嵌合抗原受体（CAR）T 细胞治疗相关的成本、效果与安全性：来自一家综合癌症中心的结果

Real world effectiveness, toxicity and costs analyses from chimeric antigen receptor (CAR)-T cell therapy are of utmost relevance to determine whether and how to offer patients highly personalized immunotherapy. In this study, we aimed at describing CAR T-cells effectiveness, safety and costs in a Portuguese Comprehensive Cancer Center.

We performed a retrospective descriptive study of adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma and transformed follicular lymphoma referred to CAR T-cell therapy, between May 2019 and February 2021. Rates of treatment response, toxicity and survival (Kaplan-Meier method) were analyzed by intention-to-treat. Direct medical costs stratified by inpatient-care, outpatient-care, and diagnostic-therapeutic procedures (DTP) were derived based on resources used and their respective unit costs. In twenty patients (median age 49. 5y; 55%male; 70%DLBCL; 50% with primary refractory disease), best overall and complete response rates were 65.

0% and 45. 0%, respectively. Median overall (OS) and progression-free survivals were 9. 2 and 7. 3 months; 12-month OS rate was 42. 6% (95%CI:23. 2-78. 3). Grade 3 cytokine release syndrome and neurotoxicity occurred in 5. 6% and 11. 1% of patients, respectively.

CAR T-cell therapy expenditure, including adverse events costs, was 7 176 196 , or 286 238 when excluding drug cost. Median cost for treated patient was 355 165 with CAR T-cell drug cost accounting for 97. 0% of the overall expense. Excluding CAR T-cell acquisition cost, inpatient-care and DTP accounted for 57% and 38% of total cost/patient, respectively.

Our findings highlight the heavy economic burden of CAR T-cell therapy driven by drug acquisition costs.