Self-reported outcome measures could improve quality of life for CAR T-Cell therapy patients

New research funded by the National Institute for Health and Care Research (NIHR) has identified a set of patient-reported outcome (PRO) measures – which are self-reporting measures of how a patient feels and functions – specifically to be used in electronic PRO (ePRO) systems for patients undergoing Chimeric Antigen Receptor (CAR) T-cell therapy.

The study, published in The Lancet eClinical Medicine, is the first of its kind to use a consensus method to select PRO tools for CAR T-cell therapy, laying the groundwork for digitally monitoring patients in real time to help improve the clinical management experience of their treatment.

Chimeric Antigen Receptor (CAR) T-cell therapies are effective for treating haematological cancers but can carry risks of toxicity as well as a wide range of side effects and symptoms, often requiring extended clinical monitoring and hospital stays for patients.

As a result, patient-reported outcomes (PROs), have been identified as potentially valuable tools for the clinical management of patients who are recipients of CAR T-cell therapies due to its non-invasive and accessible nature.

Researchers from the Centre for Patient Reported Outcome Research, NIHR Blood and Transplant Research Unit in Precision Cellular Therapeutics and the NIHR Birmingham Biomedical Research Centre conducted a rapid review and Delphi consensus study with patients and healthcare professionals to identify and select PRO measures to include a digital system for use in clinical practice.

The study employed a two-round modified Delphi method which involved 19 participants, including CAR T-cell patients and healthcare professionals, from a UK NHS cellular therapy centre and professional networks. The goal was to reach consensus on the most relevant, comprehensive, and understandable PRO measures for use in clinical settings.

Consensus was achieved after the first round, leading to the selection of the following PRO measures for the new system:

• Symptom Burden Questionnaire™ (SBQ™): to assess treatment-related symptoms
• Quality of Life in Adult Cancer Survivors (QLACS): to evaluate broader impacts of cancer and CAR T-cell therapy
• EQ-5D-5L: to measure health-related quality of life
• FACT-GP5: a single-item global indicator of treatment tolerability

These tools will form the foundation of the new PRO-CAR-T ePRO system, designed to support real-time, remote monitoring of patients throughout their treatment journey.

“Implementing electronic PRO systems could support remote monitoring the treatment of patients for acute, late, and long-term effects of CAR T-cell therapy with the benefit of enhancing both clinical care and vitally, enhancing the patient experience.”

Dr Sarah Hughes, University of Birmingham

Dr Sarah Hughes, the lead author of the study, commented:

“Implementing electronic PRO systems could support remote monitoring the treatment of patients for acute, late, and long-term effects of CAR T-cell therapy with the benefit of enhancing both clinical care and vitally, enhancing the patient experience.

“This is a key step towards establishing patient-centred, accessible care for those receiving CAR T-cell therapy. We hope that by integrating a more holistic approach into routine monitoring, we can improve outcomes and quality of life for all.”

If successfully implemented across clinical practice, electronic PRO systems help doctors to respond more quickly to problems – therefore, improving the quality of care patients receive – whilst also helping to make CAR T-cell therapy more accessible and cost-effective.

However, as the field of CAR T-cell therapies evolves and other PRO measures are developed and validated for use with patients, further research and studies will be necessary to ensure the best available measures are continuously selected.

Dr Hughes added:

“Whilst the number of studies of CAR T-cell therapies reporting PROs is growing, the number of studies describing the implementation of PROs in the context of routine clinical care of CAR-T patients remains extremely limited.

“Our findings highlight not only the importance of stakeholder-driven research amongst both patients and healthcare professionals, but also the ongoing need for further investigation within this rapidly evolving field.”