Effects of CTL019 Therapy on Patient Reported Outcomes as Measured by PedsQL Questionnaire
Study of Efficacy and Safety of CTL019 in Pediatric ALL Patients · Source last updated 2024-02-13
Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.
Measure
MEAN
Unit
Scores on a scale
Interval / dispersion
Standard Error
Time frame
Month 3, M6, M12, M24, M60
What was measured
The PedsQL questionnaire was for patients ≥ 8-years-old who achieved BOR as CR or CRi within 3 months and the questionnaire was on emotional, social, school, physical, and psychosocial health. Scores are transformed on a scale from 0 to 100, with the sum of all the items over the number of items answered on all the scales. The total scale score is the averaged value of scores of subscales, which means for each subscale and the total scale, the allowed ranges are 0-100. Higher scores on the PedsQL questionnaire for these subscales indicate consistent improvement of health-related quality of life (HRQol).
Analysis population: FAS: the Full analysis set comprised all patients who received infusion of tisagenlecleucel and who had evaluable results at the measured time points. This analysis included a subset of FAS who had any data of the particular questionnaire at the specific timepoint. Therefore, no participants in Cohort 1 had questionnaire data for tisagenlecleucel at the measured time points, so no data was collected for Cohort 1.
Groups in this outcome
Main Cohort: Single Dose of CTL019
Pediatric patients with relapsed or refractory B-cell ALL who were treated with single dose of tisagenlecleucel (CTL019).
Cohort 1: Single Dose of CTL019
Pediatric B-ALL patients who are very high risk at first relapse.
Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
39
Cohort 1: Single Dose of CTL019
0
Reported measurements
M3 change from baseline - Emotional
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
39
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
14.5
17.98
Not reported
Not reported
Not reported
M6 change from baseline - Emotional
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
37
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
15.9
18.96
Not reported
Not reported
Not reported
M12 change from baseline - Emotional
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
23
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
24.6
23.74
Not reported
Not reported
Not reported
M24 change from baseline - Emotional
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
20
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
27.02
21.85
Not reported
Not reported
Not reported
M60 change from baseline - Emotional
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
14
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
21.4
24.53
Not reported
Not reported
Not reported
M3 change from baseline - Social
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
39
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
7.6
13.90
Not reported
Not reported
Not reported
M6 change from baseline - Social
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
37
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
8.4
17.04
Not reported
Not reported
Not reported
M12 change from baseline - Social
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
23
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
14.8
16.68
Not reported
Not reported
Not reported
M24 change from baseline - Social
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
20
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
17.3
15.85
Not reported
Not reported
Not reported
M60 change from baseline - Social
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
14
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
17.9
14.77
Not reported
Not reported
Not reported
M3 change from baseline - School
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
30
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
8.9
14.35
Not reported
Not reported
Not reported
M6 change from baseline - School
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
29
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
10.0
16.58
Not reported
Not reported
Not reported
M12 change from baseline - School
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
20
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
19.0
19.97
Not reported
Not reported
Not reported
M24 change from baseline - School
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
18
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
13.9
23.98
Not reported
Not reported
Not reported
M60 change from baseline - School
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
13
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
17.7
24.46
Not reported
Not reported
Not reported
M3 change from baseline - Physical
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
39
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
17.5
18.36
Not reported
Not reported
Not reported
M6 change from baseline - Physical
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
37
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
21.6
25.81
Not reported
Not reported
Not reported
M12 change from baseline - Physical
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
23
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
31.1
28.57
Not reported
Not reported
Not reported
M24 change from baseline - Physical
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
20
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
37.4
25.12
Not reported
Not reported
Not reported
M60 change from baseline - Physical
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
14
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
37.1
24.90
Not reported
Not reported
Not reported
M3 change from baseline - Psychosocial health
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
39
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
10.4
12.38
Not reported
Not reported
Not reported
M6 change from baseline - Psychosocial health
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
37
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
11.0
14.08
Not reported
Not reported
Not reported
M12 change from baseline - Psychosocial health
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
23
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
19.8
16.80
Not reported
Not reported
Not reported
M24 change from baseline - Psychosocial health
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
20
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
20.1
16.34
Not reported
Not reported
Not reported
M60 change from baseline - Psychosocial health
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
14
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
18.9
15.15
Not reported
Not reported
Not reported
M3 change from baseline - Total score
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
39
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
13.0
13.28
Not reported
Not reported
Not reported
M6 change from baseline - Total score
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
37
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
14.8
17.00
Not reported
Not reported
Not reported
M12 change from baseline - Total score
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
23
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
2.8
19.56
Not reported
Not reported
Not reported
M24 change from baseline - Total score
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
20
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
26.2
16.70
Not reported
Not reported
Not reported
M60 change from baseline - Total score
Analysis denominator · Participants
Group
Source count
Main Cohort: Single Dose of CTL019
14
Cohort 1: Single Dose of CTL019
0
Values in Scores on a scale · Interval/dispersion: Standard Error
Group
Value
Spread
Lower
Upper
Comment
Main Cohort: Single Dose of CTL019
25.3
15.45
Not reported
Not reported
Not reported
Complete source fields
classes
categories
measurements
group Id
OG000
spread
17.98
value
14.5
denoms
counts
group Id
OG000
value
39
group Id
OG001
value
0
units
Participants
title
M3 change from baseline - Emotional
categories
measurements
group Id
OG000
spread
18.96
value
15.9
denoms
counts
group Id
OG000
value
37
group Id
OG001
value
0
units
Participants
title
M6 change from baseline - Emotional
categories
measurements
group Id
OG000
spread
23.74
value
24.6
denoms
counts
group Id
OG000
value
23
group Id
OG001
value
0
units
Participants
title
M12 change from baseline - Emotional
categories
measurements
group Id
OG000
spread
21.85
value
27.02
denoms
counts
group Id
OG000
value
20
group Id
OG001
value
0
units
Participants
title
M24 change from baseline - Emotional
categories
measurements
group Id
OG000
spread
24.53
value
21.4
denoms
counts
group Id
OG000
value
14
group Id
OG001
value
0
units
Participants
title
M60 change from baseline - Emotional
categories
measurements
group Id
OG000
spread
13.90
value
7.6
denoms
counts
group Id
OG000
value
39
group Id
OG001
value
0
units
Participants
title
M3 change from baseline - Social
categories
measurements
group Id
OG000
spread
17.04
value
8.4
denoms
counts
group Id
OG000
value
37
group Id
OG001
value
0
units
Participants
title
M6 change from baseline - Social
categories
measurements
group Id
OG000
spread
16.68
value
14.8
denoms
counts
group Id
OG000
value
23
group Id
OG001
value
0
units
Participants
title
M12 change from baseline - Social
categories
measurements
group Id
OG000
spread
15.85
value
17.3
denoms
counts
group Id
OG000
value
20
group Id
OG001
value
0
units
Participants
title
M24 change from baseline - Social
categories
measurements
group Id
OG000
spread
14.77
value
17.9
denoms
counts
group Id
OG000
value
14
group Id
OG001
value
0
units
Participants
title
M60 change from baseline - Social
categories
measurements
group Id
OG000
spread
14.35
value
8.9
denoms
counts
group Id
OG000
value
30
group Id
OG001
value
0
units
Participants
title
M3 change from baseline - School
categories
measurements
group Id
OG000
spread
16.58
value
10.0
denoms
counts
group Id
OG000
value
29
group Id
OG001
value
0
units
Participants
title
M6 change from baseline - School
denoms
counts
group Id
OG000
value
39
group Id
OG001
value
0
units
Participants
description
The PedsQL questionnaire was for patients ≥ 8-years-old who achieved BOR as CR or CRi within 3 months and the questionnaire was on emotional, social, school, physical, and psychosocial health. Scores are transformed on a scale from 0 to 100, with the sum of all the items over the number of items answered on all the scales. The total scale score is the averaged value of scores of subscales, which means for each subscale and the total scale, the allowed ranges are 0-100. Higher scores on the PedsQL questionnaire for these subscales indicate consistent improvement of health-related quality of life (HRQol).
dispersion Type
Standard Error
groups
description
Pediatric patients with relapsed or refractory B-cell ALL who were treated with single dose of tisagenlecleucel (CTL019).
id
OG000
title
Main Cohort: Single Dose of CTL019
description
Pediatric B-ALL patients who are very high risk at first relapse.
id
OG001
title
Cohort 1: Single Dose of CTL019
param Type
MEAN
population Description
FAS: the Full analysis set comprised all patients who received infusion of tisagenlecleucel and who had evaluable results at the measured time points. This analysis included a subset of FAS who had any data of the particular questionnaire at the specific timepoint. Therefore, no participants in Cohort 1 had questionnaire data for tisagenlecleucel at the measured time points, so no data was collected for Cohort 1.
reporting Status
POSTED
time Frame
Month 3, M6, M12, M24, M60
title
Effects of CTL019 Therapy on Patient Reported Outcomes as Measured by PedsQL Questionnaire
Triangle