BLISS - Blood cancer clinical trials Long-term follow-up using Integrated healthcare SystemS data

The BLISS project aims to establish a research database by linking routinely collected healthcare systems data (HSD) such as NHS England (NHSE) data with clinical trial data from approximately 5,500 participants enrolled in completed multiple myeloma trials conducted by the University of Leeds CTRU. The primary objective is to evaluate the feasibility and validity of using NHSE data to support long-term follow-up and endpoint assessment in clinical trials.

Specific questions the data processing aims to answer include:

• How accurate and complete is HSD in capturing key clinical trial endpoints such as mortality, cancer registration, treatment, and hospital admissions?

• Can HSD be used to reliably extend follow-up for trial participants, and how does it compare to data collected within the trials?

• To what extent do trial participants reflect the diversity and characteristics of the broader patient population, and how do these factors influence outcomes?

• Is time to next line of treatment a valid surrogate for progression-free survival when measured using HSD?

• How is fatality assessed and how predictive are frailty indices derived from HSD for clinical outcomes, and can they inform future trial design?

• How is bone disease treated and what are the treatment patterns and outcomes for myeloma bone disease as recorded in HSD?

The project will also generate evidence to support the use of HSD in future prospective trials, offering a cost-effective and scalable approach to trial follow-up that enhances research capacity and accelerates evidence generation in blood cancer research.

The University of Leeds will transfer data to NHS England. The data will consist of identifying details (specifically NHS Number, Date of Birth, Sex, date of recruitment and a unique BLISS person ID) for the cohort to be linked with NHS England data.

NHS England will provide the relevant records from the Hospital Episode Statistics (HES) Accident and Emergency (A&E), Admitted Patient Care (APC) and Outpatients (OP) datasets, the National Disease Registration Service (NDRS) Systemic Anti-Cancer Therapy (SACT) dataset, NDRS National Radiotherapy Dataset (RTDS), NDRS Cancer Registration dataset, the Emergency Care Dataset (ECDS) and the Civil Registration of Deaths datasets to the University of Leeds. The Data will contain no direct identifying data items but will contain a unique person ID which can be used to link the Data with other record level data already held by the recipient.

The Data will not be transferred to any other location.

The Data will be stored on servers at the University of Leeds Clinical Trials Research Unit (CTRU). The data is backed up on the Microsoft Azure platform. The Data will be accessed onsite at the premises of the University of Leeds from the Leeds CTRU secure storage area.

The Data will be linked at person record level with existing trial data including the following trials: Myeloma X, Myeloma XI, MUK One, MUK Three, MUK Four, MUK Five, MUK Six, MUK Seven, MUK Eight, MUK Twelve.

These trials are closed to further follow-up and new data collection will only be via linked NHSE data.

The outputs expected to be produced as a result of the data processing include:

Clinical outputs:

• Peer‑reviewed journal articles reporting clinical findings (approx. 1–2 per year)

• Clinical trial findings contributing to evidence base (implicit in publications)

Methodological outputs:

• A curated database for future health research (restricted to University of Leeds study team during project)

• Methodological publications in peer‑reviewed journals

• Public release of code and code lists on GitHub (open access)

• Reports to the funder (Blood Cancer UK)

The outputs will not contain NHS England Data and will only contain aggregated information with small numbers suppressed as appropriate in line with the relevant disclosure rules for the dataset(s) from which the information was derived.

• The outputs will be communicated to the relevant recipients via:

• Presentations at haematological and clinical trial conferences (e.g., EHA, ASH)

• Dissemination of results to clinicians, patients, and stakeholders via webinars, lay summaries, and the BLISS website

• End‑of‑project dissemination event and stakeholder meetings

• Presentations to the wider BLISS team (quarterly PMG, annual Steering Group)

• Presentations to the PPI Advisory Group (biannual meetings)

• Conference presentations at methodological meetings (e.g., ICTMC, CRUK Data‑driven Cancer Research Conference)

• Presentations at local, regional, and national wider stakeholder meetings.

Clinical Benefits:

The project will provide evidence that supports patients and clinical teams by focusing on:

• Long-term effects of current treatments - mortality data from completed trials will be analysed to assess overall survival and treatment effectiveness. These findings will help determine whether progress toward curing myeloma is being made.

• Outcomes for patients with frailty and bone disease - frailty and bone disease have a major impact on quality of life. Research will evaluate different frailty measures to identify the most suitable scoring system for use in future trials, ensuring treatment strategies can be tailored more effectively. Work on bone disease will examine bisphosphonate schedules and palliative radiotherapy use alongside modern therapies, generating evidence to guide future interventions that reflect both clinical and patient priorities.

• Diversity and disparities in trial populations - analysis of trial participant characteristics will show whether populations are representative of the wider myeloma community. Any gaps will highlight barriers to inclusive research and inform strategies to improve recruitment. Outcomes will also be assessed across different patient groups to support more personalised care.

By addressing these areas, the project will inform future trials, expand knowledge, and contribute to better patient care.

Methodological Benefits:

The project will strengthen the case for using healthcare systems data (HSD) in clinical trial follow-up, demonstrating its reliability as real-world evidence. This supports national priorities to deliver faster and more efficient trials.

By showing that HSD can reduce or replace traditional trial data collection, blood cancer trials can become more efficient, cost-effective, and patient-centred. The University of Leeds Clinical Trials Unit will integrate HSD into future trial designs and share best practices across the UKCRC Registered Clinical Trials Unit network.

Collaboration with industry partners will highlight the UK’s strengths in data-enabled research, encouraging companies to bring innovative blood cancer treatments to UK trials

Yielded Benefits is not a requirement for new applications.