Axicabtagene Ciloleucel CAR-T 疗法治疗大 B 细胞淋巴瘤的真实世界结局：一项单中心观察性研究

Axicabtagene Ciloleucel (Axi-cel) has recently transformed the treatment landscape for relapsed or refractory large B-cell lymphoma (R/R LBCL). Despite promising results in pivotal trials like ZUMA-1 and ZUMA-7, there is limited data on its outcomes in the Middle East and North Africa (MENA) region.

This observational study analyzed patients with R/R LBCL treated with Axi-cel at King Faisal Specialist Hospital and Research Center (KFSH and RC), Riyadh, Saudi Arabia, from February 2023 to November 2024. The primary endpoints were overall survival (OS) and progression-free survival. Secondary outcomes included response rates and toxicity.

A total of 77 patients were included. The median age was 53 years. Most patients presented with advanced-stage disease (75% Stage IV) and bone marrow involvement was observed in 34%. The best overall response rate was 78%, with a complete response (CR) rate of 49%. CR rates were comparable between second-line (48%) and third-line or beyond (50%) groups. Median OS was not reached for both the second-line and third-line or beyond, favoring the second-line in OS (hazards ratio [HR] = 2.98, P = 0.03). The estimated 12-month OS was 71.4% for the entire group and 86.2% for the second-line cohort, compared to 57% for the third-line and beyond cohort. The absence of bone marrow involvement (HR = 0.28, P = 0.008) and elevated lactate dehydrogenase (LDH) levels (HR = 2.69, P = 0.041) were significant risk factors on multivariate analysis for OS. The incidence of cytokine release syndrome was 94% (Grade 3 in 4%) and immune effector cell-associated neurotoxicity syndrome in 39% of patients (Grade 3 in 17%).

Real-world data from the MENA region on the safety and efficacy of Axi-cel in patients with R/R LBCL were comparable to those from the registrational ZUMA-1 and ZUMA-7 trials. Bone marrow involvement, elevated LDH and advanced disease emerged as adverse prognostic markers.