Health care utility and real-world comparison of CAR T-cell therapy versus autologous stem cell transplantation in Relapsed/Refractory diffuse large B-cell lymphoma: A propensity score-matched outcomes analysis

Abstract Background: Chimeric antigen receptor (CAR) T-cell therapy has transformed the treatment landscape for relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL). However, comparative real-world data between CAR T and autologous stem cell transplantation (ASCT) remain limited. We conducted a large, multi-institutional comparative effectiveness study to evaluate outcomes associated with these two modalities. Methods: Using the TriNetX Global Collaborative Network, we identified adults (≥18 years) with DLBCL who received either CAR T-cell therapy (axicel or lisocel) or ASCT between February 2021 and August 2024. Patients were 1:1 propensity score matched based on demographics, comorbidities, treatment exposures, and laboratory parameters, resulting in 638 patients per group. The primary outcome was overall survival (OS). Secondary outcomes included hospitalization, ICU admission, cytopenia, and infection-related events. Results: Following matching, the patient demographics including age were similar between cohorts (CAR T: 61.6 years vs. ASCT: 62.0 years; p=0.598). For the primary outcome, overall survival was inferior in the CAR T cohort compared to ASCT, with a hazard ratio (HR) of 1.81 (95% CI, 1.46–2.26; p<0.001). In secondary outcomes, hospitalization was less frequent in the CAR T group, with a risk difference of –9.8% (95% CI, –18.4 to –1.2; p=0.026). ICU admission was more likely in the CAR T group, with an HR of 1.48 (95% CI, 1.11–1.98; p=0.008). Rates of cytopenia were similar between groups (risk difference 2.2%; 95% CI, –9.0 to 13.4; p=0.701). Infection-related events were significantly more frequent in the CAR T cohort, with an HR of 3.63 (95% CI, 1.87–7.06; p<0.001). Conclusions: In this real-world matched analysis, ASCT was associated with improved overall survival compared to CAR T-cell therapy in patients with R/R DLBCL, which could reflect the fact that patients eligible for ASCT were those who achieved remission status and transplant eligible. For healthcare utility perspective, CAR T therapy was linked to lower hospitalization risk, yet associated with increased ICU utilization and infectious complications compared to those of ASCT.