Myelodysplastic syndromes Cancer Myelodysplastic syndromes
Conditions
Interventions
All eligible, informed and consented patients will receive the following two transfusion treatment interventions as below, with randomised allocation of the treatment sequence (i.e. AB or BA).
Arm A
Sponsors
Monash University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 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.
Exclusion criteria
Exclusion criteria: 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Time interval between transfusions measured using patient records. The feasibility of delivering a weekly transfusion schedule is defined by a difference in median time between transfusions of >7 days between the two treatment arms | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Number of RBC units transfused per arm measured using lab IT system at end of 12 weeks study period 2. Proportion of transfusions in the weekly arm provided with fully matched RBC units measured using lab IT system at end of 12 weeks study period 3. Proportion of Hb measures falling below, within, and above the target range (to ensure the transfusion strategy maintains Hb levels in the target ranges) measured using blood test at 3 month follow up 4. Time from patient admission to the transfusion centre until the commencement of RBC transfusion measured using trial worksheet timings at end of 12 weeks study period 5. Quality of life measured at randomisation & pre-transfusion 5.1. QUALMS-1 5.2. EQ-5D-5L 5.3. EORTC-QLQ-C30 6. Outcomes of rehabilitation measured using CIQ-R at randomisation & study visit 2 (week 6) 7. Functional activity thresholds and measures (via 6-minute walk test, handgrip strength and accelerometer): 7.1. Accelerometer: at enrolment visit, study visit 1 (week 3), study visit 3 (week 9) 7.2. Functional test (6MWT & Grip): Using Dynamometer and tape measure at enrolment visit, randomisation, pre-transfusion at last transfusion visits in each arm. 8. Numbers of adverse events, including transfusion reactions, per arm, and others e.g. disease progression measured using participant notes/review appointments at Randomisation, Study visit 1 (week 3), study visit 2 (week 6), 3 month follow up 9. Number of patients developing new RBC alloantibodies measured using lab IT system at end of 12 weeks study period 10. Enrolment rates; number of screening failures measured using As and when using Enrolment log 11. Effect of more frequent transfusions on iron biomarker assays measured using stored biomarker samples at future time 12. Estimates of carryover effect between different transfusion treatments measured using statistical analysis at trial end 13. Exploring patient and staff experiences of the weekly transfusion strategy measured using struct | — |
Countries
England, United Kingdom
Outcome results
None listed