CAR T/cell therapy Registry
Research type
Research Database
IRAS ID
336254
Contact name
Maeve O'Reilly
Contact email
Research summary
CAR T Registry for B-cell/other malignancies
REC name
East Midlands - Derby Research Ethics Committee
REC reference
24/EM/0221
Date of REC Opinion
12 Nov 2024
REC opinion
Further Information Favourable Opinion
Data collection arrangements
The database/registry will serve as a platform to establish the following:
(a) Safety outcomes: Clinical trials provide initial safety data. However, clinical trial patients are often not a representative sample of candidates for a particular disease indication due to strict eligibility criteria. Real-world registries can assess safety in a broader patient population, including older patients, those with co-morbidities and those that may have been excluded from clinical studies.
(b) Efficacy outcomes: Clinical trial patients are often carefully selected and the environment is carefully controlled. Understanding the effectiveness of CAR T therapy in standard-of-care practice, including in different patient subgroups, such as older patients, those with organ dysfunction and high-risk disease, can provide valuable insights into the true potential of this therapy. This will help clinicians and patients make informed decisions.
(c) Comparative effectiveness: As new therapeutic options enter the treatment pathway for B-cell malignancies/other malignancies, a real-world CAR T/cell therapt registry can facilitate comparative effectiveness research for CAR T therapy versus other newer agents in many different patient subgroups. This will aid informed decision-making for patients and doctors.
(d) Patient selection and adaptations to clinical care: Analysis of real-world datasets can identify efficacy and safety patterns over time. This information may refine patient selection criteria and optimise toxicity management strategies with a view to improving patient care and outcome.
Overall, collecting real-world data on CAR T-cell therapy is essential for the understanding of safety, efficacy and comparative effectiveness of this treatment modality, with a view to optimising patient selection and treatment strategies. This information can guide clinical decision-making, improve patient care, and contribute to the ongoing development of this innovative therapy.
Research programme
Chimeric antigen receptor (CAR) T-cell therapy has transformed the treatment of chemo-refractory B-cell malignancies with 40-50% of patients achieving prolonged durable remissions. However, a substantial proportion of patients will relapse and the mechanisms of CAR T failure are incompletely understood. Whilst there is robust supportive clinical trial data for the use of this treatment modality, real-world efficacy and safety outcomes are key components of informed-decision making for patients in the National Health Service (NHS). This is all the more relevant as competing therapeutic options, such as bispecific antibodies, become available in the UK. Clinical outcomes post CAR T are influenced by a variety of factors including patient selection, patient fitness, disease risk, disease burden, drop out, turnaround times, response to bridging therapy and higher-grade toxicities after infusion. Many of the aforementioned factors have emerged from detailed analysis of international real-world datasets and from experience in real-world clinical practice. A comprehensive, multi-centre registry to assess real-world CAR T outcomes will be a valuable resource and may guide future decision-making for doctors and patients in the NHS. The approval of other cellular immunotherapy approaches in the NHS in the coming years, including solid tumours (e.g tumour infiltrating lymphocytes/TCR-engineered T-cells) will warrant a similar approach to understanding the real-world outcomes of licensed cell therapy products.
Research database title
CAR T Registry for B-cell/other malignancies
Establishment organisation
University College London Hospital
Establishment organisation address
250 Euston Road
NW12PG