美国 axicabtagene ciloleucel 治疗大 B 细胞淋巴瘤的真实世界证据

Axicabtagene ciloleucel (axi-cel) is a standard-of-care for patients with relapsed or refractory (r/r) large B cell lymphoma who have received 2 or more lines of prior therapy. Patients receiving axi-cel in the real world could have broader a demographic, disease, and treatment profile compared with that of the cohort in the pivotal ZUMA-1 trial. The present study was conducted to evaluate the outcomes of axi-cel therapy in the real-world setting. A total of 1297 patients receiving commercial axi-cel between 2017 and 2020 were selected from the Center for International Blood and Marrow Transplant Research's data registry, of whom 739 (57%) would have been ineligible for inclusion in the ZUMA-1 cohort. Efficacy and safety outcomes were described for the entire cohort and by ZUMA-1 eligibility. Their associations with age, Eastern Cooperative Oncology Group Performance Score, and comorbidities were evaluated using multivariable logistic and Cox regressions. At a median follow-up of 12. 9 months, the overall response rate (ORR) was 73%, with a 56% complete response (CR) rate. Median overall survival (OS) and progression-free survival (PFS) were 21.

8 months (95% confidence interval [CI], 17. 4 to 28. 8 months) and 8. 6 months (95% CI, 6. 5 to 12. 1 months), respectively. Duration of response (DOR) was comparable in the ZUMA-1 ineligible patients and ZUMA-1 eligible patients (62% by 1 year [95% CI, 57% to 66%] versus 67% [95% CI, 62% to 72%]). Patients age 65 years had favorable ORR (odds ratio [OR], 1. 39; 95% CI, 1. 05 to 1. 83) despite having a higher risk of cytokine release syndrome (CRS) (OR, 1. 41; 95% CI, 1. 02 to 1. 94) and immune effector cell-associated neurotoxicity syndrome (ICANS) (OR, 1. 77; 95% CI, 1. 39-2. 26).

Eastern Cooperative Oncology Group Performance Score 2 was associated with inferior efficacy outcomes (OR for ORR, 0. 32; 95% CI, 0. 18-0. 56; hazard ratio [HR] for OS, 3. 27; 95% CI, 2. 37 to 4. 52) and higher incidence of ICANS (OR, 2. 63; 95% CI, 1. 40 to 4. 93).

The patients ineligible for ZUMA-1 still had a durable response with axi-cel. Elderly patients had favorable efficacy outcomes despite higher rates of CRS and ICANS. Patient selection for standard-of-care axi-cel should consider comorbidities and risk-to-benefit ratio rather than be based strictly on ZUMA-1 eligibility.