidomide, Azacitidine, Hydroxycarbamide, experimental agents), as these may exert their own effects on the patients’ QoL and transfusion requirements.
•4. Patients with myelofibrosis
•5. Patients presenting with active bleeding or evidence of significant haemolysis
•6. Splenomegaly >5cm below the costal margin
•7. Patients with >2 known RBC alloantibodies, and/or patients with rare antigen types which complicate cross-matching

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