Statistical analysis
PD, tumor, BOR, death
DISEASE PROGRESSION, DISEASE, TUMOR
The sample size for this study was not pre-specified. This phase 1 study was not statistically powered to evaluate either safety or efficacy; hence, the data were summarized descriptively. No formal hypothesis testing was planned. ORR was defined as the proportion of patients with a BOR of confirmed complete response or partial response per RECIST version 1.1 relative to the total number of patients in the corresponding analysis population. DCR was defined as the percentage of patients whose disease reduced or remained stable as per RECIST version 1.1. PFS, OS, TTR and DoR with 95% CIs were estimated using Kaplan–Meier methods. BOR per RECIST version 1.1 and ORR with 95% CIs were summarized. Censoring of data for DoR and PFS was based on US Food and Drug Administration censoring rules (If there are no adequate tumor assessments after the patient achieved confirmed response, the DoR will be censored and have a duration set to 1.If a patient is known to be alive and progression free, DoR will be censored on the day of the last adequate tumor assessment.Use of active curative anti-cancer therapy after T cell infusion (that is, before disease progression) will also be considered as meeting the PD criterion. If a patient is given subsequent curative anti-tumor therapies, curative anti-cancer therapies and curative anti-cancer surgeries other than the study treatment (not including approved palliative radiation and diagnostic procedures, such as surgical biopsies before PD or death), DoR will be censored on the date of the last progression-free tumor assessment before the start date of the anti-tumor treatment.If a patient discontinues the interventional phase of the study before PD, DoR will be censored on the date of last adequate progression-free tumor assessment.If a patient misses two or more consecutive post-baseline tumor assessments and the following assessment is a PD, or if a patient misses two or more consecutive post-baseline tumor assessments and then dies, DoR will be censored on the date of the last adequate tumor assessment.Censoring will occur as follows for PFS:If a patient has an inadequate baseline scan, PFS will be censored and have a duration set to 1.If there are no adequate post-baseline tumor assessments after T cell infusion or date of death recorded, PFS will be censored and have a duration set to 1.If a patient is known to be alive and progression free, PFS will be censored on the day of the last adequate tumor assessment.To note, use of active curative anti-cancer therapy after T cell infusion (that is, before disease progression) will also be considered as meeting the PD criterion. If a patient is given subsequent curative anti-tumor therapies, curative anti-cancer therapies and curative anti-cancer surgeries other than the study treatment (not including approved palliative radiation and diagnostic procedures, such as surgical biopsies before PD or death), DoR will be censored on the date of the last progression-free tumor assessment before the start date of the anti-tumor treatment.If a patient discontinues the interventional phase before PD, PFS will be censored on the date of the last adequate tumor assessment.If a patient misses two or more consecutive post-baseline tumor assessments and the following assessment is a PD, or if a patient misses two or more consecutive post-baseline tumor assessments and then dies, PFS will be censored on the date of the last adequate tumor assessment.Safety data were summarized using descriptive statistics. Persistence of afami-cel, RCL and cytokines were summarized descriptively. Analyses were performed with SAS software version 9.4.