Study Title

An n-of-1 feasibility trial of weekly interval red cell transfusion schedule in MDS (The REDDS-2 Study)

Coordinating Organisation

Transfusion Research Unit (TRU), Monash University, Australia

Universal Trial Number

U1111-1231-6575

ANZCTR Number

ACTRN12619001053112p

Sample Size

30 participants across Australia, UK, and the Netherlands

Background

Current standard of transfusion care in transfusion-dependent MDS is based on a policy for regular red blood cell transfusions that is typically multiple (2–4) units every 3–4 weeks. However, an alternative strategy, specifically lower dose and weekly transfusions of red cells, could be more effective and acceptable.

Current transfusion schedules are also characterised by multiple delays (e.g. planning transfusion in busy hospital units, requirements of comprehensive pre-transfusion testing). To facilitate timely delivery of weekly RBC transfusion, we will provide phenotype or genotype-matched RBCs, without the delays of awaiting prospective cross-matching results on the day of transfusion. Routine pre-transfusion testing results will inform the following week’s transfusion strategy.

Trial Objective

The REDDS-2 Study is a two-part study that will assess red blood cell transfusion schedules in patients with transfusion-dependent MDS.

The first part of the study is a pilot n-of-1 study to assess the feasibility of a weekly red blood cell transfusion schedule and to understand the relevant tools (quality of life [QoL], physical measures, and clinical outcomes) to be used to collect patient outcome data in a larger trial. N-of-1 studies are single patient clinical trials and focus on investigating the effectiveness of a treatment tailored for an individual patient. For this study, data from one individual will also be combined with data from other individuals participating in the study.

The second part is a qualitative study to explore the experiences of patients and staff members with the new transfusion schedule.The overarching objective of this study is to inform the design and conduct of a definitive trial to compare different red blood cell transfusion strategies in patients with transfusion-dependent MDS.

Aims

1. Establish the feasibility of delivering weekly red cell transfusions using matched red cells rather than waiting for results of contemporaneous cross-matching.
2. Determine the appropriate tools to measure the impact of variation in transfusion frequency on patients’ health-related QoL and physical function, including a primary outcome to be measured in a definitive trial.
3. Develop the intervention protocol for a trial for red cells transfusion. The ultimate goal is to undertake a definitive phase III randomised trial to compare different strategies for red cells transfusion in patients with transfusion-dependent MDS.
4. Explore patient and staff experiences of the new weekly transfusion schedule including: potential enablers, barriers, positive and negative experiences, acceptability and comparison to the standard transfusion schedule. This will provide greater in-depth understanding of the weekly transfusion schedule and provide information to develop future clinical transfusion trials.

Primary outcome

Feasibility of delivering a weekly transfusion schedule, as defined by a difference in median time between transfusions of ≥7 days between the two treatment arms.

Funding

• National Blood Authority, National Blood Sector Research and Development Grant
• Australian & New Zealand Society of Blood Transfusion Research Fund Grant
• Monash Health Research Fund Grant
• Newcastle upon Tyne Hospitals NHS Charity Fund

Collaborating Institutions