Authors' objectives: Malignant haematologic diseases are of great importance worldwide with increasing incidence rates. CAR-T cell therapies have been approved as a treatment option for some of these types of cancer. CAR-T cells are the patient's own T immune cells, which are genetically modified outside the body, returned to the patient and used for autologous immunotherapy. The aim of this work was to analyse the effectiveness and safety of CAR-T cell therapies based on real-world evidence and to provide an update of the current evidence.

Authors' results and conclusions: Results: A total of 26 full texts with a total of 2716 real-world patient data were identified for the search period from April 2022 to July 2024 and included in this analysis. All included studies were non-randomised, of which 14 were observational single-arm studies and 12 were indirect comparative studies with external control arms. The quality of the observational studies was rated as poor for six of them and as fair for eight. The risk of bias in the comparative studies is high and was rated as critical in all of them. Due to the heterogeneity of the studies and the characteristics of the cohorts, a narrative presentation of the results was made. The outcomes indicated that the treatment results in real-world settings are largely comparable to the results of pivotal trials. The comparative studies showed that treatment with CAR-T cells is associated with better results than treatment without CAR-T cells. Compared to previous publications, studies with longer follow-up times, further cancer types and products could be included. When comparing the outcomes with previous publications, largely similar effectiveness and safety real-world results were found. This review identified better survival rates in some cases, but also OS and PFS rates that decreased with increasing follow-up time. The response rates for the newer indications treated with CAR-T cells appeared to be higher. MCL patients treated with brexucabtagene had the highest incidence rates of relevant safety outcomes. Conclusion: Although there is an increasing body of evidence of CAR-T cell treatment in practice, the effectiveness and safety results cannot be assessed with certainty due to the identified limitations.

Authors' methods: A systematic literature search was performed based on an a priori defined PICO research question and the six CAR-T products that were approved by the EMA. The following databases were searched: Pubmed, Cochrane Library and Epistemonikos. The results were presented for the relevant cancer types using data extraction tables. A quality and risk of bias assessment was performed.

Project Status: Completed

Year Published: 2025

URL for published report: https://eprints.aihta.at/1562/1/HTA-Projektbericht_Nr.166.pdf

URL for additional information: https://eprints.aihta.at/1562/

English language abstract: An English language summary is available

Publication Type: Full HTA

Country: Austria

Organisation Name: Austrian Institute for Health Technology Assessment

Contact Address: Josefstaedter Strasse 39, A-1080 Vienna, Austria

Contact Name: office@aihta.at

Contact Email: office@aihta.at