Terapie CAR-T: focus sull’impiego di axicabtagene ciloleucel in seguito all’ampliamento delle indicazioni terapeutiche

CAR-T therapies: focus on the use of axicabtagene ciloleucel following the expansion of therapeutic indications. Summary. Introduction. CAR-T cell therapies represent a promising treatment option for relapsed or refractory diffuse large B-cell lymphoma (DLBCL R/R), offering novel therapeutic opportunities in both advanced and earlier stages of the disease. Among the currently approved CAR-T therapies, axicabtagene ciloleucel (axi-cel) has demonstrated significant efficacy and safety, as shown in the ZUMA-1 and ZUMA-7 trials. These studies led to the approval of expanded therapeutic indications by key regulatory authorities. This article aims to assess the impact of the expanded indications of axi-cel in clinical practice, focusing on the treatments administered, the associated costs, and the adverse events reported. Materials and methods. Data from treatments administered between January 1, 2022, and September 30, 2024 were extracted from the informatic pharmacy management system and the prescription records from the AIFA monitoring registry. Data were stratified by patient characteristics, therapeutic indication, year of infusion, and adverse events using Excel. An analysis of the treatment-related costs was also performed, considering the application of negotiated agreements. Results. A total of 32 patients diagnosed with relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL) were treated. In 2022 and 2023, 16 patients received treatment following at least two lines of systemic therapy. During the first three quarters of 2024, 16 treatments were administered, half of which occurred in the second-line setting. Adverse events were observed in 78% of treated patients: cytokine release syndrome (CRS) in 41%, neurological toxicity in 3%, combined CRS and neurotoxicity in 16%, and neutropenia in 9%. Three deaths were reported, all attributed to disease progression. The increase in treatments corresponded to a rising trend in associated healthcare expenditure, which grew from €2,256,579.07 in 2022 to €2,449,295.19 in 2023, reaching €3,352,359.21 by September 30, 2024. Conclusions. The use of axi-cel in DLBCL R/R treatment is increasing, driven by expanded indications to earlier stages of the disease. This growth carries significant economic implications and underscores the importance of ongoing collaboration and continuous professional development to optimize the management of these highly innovative, personalized therapies.