Real-world outcomes of tisagenlecleucel treatment in patients with relapsed or refractory large B-cell lymphomas: a nationwide registry.

[BACKGROUND] Chimeric antigen receptor (CAR) T-cell therapy has transformed treatment for patients with relapsed or refractory large B-cell lymphoma (LBCL). However, real-world data in Japan remain limited.

[METHODS] We retrospectively analyzed 489 LBCL patients treated with tisagenlecleucel (tisa-cel) using a nationwide registry.

[RESULTS] The median patient age was 62 years, with 89% having undergone ≥3 prior therapies. At infusion, 22% were in a complete response. The estimated 1-year progression-free survival (PFS) and overall survival (OS) rates were 43.2% (95%CI, 37.8-49.3%) and 65.9% (95% CI, 61.0-71.1%), respectively. Multivariable analysis revealed that better Karnofsky performance status, a history of stem cell transplantation, absence of extranodal lesion, higher absolute lymphocyte count, and lower lactate dehydrogenase level prior to lymphodepleting therapy were significantly associated with better PFS and OS. Older age (≥65 years) at infusion was associated with better PFS, while shorter duration from apheresis to infusion (<60 days) was associated with better OS. Cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome occurred in 71% and 5% of patients, respectively.

[CONCLUSIONS] This study provides the largest real-world dataset on tisa-cel treatment for LBCL. Our results showed real-world outcomes and early safety consistent with prior trials.