Statistical analysis
DISEASE
This is a subgroup analysis of all patients enrolled in the ELARA trial from institutions located in Japan, with a data cutoff date (March 29, 2021) established after 90 infused patients in the overall study population were followed for at least 12 months from infusion or discontinued earlier. The efficacy analysis set (EAS) included all patients who received tisagenlecleucel infusion and had measurable disease pre-infusion per IRC review, the safety set included all infused patients, and the cellular kinetic analysis set included all patients in the EAS who provided at least one cellular kinetic parameter. All data analyses were descriptive owing to the small size of the Japanese subgroup. The CRR and ORR were reported with the 2-sided 95% exact Clopper-Pearson confidence interval (CI). The DOR, PFS, and OS were estimated using the Kaplan–Meier method, and their individual data were also presented along with the best overall response. The frequency and incidence proportion of AEs were reported, and cellular kinetics parameters were summarized using appropriate descriptive statistics. All data analyses were performed, and outputs were generated using SAS version 9.4. A detailed description of the overall study design and statistical analysis for the ELARA trial has been published previously [