ORR is defined as the percentage of participants with a best overall disease response of complete remission (CR) or Complete remission with incomplete blood count recovery (CRi), where the best overall disease response is defined as the best disease response recorded from CTL019 infusion until the start of new anticancer therapy. Best response was assigned in the following order: CR, CRi, CR or CRi with residual mediastinal disease, No response and Unknown.

Efficacy Analysis Set (EAS): A subset of all infused patients who were treated with CTL019 at least 6 months prior to the clinical data cutoff. In the final analysis, it is identical to the FAS

Overall Remission Rate (ORR), which includes Complete Remission (CR) and Complete Remission with Incomplete Blood Count Recovery (CRi), as determined by assessments of peripheral blood, bone marrow, CNS symptoms, physical exam (PE) and cerebrospinal fluid (CSF). This primary endpoint was based on the local investigator assessment. No participants with lymphoblastic lymphoma were infused in this study.

Efficacy Analysis Set (EAS): A subset of all infused patients who were treated with CTL019 at least 6 months prior to the clinical data cutoff. In the final analysis, it is identical to the FAS. No patients with Lymphoblastic Lymphoma were enrolled in this trial, hence there is no data to report.

Evaluate the percentage of participants who achieved CR or CRi at Month 6 without SCT between tisagenlecleucel infusion and Month 6 response assessment.

Efficacy Analysis Set (EAS): A subset of all infused patients who were treated with CTL019 at least 6 months prior to the clinical data cutoff. In the final analysis, it is identical to the FAS.

Evaluate the percentage of subjects who achieved CR or CRi and then proceeded to SCT while in remission prior to Month 6 response assessment.

Efficacy Analysis Set (EAS): A subset of all infused patients who were treated with CTL019 at least 6 months prior to the clinical data cutoff. In the final analysis, it is identical to the FAS.

DOR is the time from achievement of CR or CRi, whichever occurs first, to relapse or death due to ALL

Efficacy Analysis Set (EAS): A subset of all infused patients who were treated with CTL019 at least 6 months prior to the clinical data cutoff. In the final analysis, it is identical to the FAS.

Percentage of participants with best overall response (BOR) of CR or CRi with MRD negative bone marrow status 6 months after CTL019 infusion among all participants who achieved CR or CRi per Local Investigator & IRC assessment

Efficacy Analysis Set (EAS): A subset of all infused patients who were treated with CTL019 at least 6 months prior to the clinical data cutoff. In the final analysis, it is identical to the FAS.

RFS is the time from achievement of CR or CRi whichever occurs first to relapse or death due to any cause during CR or CRi.

Efficacy Analysis Set (EAS): A subset of all infused patients who were treated with CTL019 at least 6 months prior to the clinical data cutoff. In the final analysis, it is identical to the FAS.

EFS is the time from date of CTL019 infusion to the earliest of death, relapse or treatment failure. Treatment failure is defined as "no response" in the study and discontinuation from the study due to any of the following reasons: death, AE, lack of efficacy, new anticancer therapy.

FAS: All subjects who received an infusion of CTL019

OS is the time from date of CTL019 infusion to the date of death due to any reason

FAS: All subjects who received an infusion of CTL019

Percentage of participants attaining CR or CRi with MRD negative bone marrow status at Day 28 +/- 4 days after CTL019 infusion per Local Investigator and IRC assessment. BM MRD were only collected and measured only within responders.

Efficacy Analysis Set (EAS): A subset of all infused patients who were treated with CTL019 at least 6 months prior to the clinical data cutoff. In the final analysis, it is identical to the FAS. BM MRD were only collected and measured only within responders.

Characterize the in vivo cellular pharmacokinetic (PK) profile (levels,persistence, trafficking) of CTL019 cells in target tissues

Pharmacokinetic Analysis Set (PAS): A subset of FAS who have at least one sample providing evaluable pharmacokinetic (PK) data

Humoral immunogenicity was measured by anti-CTL019 antibodies in human serum using a flow cytometry method. (Prevalence and incidence of immunogenicity to CTL019)

Safety set: All subjects who received an infusion of tisagenlecleucel

ORR within 6 months after infusion of CTL019 per Local & IRC assessment by baseline bone marrow tumor burden presence.

EAS: Efficacy Analysis Set (EAS): A subset of full analysis set (FAS) who were treated with CTL019 at least 6 months prior to the clinical data cutoff FAS: All subjects who received an infusion of CTL019. Data is based on data cut-off date of 6-Oct-2017, with only 42 participants and 12 having a low bone marrow tumor burden at enrollment.

ORR within 6 months after infusion of CTL019 per Local Investigator & IRC assessment by high baseline bone marrow tumor burden presence.

EAS: Efficacy Analysis Set (EAS): A subset of full analysis set (FAS) who were treated with CTL019 at least 6 months prior to the clinical data cutoff FAS: All subjects who received an infusion of CTL019. Data is based on data cut-off date of 6-Oct-2017, with only 42 participants and 30 having a high bone marrow tumor burden at enrollment

ORR within 6 months after infusion of CTL019 per Local Investigator & IRC assessment by baseline extramedullary disease presence of Yes.

EAS: Efficacy Analysis Set (EAS): A subset of full analysis set (FAS) who were treated with CTL019 at least 6 months prior to the clinical data cutoff FAS: All subjects who received an infusion of CTL019. Data is based on data cut-off date of 6-Oct-2017, with only 42 participants and 2 participants having a baseline extramedullary presence.

ORR within 6 months after infusion of CTL019 per Local Investigator & IRC assessment by baseline extramedullary disease presence of No.

EAS: Efficacy Analysis Set (EAS): A subset of full analysis set (FAS) who were treated with CTL019 at least 6 months prior to the clinical data cutoff FAS: All subjects who received an infusion of CTL019. Data is based on data cut-off date of 6-Oct-2017, with only 42 participants and 40 participants having no baseline extramedullary presence.

BM MRD status was by Local Investigator and IRC assessment within 6 months after infusion of CTL019 by baseline bone marrow tumor burden. BM MRD were collected and measured only within responders.

Efficacy Analysis Set: A subset of FAS treated with CTL019 at least 6 months prior to the clinical data cutoff FAS: All subjects who received an infusion of CTL019. Data is based on cut-off date of 6-Oct-2017, with only 42 participants and 30 having a high baseline bone marrow tumor burden. BM MRD were collected and measured only within responders

BM MRD status was per Local Investigator and IRC assessment within 6 months after infusion of CTL019 by low baseline bone marrow tumor burden. BM MRD were collected and measured only within responders.

Efficacy Analysis Set: A subset of FAS treated with CTL019 at least 6 months prior to the clinical data cutoff FAS: All subjects who received an infusion of CTL019. Data is based on cut-off date of 6-Oct-2017, with only 42 participants and 12 having a high baseline bone marrow tumor burden. BM MRD were collected and measured only within responders

BM MRD status was by Local Investigator and IRC assessment within 6 months after infusion pf CTL019 by baseline extramedullary disease presence of Yes. BM MRD were collected and measured only within responders.

Efficacy Analysis Set: A subset of FAS treated with CTL019 at least 6 months prior to the clinical data cutoff. FAS: All subjects who received an infusion of CTL019. Data is based on cut-off date of 6-Oct-2017, with only 42 participants & 2 having a BL extramedullary disease presence of Yes. BM MRD were collected & measured only within responders.

BM MRD status WAS per Local Investigator and IRC assessment within 6 months after infusion pf CTL019 by baseline extramedullary disease presence of No. BM MRD were collected and measured only within responders.

Efficacy Analysis Set: A subset of FAS treated with CTL019 at least 6 months prior to the clinical data cutoff. FAS: All subjects who received an infusion of CTL019. Data is based on cut-off date of 6-Oct-2017, with only 42 participants & 40 having a BL extramedullary disease presence of No. BM MRD were collected & measured only within responders.

DoR per Local Investigator & IRC assessment by low baseline marrow tumor burden

EAS: Efficacy Analysis Set (EAS): A subset of full analysis set (FAS) who were treated with CTL019 at least 6 months prior to the clinical data cutoff FAS: All subjects who received an infusion of CTL019. Data is based on data cut-off date of 6-Oct-2017, with only 42 participants and 12 having a low baseline marrow tumor burden

DoR per Local Investigator & IRC assessment by high baseline bone marrow tumor burden

EAS: Efficacy Analysis Set (EAS): A subset of full analysis set (FAS) who were treated with CTL019 at least 6 months prior to the clinical data cutoff FAS: All subjects who received an infusion of CTL019. Data is based on data cut-off date of 6-Oct-2017, with only 42 participants and 30 having a high baseline marrow tumor burden

DoR per Local Investigator & IRC assessment by baseline extramedullary disease presence of Yes.

EAS: Efficacy Analysis Set (EAS): A subset of full analysis set (FAS) who were treated with CTL019 at least 6 months prior to the clinical data cutoff FAS: All subjects who received an infusion of CTL019. Data is based on data cut-off date of 6-Oct-2017, with only 42 participants and 2 having a baseline extramedullary disease presence of Yes

DoR per Local Investigator & IRC assessment by baseline extramedullary disease presence of No

EAS: Efficacy Analysis Set (EAS): A subset of full analysis set (FAS) who were treated with CTL019 at least 6 months prior to the clinical data cutoff FAS: All subjects who received an infusion of CTL019. Data is based on data cut-off date of 6-Oct-2017, with only 42 participants and 40 having a baseline extramedullary disease presence of No

Activation of T cells in PBMC collected from subjects in response to mCAR19 -derived peptides was used to assess the cellular immunogenicity against tisagenlecleucel. CD4 and CD8 T cell net responses (in %) were calculated for 2 non-overlapping CTL019 peptide pools (i.e., Pool 1 and Pool 2). (Lymphocyte subsets of B and T cells and description of associated safety events)

Safety set: All subjects who received an infusion of tisagenlecleucel

Characterize the in vivo cellular pharmacokinetic (PK) profile. AUC0-28d and AUC0-84d is defined as the AUC from time zero to day 28 and 84 or other disease assessment days, in peripheral blood (% or copies/μg x days). Data was only reported for evaluable PK parameters. The Overall Number of Participants Analyzed represents all participants for which a baseline assessment was collected for this Outcome Measure, and therefore these participants did contribute data to this estimation parameter, whereas the Number Analyzed per Row represents the number of participants with data available at either 28 or 84 days.

Pharmacokinetic Analysis Set (PAS): A subset of FAS who have at least one sample providing evaluable pharmacokinetic (PK) data.

Characterize the in vivo cellular pharmacokinetic (PK) profile. Cmax is defined as the maximum (peak) observed in peripheral blood drug concentration after single dose administration (% or copies/μg).

Pharmacokinetic Analysis Set (PAS): A subset of FAS who have at least one sample providing evaluable pharmacokinetic (PK) data

Characterize the in vivo cellular pharmacokinetic (PK) profile. Tmax is defined as the time to reach maximum (peak) peripheral blood drug concentration after single dose administration (days)

Pharmacokinetic Analysis Set (PAS): A subset of FAS who have at least one sample providing evaluable pharmacokinetic (PK) data

Characterize the in vivo cellular pharmacokinetic (PK) profile. T1/2 is defined as the half-life associated with the disposition phase slopes (alpha, beta, gamma etc.) of a semi logarithmic concentration-time curve (days) in peripheral blood

Pharmacokinetic Analysis Set (PAS): A subset of FAS who have at least one sample providing evaluable pharmacokinetic (PK) data

Characterize the in vivo cellular pharmacokinetic (PK) profile. Clast is defined as the last observed quantifiable concentration in peripheral blood (% or copies/ug)

Pharmacokinetic Analysis Set (PAS): A subset of FAS who have at least one sample providing evaluable pharmacokinetic (PK) data

Characterize the in vivo cellular pharmacokinetic (PK) profile. Tlast is defined as the time of last observed quantifiable concentration in peripheral blood (days)"

Pharmacokinetic Analysis Set (PAS): A subset of FAS who have at least one sample providing evaluable pharmacokinetic (PK) data

The CD19 status of bone marrow/blood relapse was categorized as follows: CD19 positive, CD19 dim, CD19 negative, CD19 positive/negative & CD19 unknown

FAS patients who achieved CR & CRi and then relapsed. FAS: All subjects who received an infusion of CTL019

This is the site of involvement of initial relapse after achieving a best overall response of CR/CRi.

FAS patients who achieved CR & CRi and then relapsed. FAS: All subjects who received an infusion of CTL019

Time to B cell recovery was defined as the time from onset of remission to the earliest time when the percentage of CD19+ total B cell among viable WBC is ≥ 1% or among lymphocyte is at least 3%.

FAS patients who achieved CR or CRi. FAS: All subjects who received an infusion of CTL019

The levels (%) of CD19+ total B cells amongst viable white blood cells (WBC) in peripheral blood

Safety set: All subjects who received an infusion of tisagenlecleucel

C-Reactive Protein at Pre-infusion, baseline, and change from baseline for Days 7, 14, 21, 28, Month 3

Safety set: All subjects who received an infusion of tisagenlecleucel

Ferritin at Pre-infusion, baseline, and change from baseline for Days 7, 14, 21, 28, Month 3

Safety set: All subjects who received an infusion of tisagenlecleucel

INF-gamma at Pre-infusion, baseline, and change from baseline for Days 7, 14, 21, 28, Month 3

Safety set: All subjects who received an infusion of tisagenlecleucel

IL-6 at Pre-infusion, baseline, and change from baseline for Days 7, 14, 21, 28, Month 3

Safety set: All subjects who received an infusion of tisagenlecleucel

IL-2 at Pre-infusion, baseline, and change from baseline for Days 7, 14, 21, 28, Month 3

Safety set: All subjects who received an infusion of tisagenlecleucel