Trial Outcomes & Findings for Biological Therapy in Treating Patients at High-Risk or With Lymphoma, Lymphoproliferative Disease, or Malignancies (NCT NCT00002663)

Last Updated: 2023-02-13

Results Overview

The ORR is defined as percentage of participants with best overall response of complete remission/response (CR) or partial remission/response (PR) based on investigator's assessment. For participants with clinically and/or radiologically evident EBV LPD or malignancies, CR is complete resolution of all clinical and radiologic evidence of lymphoma, confirmed by biopsy of the affected tissues when indicated, lasting for at least 3 weeks following completion of a cycle of tabelecleucel; and PR is a 50 % or greater reduction in the size of all lymphomatous lesions as determined by computed tomography (CT) or magnetic resonance imaging (MRI) measurements of tumor volume, which was maintained for at least 3 weeks following completion of a cycle of tabelecleucel. For participants without clinically and/or radiologically evident tumors with increasing levels of EBV DNA, CR is clearance of EBV without subsequent development of EBV+ LPD; and PR is at least a 10-fold decrease in EBV DNA levels.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

58 participants

Primary outcome timeframe

From Day 1 through 251.1 months after Day 1 dose

Results posted on

2023-02-13

Participant Flow

A total of 58 participants were treated. However, 1 participant was analyzed in 2 different arms due to treatment. This participant was counted in only 1 arm for Participant Flow results and was counted in each arm for Outcome Measures.

Participant milestones

Participant milestones
Measure	EBV+ PTLD-HCT R/R Rituximab (Tab-cel Only) Participants with Epstein-Barr virus positive (EBV+) posttransplant lymphoproliferative disorders (PTLD) hematopoietic cell transplant (HCT) who were relapse/refractory (R/R) to rituximab received IV infusion of tabelecleucel (Tab-cel) on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-SOT R/R Rituximab + Chemo (Tab-cel Only) Participants with EBV+PTLD solid organ transplant (SOT) who were R/R to rituximab and chemotherapy received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ AID-LPD (Tab-cel Only) Participants with EBV+ acquired immunodeficiency (AID) lymphoproliferative disorder (LPD) received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ LMS (Tab-cel Only) Participants with EBV+ leiomyosarcoma (LMS) received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ NPC (Tab-cel Only) Participants with EBV+ nasopharyngeal carcinoma (NPC) received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-HCT R/R Rituximab (EBV-CTLs Only) Participants with EBV+PTLD HCT who were R/R to rituximab or rituximab naive received donor-derived EBV cytotoxic T lymphocyte (CTL) on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-SOT R/R Rituximab + Chemo (EBV-CTLs Only) Participants with EBV+PTLD SOT received donor-derived EBV-CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ Viremia (EBV-CTLs Only) Participants with EBV+ viremia received donor-derived EBV-CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+PID-LPD (Tab-cel or EBV-CTLs Only) Participants with EBV+ primary immunodeficiency (PID) LPD received IV infusion of tabelecleucel or donor-derived EBV- CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ Lymphoma (Tab-cel or EBV-CTLs Only) Participants with EBV+ lymphoma received IV infusion of tabelecleucel or donor-derived EBV- CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
Overall Study STARTED	11	4	2	5	1	23	2	6	2	2
Overall Study COMPLETED	4	2	1	3	0	8	1	4	0	0
Overall Study NOT COMPLETED	7	2	1	2	1	15	1	2	2	2

Reasons for withdrawal

Reasons for withdrawal
Measure	EBV+ PTLD-HCT R/R Rituximab (Tab-cel Only) Participants with Epstein-Barr virus positive (EBV+) posttransplant lymphoproliferative disorders (PTLD) hematopoietic cell transplant (HCT) who were relapse/refractory (R/R) to rituximab received IV infusion of tabelecleucel (Tab-cel) on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-SOT R/R Rituximab + Chemo (Tab-cel Only) Participants with EBV+PTLD solid organ transplant (SOT) who were R/R to rituximab and chemotherapy received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ AID-LPD (Tab-cel Only) Participants with EBV+ acquired immunodeficiency (AID) lymphoproliferative disorder (LPD) received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ LMS (Tab-cel Only) Participants with EBV+ leiomyosarcoma (LMS) received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ NPC (Tab-cel Only) Participants with EBV+ nasopharyngeal carcinoma (NPC) received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-HCT R/R Rituximab (EBV-CTLs Only) Participants with EBV+PTLD HCT who were R/R to rituximab or rituximab naive received donor-derived EBV cytotoxic T lymphocyte (CTL) on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-SOT R/R Rituximab + Chemo (EBV-CTLs Only) Participants with EBV+PTLD SOT received donor-derived EBV-CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ Viremia (EBV-CTLs Only) Participants with EBV+ viremia received donor-derived EBV-CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+PID-LPD (Tab-cel or EBV-CTLs Only) Participants with EBV+ primary immunodeficiency (PID) LPD received IV infusion of tabelecleucel or donor-derived EBV- CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ Lymphoma (Tab-cel or EBV-CTLs Only) Participants with EBV+ lymphoma received IV infusion of tabelecleucel or donor-derived EBV- CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
Overall Study Death	4	1	1	0	0	13	0	1	1	1
Overall Study Lost to Follow-up	3	0	0	1	0	1	0	0	0	0
Overall Study Other	0	1	0	0	0	1	1	0	0	0
Overall Study Physician Decision	0	0	0	0	1	0	0	0	1	1
Overall Study Adverse Event	0	0	0	1	0	0	0	1	0	0

Baseline Characteristics

Biological Therapy in Treating Patients at High-Risk or With Lymphoma, Lymphoproliferative Disease, or Malignancies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	EBV+ PTLD-HCT R/R Rituximab (Tab-cel Only) n=11 Participants Participants with EBV+ PTLD HCT who were R/R to rituximab received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-SOT R/R Rituximab + Chemo (Tab-cel Only) n=4 Participants Participants with EBV+PTLD SOT who were R/R to rituximab and chemotherapy received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ LMS (Tab-cel Only) n=5 Participants Participants with EBV+ LMS received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-HCT R/R Rituximab (EBV-CTLs Only) n=23 Participants Participants with EBV+PTLD HCT who were R/R to rituximab or rituximab naive received donor-derived EBV-CTL on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ Viremia (EBV-CTLs Only) n=7 Participants Participants with EBV+ viremia received donor-derived EBV-CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	Total n=50 Participants Total of all reporting groups
Age, Customized <18 years	7 Participants n=5 Participants	3 Participants n=7 Participants	4 Participants n=5 Participants	8 Participants n=4 Participants	3 Participants n=21 Participants	25 Participants n=10 Participants
Age, Customized =>18 years	4 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	15 Participants n=4 Participants	4 Participants n=21 Participants	25 Participants n=10 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	7 Participants n=4 Participants	2 Participants n=21 Participants	19 Participants n=10 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	16 Participants n=4 Participants	5 Participants n=21 Participants	31 Participants n=10 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	2 Participants n=4 Participants	1 Participants n=21 Participants	5 Participants n=10 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	4 Participants n=4 Participants	1 Participants n=21 Participants	7 Participants n=10 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	10 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	17 Participants n=4 Participants	5 Participants n=21 Participants	38 Participants n=10 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants	1 Participants n=21 Participants	5 Participants n=10 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	2 Participants n=4 Participants	1 Participants n=21 Participants	6 Participants n=10 Participants
Race (NIH/OMB) White	6 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants	18 Participants n=4 Participants	5 Participants n=21 Participants	33 Participants n=10 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	2 Participants n=4 Participants	0 Participants n=21 Participants	6 Participants n=10 Participants

PRIMARY outcome

Timeframe: From Day 1 through 251.1 months after Day 1 dose

Population: Full analysis set included all participants who received at least one dose of study treatment (tabelecleucel or EBV-CTLs). Results from arms with \<= 3 participants (as shown in the participant flow) are excluded due to the low numbers of participants; these low numbers pose a risk for participant re-identification in these rare indications of this single center study.

Outcome measures

Outcome measures
Measure	EBV+ PTLD-HCT R/R Rituximab (Tab-cel Only) n=11 Participants Participants with EBV+ PTLD following HCT who were R/R to rituximab received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-SOT R/R Rituximab + Chemo (Tab-cel Only) n=4 Participants Participants with EBV+PTLD following SOT who were R/R to rituximab and chemotherapy received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ LMS (Tab-cel Only) n=5 Participants Participants with EBV+ following LMS received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-HCT R/R Rituximab (EBV-CTLs Only) n=23 Participants Participants with EBV+PTLD following HCT who were R/R to rituximab or rituximab naive received donor-derived EBV-CTL on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ Viremia (EBV-CTLs Only) n=7 Participants Participants with EBV+ following viremia received donor-derived EBV-CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
Objective Response Rate (ORR)	63.6 Percentage of participants Interval 30.8 to 89.1	50.0 Percentage of participants Interval 6.8 to 93.2	0 Percentage of participants Interval 0.0 to 52.2	43.5 Percentage of participants Interval 23.2 to 65.5	57.1 Percentage of participants Interval 18.4 to 90.1

SECONDARY outcome

Timeframe: From Day 1 through 251.1 months after Day 1 dose

The OS is defined as the time from the first dose of tabelecleucel or EBV-CTLs to the date of death due to any cause. The OS was estimated using Kaplan-Meier method.

Outcome measures

Outcome measures
Measure	EBV+ PTLD-HCT R/R Rituximab (Tab-cel Only) n=11 Participants Participants with EBV+ PTLD following HCT who were R/R to rituximab received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-SOT R/R Rituximab + Chemo (Tab-cel Only) n=4 Participants Participants with EBV+PTLD following SOT who were R/R to rituximab and chemotherapy received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ LMS (Tab-cel Only) n=5 Participants Participants with EBV+ following LMS received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-HCT R/R Rituximab (EBV-CTLs Only) n=23 Participants Participants with EBV+PTLD following HCT who were R/R to rituximab or rituximab naive received donor-derived EBV-CTL on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ Viremia (EBV-CTLs Only) n=7 Participants Participants with EBV+ following viremia received donor-derived EBV-CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
Overall Survival (OS)	14.8 Months Interval 0.9 to Upper limit of confidence interval is not estimable due to insufficient events being observed at the time of the analysis.	84.8 Months Interval 1.1 to 115.0	NA Months Interval 60.6 to Median and upper limit of confidence interval are not estimable due to insufficient events being observed at the time of the analysis.	18.6 Months Interval 1.5 to Upper limit of confidence interval is not estimable due to insufficient events being observed at the time of the analysis.	NA Months Interval 2.5 to Median and upper limit of confidence interval are not estimable due to insufficient events being observed at the time of the analysis.

SECONDARY outcome

Timeframe: From Day 1 through 12 months after Day 1 dose

Percentage of participants with OS at 12 months are reported. The OS is defined as the time from the first dose of tabelecleucel or EBV-CTLs to the date of death due to any cause. The OS was estimated using Kaplan-Meier method.

Outcome measures

Outcome measures
Measure	EBV+ PTLD-HCT R/R Rituximab (Tab-cel Only) n=11 Participants Participants with EBV+ PTLD following HCT who were R/R to rituximab received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-SOT R/R Rituximab + Chemo (Tab-cel Only) n=4 Participants Participants with EBV+PTLD following SOT who were R/R to rituximab and chemotherapy received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ LMS (Tab-cel Only) n=5 Participants Participants with EBV+ following LMS received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-HCT R/R Rituximab (EBV-CTLs Only) n=23 Participants Participants with EBV+PTLD following HCT who were R/R to rituximab or rituximab naive received donor-derived EBV-CTL on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ Viremia (EBV-CTLs Only) n=7 Participants Participants with EBV+ following viremia received donor-derived EBV-CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
OS Rate at 12 Months	54.5 Percentage of participants Interval 22.9 to 78.0	75.0 Percentage of participants Interval 12.8 to 96.1	100.0 Percentage of participants Interval 100.0 to 100.0	52.2 Percentage of participants Interval 30.5 to 70.0	85.7 Percentage of participants Interval 33.4 to 97.9

SECONDARY outcome

Timeframe: From Day 1 through 251.1 months after Day 1 dose

The OS at follow-up time are reported. The OS is defined as the time from the first dose of tabelecleucel or EBV-CTLs to the date of death due to any cause. The OS was estimated using Kaplan-Meier method.

Outcome measures

Outcome measures
Measure	EBV+ PTLD-HCT R/R Rituximab (Tab-cel Only) n=11 Participants Participants with EBV+ PTLD following HCT who were R/R to rituximab received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-SOT R/R Rituximab + Chemo (Tab-cel Only) n=4 Participants Participants with EBV+PTLD following SOT who were R/R to rituximab and chemotherapy received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ LMS (Tab-cel Only) n=5 Participants Participants with EBV+ following LMS received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-HCT R/R Rituximab (EBV-CTLs Only) n=23 Participants Participants with EBV+PTLD following HCT who were R/R to rituximab or rituximab naive received donor-derived EBV-CTL on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ Viremia (EBV-CTLs Only) n=7 Participants Participants with EBV+ following viremia received donor-derived EBV-CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
OS Follow-up Time	14.82 Months Interval 0.5 to 88.9	63.56 Months Interval 1.1 to 115.0	77.40 Months Interval 29.5 to 109.3	18.63 Months Interval 0.2 to 251.1	62.98 Months Interval 2.5 to 91.3

SECONDARY outcome

Timeframe: From Day 1 through 251.1 months after Day 1 dose

Population: Full analysis set included all participants who received at least one dose of study treatment (tabelecleucel or EBV-CTLs). Participants who achieved CR or PR were analyzed for this outcome measure. Results from arms with \<= 3 participants (as shown in the participant flow) are excluded due to the low numbers of participants; these low numbers pose a risk for participant re-identification in these rare indications of this single center study.

The TTR was defined as the time from the date of the first dose of tabelecleucel or EBV-CTLs to the date of a PR or CR, whichever occurred first. For participants with clinically and/or radiologically evident EBV LPD or malignancies, CR was defined as complete resolution of all clinical and radiologic evidence of lymphoma, confirmed by biopsy of the affected tissues when indicated, lasting for at least 3 weeks following completion of a cycle of tabelecleucel or EBV-CTLs; and a PR was defined as a 50 % or greater reduction in the size of all lymphomatous lesions as determined by CT or MRI scan measurements of tumor volume, which was maintained for at least 3 weeks following completion of a cycle of tabelecleucel or EBV-CTLs. For participants without clinically and/or radiologically evident tumors with increasing levels of EBV DNA, CR was defined as clearance of EBV without subsequent development of EBV+ LPD; and PR was defined as at least a 10-fold decrease in EBV DNA levels.

Outcome measures

Outcome measures
Measure	EBV+ PTLD-HCT R/R Rituximab (Tab-cel Only) n=7 Participants Participants with EBV+ PTLD following HCT who were R/R to rituximab received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-SOT R/R Rituximab + Chemo (Tab-cel Only) n=2 Participants Participants with EBV+PTLD following SOT who were R/R to rituximab and chemotherapy received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ LMS (Tab-cel Only) Participants with EBV+ following LMS received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-HCT R/R Rituximab (EBV-CTLs Only) n=10 Participants Participants with EBV+PTLD following HCT who were R/R to rituximab or rituximab naive received donor-derived EBV-CTL on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ Viremia (EBV-CTLs Only) n=4 Participants Participants with EBV+ following viremia received donor-derived EBV-CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
Time to Response (TTR)	1.18 Months Interval 0.8 to 2.4	6.74 Months Interval 3.1 to 10.4	—	1.45 Months Interval 0.6 to 5.1	1.22 Months Interval 1.1 to 1.4

SECONDARY outcome

Timeframe: From Day 1 through 251.1 months after Day 1 dose

Population: Full analysis set included all participants who received at least one dose of study treatment (tabelecleucel or EBV-CTLs). Participants who achieved CR, PR or SD were analyzed for this outcome measure. Results from arms with \<= 3 participants (as shown in the participant flow) are excluded due to the low numbers of participants; these low numbers pose a risk for participant re-identification in these rare indications of this single center study.

The CBR was the proportion of participants who have achieved a CR, PR or SD assessed at least 28 days after first dose date of study drug. For participants with clinically and/or radiologically evident EBV LPD or malignancies, CR as complete resolution of all clinical and radiologic evidence of lymphoma, confirmed by biopsy of affected tissues when indicated, lasting for at least 3 weeks following completion of a cycle of tabelecleucel or EBVCTLs; and a PR as a \>= 50% reduction in size of all lymphomatous lesions as determined by CT or MRI scan measurements of tumor volume, maintained for at least 3 weeks following completion of a cycle of tabelecleucel or EBV-CTLs. For participants without clinically and/or radiologically evident tumors with increasing levels of EBV DNA, CR as clearance of EBV without subsequent development of EBV+ LPD; and PR as at least a 10-fold decrease in EBV DNA levels. The CBR was included specifically as clinically meaningful for solid tumor, namely LMS.

Outcome measures

Outcome measures
Measure	EBV+ PTLD-HCT R/R Rituximab (Tab-cel Only) n=11 Participants Participants with EBV+ PTLD following HCT who were R/R to rituximab received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-SOT R/R Rituximab + Chemo (Tab-cel Only) n=4 Participants Participants with EBV+PTLD following SOT who were R/R to rituximab and chemotherapy received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ LMS (Tab-cel Only) n=5 Participants Participants with EBV+ following LMS received IV infusion of tabelecleucel on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ PTLD-HCT R/R Rituximab (EBV-CTLs Only) n=23 Participants Participants with EBV+PTLD following HCT who were R/R to rituximab or rituximab naive received donor-derived EBV-CTL on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.	EBV+ Viremia (EBV-CTLs Only) n=7 Participants Participants with EBV+ following viremia received donor-derived EBV-CTLs on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
Clinical Benefit Rate (CBR)	63.6 Percentage of participants Interval 30.8 to 89.1	75.0 Percentage of participants Interval 19.4 to 99.4	80.0 Percentage of participants Interval 28.4 to 99.5	47.8 Percentage of participants Interval 26.8 to 69.4	71.4 Percentage of participants Interval 29.0 to 96.3

Adverse Events

Overall Total

Serious events: 40 serious events

Other events: 0 other events

Deaths: 32 deaths

Serious adverse events

Serious adverse events
Measure	Overall Total n=58 participants at risk All eligible participants with EBV+ received IV infusion of tabelecleucel or donor-derived EBV-CTL on Days 1, 8, and 15 and were observed for 3 weeks. After the observation period, additional courses (2 courses) may have been provided in the absence of disease progression or unacceptable toxicity.
Blood and lymphatic system disorders Febrile neutropenia	5.2% 3/58 • Number of events 4 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Blood and lymphatic system disorders Haemolysis	1.7% 1/58 • Number of events 2 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Cardiac disorders Left ventricular dysfunction	3.4% 2/58 • Number of events 2 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Cardiac disorders Pericardial effusion	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Cardiac disorders Pericarditis	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Cardiac disorders Sinus bradycardia	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Cardiac disorders Ventricular arrhythmia	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Endocrine disorders Hypopituitarism	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Gastrointestinal disorders Abdominal pain	5.2% 3/58 • Number of events 3 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Gastrointestinal disorders Anal ulcer	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Gastrointestinal disorders Ascites	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Gastrointestinal disorders Colitis	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Gastrointestinal disorders Diarrhoea haemorrhagic	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Gastrointestinal disorders Haematochezia	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Gastrointestinal disorders Nausea	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Gastrointestinal disorders Odynophagia	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Gastrointestinal disorders Stomatitis	3.4% 2/58 • Number of events 2 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
General disorders Device related thrombosis	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
General disorders Disease progression	5.2% 3/58 • Number of events 4 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
General disorders Oedema	3.4% 2/58 • Number of events 2 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
General disorders Pyrexia	22.4% 13/58 • Number of events 14 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Hepatobiliary disorders Cholecystitis	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Hepatobiliary disorders Hepatic failure	3.4% 2/58 • Number of events 2 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Immune system disorders Hypersensitivity	3.4% 2/58 • Number of events 2 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Immune system disorders Renal transplant failure	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Immune system disorders Serum sickness	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Device related infection	13.8% 8/58 • Number of events 9 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Ear infection bacterial	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Ear infection staphylococcal	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Escherichia urinary tract infection	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Gastroenteritis rotavirus	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Gastrointestinal viral infection	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Infection	6.9% 4/58 • Number of events 5 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Neutropenic infection	5.2% 3/58 • Number of events 4 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Otitis media	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Paronychia	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Pharyngitis	3.4% 2/58 • Number of events 2 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Pneumonia	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Pneumonia fungal	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Sinusitis bacterial	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Staphylococcal infection	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Infections and infestations Viral upper respiratory tract infection	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Investigations Blood bilirubin increased	3.4% 2/58 • Number of events 2 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Investigations Blood creatine phosphokinase increased	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Investigations Blood creatinine increased	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Investigations Electrocardiogram QT prolonged	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Investigations Neutrophil count decreased	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Metabolism and nutrition disorders Decreased appetite	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Metabolism and nutrition disorders Dehydration	5.2% 3/58 • Number of events 4 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Metabolism and nutrition disorders Hyperglycaemia	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Kaposi's sarcoma	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Non-Hodgkin's lymphoma recurrent	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Thyroid cancer	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Nervous system disorders Cerebral haemorrhage	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Nervous system disorders Depressed level of consciousness	1.7% 1/58 • Number of events 3 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Nervous system disorders Intracranial pressure increased	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Nervous system disorders Nervous system disorder	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Nervous system disorders Neuralgia	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Nervous system disorders Peripheral motor neuropathy	3.4% 2/58 • Number of events 2 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Nervous system disorders Seizure	5.2% 3/58 • Number of events 3 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Psychiatric disorders Mental status changes	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Renal and urinary disorders Renal failure	5.2% 3/58 • Number of events 3 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Renal and urinary disorders Urinary retention	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Respiratory, thoracic and mediastinal disorders Acute respiratory distress syndrome	5.2% 3/58 • Number of events 3 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Respiratory, thoracic and mediastinal disorders Hypoxia	8.6% 5/58 • Number of events 7 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Respiratory, thoracic and mediastinal disorders Obstructive airways disorder	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Respiratory, thoracic and mediastinal disorders Organising pneumonia	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Respiratory, thoracic and mediastinal disorders Pleural effusion	3.4% 2/58 • Number of events 2 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Respiratory, thoracic and mediastinal disorders Pneumonia aspiration	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Respiratory, thoracic and mediastinal disorders Pneumonitis	5.2% 3/58 • Number of events 5 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Respiratory, thoracic and mediastinal disorders Pneumothorax	1.7% 1/58 • Number of events 2 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Respiratory, thoracic and mediastinal disorders Pulmonary haemorrhage	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Vascular disorders Embolism	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Vascular disorders Haemorrhage	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Vascular disorders Hypertension	1.7% 1/58 • Number of events 1 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Vascular disorders Hypotension	3.4% 2/58 • Number of events 2 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.
Vascular disorders Venoocclusive disease	1.7% 1/58 • Number of events 2 • From Day 1 through 251.1 months after Day 1 dose Combined AE data for all enrolled participants are reported because few arms had \<=3 participants which poses a risk of participant identification with rare indications in this study. Treatment-emergent SAEs includes events collected retrospectively and prospectively per protocol. Non-serious AEs were not collected, hence reported as '0'. In participant flow, 'Deaths' are reported as a reason for study discontinuation, whereas in 'All-Cause Mortality' deaths due to any cause are reported.

Other adverse events

Adverse event data not reported

Additional Information

Study Director

Atara Biotherapeutics

Phone: 650-278-8930

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place