Statistical analysis
DISEASE, EVENT
A one-sample binomial design was used to calculate the initial sample size. We hypothesized that axi-cel would yield a CMR at 3 months of 34% compared to 12% with a historical SOC estimated from a retrospective, real-world cohortEfficacy and safety analyses were performed on the mFAS, which included all patients who signed an informed consent and were infused with axi-cel. A sensitivity analysis of the primary end point was also conducted on all patients who signed an informed consent and had leukapheresis (FAS). We calculated CMR with exact Clopper–Pearson CI values, without adjustment for multiplicity. Patients without response assessment, due to any reason, were considered as nonresponders. We used the Kaplan–Meier method to estimate medians and 95% CI values for PFS, EFS, OS and DOR. If patients did not have an event at the time of the PFS, EFS and DOR analysis, they were censored at the date of the last disease assessment. For assessment of OS, alive patients were censored at their last follow-up date. Safety end points were assessed using descriptive statistics (counts and percentages). A subgroup analysis of CMR at 3 months was conducted for prespecified covariates (such as sex) and a Forest plot was provided.Collected data were entered using the Electronic Data Capture system from Ennov v.8.1 (Ennov). Sample size calculation was performed using EAST v.6.5 (Cytel). All statistical analyses were performed using SAS v.9.3 or higher (SAS Institute) and AdClin v.3.2.2 or higher (AdClin).