Cancer Medicines Outcomes Programme Public Health Scotland (CMOP-PHS) report
CAR-T for haematological malignancies 2020 to 2023

About this release

This release by Public Health Scotland (PHS) is a Cancer Medicines Outcomes Programme-Public Health Scotland collaboration and uses information from the National SACT dataset.

The aim of this work was to describe how chimeric antibody receptor therapies (CAR-T) are used for patients with haematological malignancies in Scotland, and the outcomes of using these treatments, between 1st January 2020 to 31st December 2023, with a follow up period until 30th June 2024. Patients were identified using the National Systemic Anti-Cancer Therapy (SACT) dataset linked to other healthcare datasets held in Public Health Scotland (PHS).

The objectives were to:

• Summarise baseline characteristics for patients subject to treatment with CAR-T.
• Describe treatment pathways before and after CAR-T (including time to next SACT).
• Estimate overall survival and report 12 and 24-month survival rates (as appropriate) among patients treated with CAR-T.
• Determine if there is an association between patient or treatment-related factors and overall survival.
• Explore data capability to report the unintended outcomes of CAR-T (e.g., emergency hospital admissions and prescriptions for intravenous antibiotics / antifungals).

Main points

• The report shows how national Systemic Anti-Cancer Therapy (SACT) data can be used to report the outcomes of chimeric antibody receptor treatments (CAR-T) in cancer patients in Scotland.
• There has been an increase in the number of patients receiving CAR-T per year between 2020 and 2023, from 15 to 26 patients annually.
• The most common CAR-T treatment prescribed was axicabtagene ciloleucel with 44 (58%) patients receiving this treatment.
• CAR-T was used most often in patients with diffuse large B-cell lymphoma (DLBCL); 67(88%) patients.
• Overall survival (OS) for the 76 patients who received CAR-T was estimated to be: 16.6 months (95% Confidence Intervals (CI) 10.8 - not reached); 60.1% (95% CI 49.7% – 72.6%) at 12 months; and 43.0% (95% CI 32.1% – 57.6%) at 24 months.
• Fifteen (19.7%) patients were admitted to either an Intensive Care Unit (ICU) or a High Dependency Unit (HDU) within 30 days of CAR-T infusion.
• Sixty-nine (90.8%) patients were identified who received two or more lines of SACT in Scotland prior to receiving CAR-T.
• There were 20 (26.3%) patients who received further SACT after CAR-T infusion for a haematological malignancy. The median time from the date of CAR-T infusion to the first subsequent SACT was 4.4 months (IQR 3.6 – 6.1).

Background

CMOP-PHS Background

The overall vision of the Cancer Medicines Outcomes Programme-Public Health Scotland (CMOP-PHS) collaboration is to better understand the real-life impact of cancer medicines on cancer patients in Scotland. Healthcare professionals may then use these findings to help support clinical decision making and enable a more bespoke and individualised approach to the provision of cancer care for all our patients. Developing, and refining, a robust and reliable process means that cancer medicines intelligence may be routinely generated to support informed decision making at individual, local, and national levels.

Together with information available from clinical trials, this additional knowledge is intended to help NHS Scotland deliver a more personalised approach to providing cancer care, and to help ensure the safe and effective use of medicines.

The CMOP-PHS collaboration builds on established clinical engagement across NHS Scotland Health Boards and integrates with key stakeholders, thereby bringing together clinical, academic, and data analysis skills from the NHS, PHS, and academia, alongside the patient and public voice. The collaboration provides organisational memory and supports workforce resilience, embedding robust processes to routinely generate and report real-world evidence (RWE). This supports the provision of patient centred cancer care and decision-making processes, setting Scotland apart as a centre of excellence in the cancer medicines RWE arena. This supports the provision of patient centred cancer care and decision-making processes, setting Scotland apart as a centre of excellence in the cancer medicines RWE arena.

CAR-T

Chimeric antigen receptor T cell therapy (CAR-T) is a new type of immunotherapy that is genetically engineered from the body's own T cells. The treatment targets T cells to a specific antigen that is present on tumour cells to generate an antitumour response.

Several CAR-T therapies have now been accepted for use in NHS Scotland by the Scottish Medicines Consortium (SMC) in the treatment of patients with relapsed and / or refractory diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma (PMBCL), mantle cell lymphoma (MCL), and B-cell acute lymphoblastic leukaemia. These therapies are: axicabtagene ciloleucel (Yescarta®); brexucabtagene autoleucel (Tecartus®); and tisagenlecleucel (Kymriah®).