Mean Pulse Rate at Baseline and the Maximum Post-Baseline Visit Recorded During the Dose-Finding Period, Part 1
Efficacy and Safety Study of Eltrombopag in Pediatric Patients With Thrombocytopenia From Chronic Idiopathic Thrombocytopenic Purpura (ITP) · Source last updated 2018-10-12
Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.
Measure
MEAN
Unit
Beats per minute
Interval / dispersion
Standard Deviation
Time frame
From Week 1 to Follow-up Week 4 of Part 1, up to Study Week 28
What was measured
Pulse rate was measured at the following scheduled time points: Screening, Day 1, each week from Week 1 to Week 24, and at each Follow-up Weeks 1-4. Baseline is defined as the value obtained on Day 1 of treatment. The maximum post-Baseline (MPB) visit included any scheduled and unscheduled post-Baseline assessment..
Analysis population: Safety Population. Only those participants available at the specified time points were analyzed (represented by n=X,X,X in the category titles). Different participants may have been analyzed at different time points, so the overall number of participants analyzed reflects everyone in the Safety Population
Groups in this outcome
Part 1 (Dose-Finding Period) Cohort 1
Participants aged between 12 and 17 years received a 24-week Open-label treatment of eltrombopag administered as a tablet. The starting dose of eltrombopag was 25 milligrams (mg), once daily (QD). The participants of East Asian ancestry began at 12.5mg QD. The maximum dose allowed was 75 mg daily. All participants enrolled in the study underwent individual dose titration based upon platelet response.
Part 1 (Dose-Finding Period) Cohort 2
Participants aged between 6 and 11 years received a 24-week Open-Label treatment of eltrombopag administered as a tablet. The starting dose of eltrombopag was based on the body weight. Participants with a weight of \<27 kilograms (kg) received 12.5 mg QD (approximately 0.5 - 0.7 mg/kg QD) and participants with a weight of \>=27 kg received 25 mg QD (approximately 0.5 - 0.8 mg/kg QD). The maximum dose allowed was 2 mg/kg and could not exceed 75 mg daily. All participants enrolled in the study underwent individual dose titration based upon platelet response.
Part 1 (Dose-Finding Period) Cohort 3
Participants aged between 1 and 5 years received a 24-week Open-Label treatment of eltrombopag administered as a dry powder for oral suspension. The starting dose of eltrombopag was 0.7 mg/kg QD. Participants of East Asian ancestry began at 0.5 mg/kg/day. The maximum dose allowed was 2 mg/kg, and could not exceed 75 mg daily. All participants enrolled in the study underwent individual dose titration based upon platelet response.
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
Part 1 (Dose-Finding Period) Cohort 1
5
Part 1 (Dose-Finding Period) Cohort 2
5
Part 1 (Dose-Finding Period) Cohort 3
5
Reported measurements
Screening, n=5, 5, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
85.2
15.71
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
88.4
8.50
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
102.0
7.97
Not reported
Not reported
Not reported
Day 1, n=4, 5, 5,
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
84.3
11.32
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
88.2
12.89
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
102.4
13.22
Not reported
Not reported
Not reported
Week 1, n= 4, 5, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
85.3
4.79
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
90.8
11.19
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
103.8
8.47
Not reported
Not reported
Not reported
Week 2, n=5, 5, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
80.4
12.97
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
84.4
16.77
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
96.8
10.66
Not reported
Not reported
Not reported
Week 3, n=5, 5, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
92.8
31.16
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
91.4
11.04
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
106.2
11.10
Not reported
Not reported
Not reported
Week 4, n=5, 5, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
86.4
10.92
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
84.0
7.62
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
98.6
14.29
Not reported
Not reported
Not reported
Week 5, n=5, 5, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
84.6
9.48
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
88.4
9.58
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
107.8
9.58
Not reported
Not reported
Not reported
Week 6, n=5, 5, 4
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
76.4
10.50
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
89.8
13.79
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
103.0
8.29
Not reported
Not reported
Not reported
Week 7, n=5, 5, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
82.0
9.46
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
96.2
17.84
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
103.2
8.93
Not reported
Not reported
Not reported
Week 8, n=5, 5, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
83.0
5.20
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
89.4
15.04
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
111.8
10.62
Not reported
Not reported
Not reported
Week 9, n=5, 5, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
80.4
13.35
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
79.2
11.34
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
101.6
5.59
Not reported
Not reported
Not reported
Week 10, n=5, 5, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
80.6
14.93
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
84.4
10.31
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
105.4
9.29
Not reported
Not reported
Not reported
Week 11, n=5, 3, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
86.6
11.04
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
97.7
16.50
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
106.6
11.19
Not reported
Not reported
Not reported
Week 12, n=4, 4, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
80.5
12.07
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
92.0
12.83
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
101.4
16.62
Not reported
Not reported
Not reported
Week 13, n=4, 4, 4
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
90.8
15.52
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
86.5
7.05
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
103.8
3.50
Not reported
Not reported
Not reported
Week 14, n=3, 3, 3
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
81.0
9.85
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
87.0
8.66
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
101.0
11.00
Not reported
Not reported
Not reported
Week 15, n=3, 4, 4
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
77.3
6.66
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
86.8
11.87
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
102.5
3.87
Not reported
Not reported
Not reported
Week 16, n=5, 3, 4
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
86.4
18.60
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
103.0
5.57
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
93.0
18.96
Not reported
Not reported
Not reported
Week 17, n=2, 0, 3
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
67.5
20.51
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
NA
NA
Not reported
Not reported
There were no participants analyzed at this time point, therefore there is no calculated standard deviation.
Part 1 (Dose-Finding Period) Cohort 3
97.7
12.01
Not reported
Not reported
Not reported
Week 18, n=3, 3, 4
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
88.0
10.44
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
86.7
17.01
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
102.8
16.88
Not reported
Not reported
Not reported
Week 19, n=3, 0, 2
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
88.0
20.81
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
NA
NA
Not reported
Not reported
There were no participants analyzed at this time point, therefore there is no calculated standard deviation.
Part 1 (Dose-Finding Period) Cohort 3
94.0
8.49
Not reported
Not reported
Not reported
Week 20, n=5, 4, 4
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
79.8
13.03
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
96.8
12.58
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
111.8
14.97
Not reported
Not reported
Not reported
Week 21, n=3, 2, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
79.0
6.93
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
75.5
3.54
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
101.0
18.63
Not reported
Not reported
Not reported
Week 22, n=3, 4, 4
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
70.3
14.36
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
86.5
9.04
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
100.0
18.63
Not reported
Not reported
Not reported
Week 23, n=4, 2, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
71.5
13.03
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
82.5
3.54
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
100.0
13.14
Not reported
Not reported
Not reported
Week 24, n=5, 5, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
79.6
7.86
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
85.8
6.91
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
96.0
4.74
Not reported
Not reported
Not reported
FU Week 1, n=1, 1, 3
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
81.0
NA
Not reported
Not reported
There were too few participants available to collect data at this time point to calculate a standard deviation.
Part 1 (Dose-Finding Period) Cohort 2
125.0
NA
Not reported
Not reported
There were too few participants available to collect data at this time point to calculate a standard deviation.
Part 1 (Dose-Finding Period) Cohort 3
107.7
10.79
Not reported
Not reported
Not reported
FU Week 2, n=3, 2, 0
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
77.7
8.74
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
84.5
28.99
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
NA
NA
Not reported
Not reported
There were no participants analyzed at this time point, therefore there is no calculated standard deviation.
FU Week 3, n=2, 1, 2
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
87.0
2.83
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
78.0
NA
Not reported
Not reported
There were too few participants available to collect data at this time point to calculate a standard deviation.
Part 1 (Dose-Finding Period) Cohort 3
98.0
8.49
Not reported
Not reported
Not reported
FU Week 4, n=1, 0, 2
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
89.0
NA
Not reported
Not reported
There were too few participants available to collect data at this time point to calculate a standard deviation.
Part 1 (Dose-Finding Period) Cohort 2
NA
NA
Not reported
Not reported
There were no participants analyzed at this time point, therefore there is no calculated standard deviation.
Part 1 (Dose-Finding Period) Cohort 3
99.0
26.87
Not reported
Not reported
Not reported
MBP, n=5, 5, 5
Values in Beats per minute · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period) Cohort 1
108.8
24.01
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 2
109.8
14.17
Not reported
Not reported
Not reported
Part 1 (Dose-Finding Period) Cohort 3
119.6
5.73
Not reported
Not reported
Not reported
Complete source fields
classes
categories
measurements
group Id
OG000
spread
15.71
value
85.2
group Id
OG001
spread
8.50
value
88.4
group Id
OG002
spread
7.97
value
102.0
title
Screening, n=5, 5, 5
categories
measurements
group Id
OG000
spread
11.32
value
84.3
group Id
OG001
spread
12.89
value
88.2
group Id
OG002
spread
13.22
value
102.4
title
Day 1, n=4, 5, 5,
categories
measurements
group Id
OG000
spread
4.79
value
85.3
group Id
OG001
spread
11.19
value
90.8
group Id
OG002
spread
8.47
value
103.8
title
Week 1, n= 4, 5, 5
categories
measurements
group Id
OG000
spread
12.97
value
80.4
group Id
OG001
spread
16.77
value
84.4
group Id
OG002
spread
10.66
value
96.8
title
Week 2, n=5, 5, 5
categories
measurements
group Id
OG000
spread
31.16
value
92.8
group Id
OG001
spread
11.04
value
91.4
group Id
OG002
spread
11.10
value
106.2
title
Week 3, n=5, 5, 5
categories
measurements
group Id
OG000
spread
10.92
value
86.4
group Id
OG001
spread
7.62
value
84.0
group Id
OG002
spread
14.29
value
98.6
title
Week 4, n=5, 5, 5
categories
measurements
group Id
OG000
spread
9.48
value
84.6
group Id
OG001
spread
9.58
value
88.4
group Id
OG002
spread
9.58
value
107.8
title
Week 5, n=5, 5, 5
categories
measurements
group Id
OG000
spread
10.50
value
76.4
group Id
OG001
spread
13.79
value
89.8
group Id
OG002
spread
8.29
value
103.0
title
Week 6, n=5, 5, 4
categories
measurements
group Id
OG000
spread
9.46
value
82.0
group Id
OG001
spread
17.84
value
96.2
group Id
OG002
spread
8.93
value
103.2
title
Week 7, n=5, 5, 5
categories
measurements
group Id
OG000
spread
5.20
value
83.0
group Id
OG001
spread
15.04
value
89.4
group Id
OG002
spread
10.62
value
111.8
title
Week 8, n=5, 5, 5
categories
measurements
group Id
OG000
spread
13.35
value
80.4
group Id
OG001
spread
11.34
value
79.2
group Id
OG002
spread
5.59
value
101.6
title
Week 9, n=5, 5, 5
categories
measurements
group Id
OG000
spread
14.93
value
80.6
group Id
OG001
spread
10.31
value
84.4
group Id
OG002
spread
9.29
value
105.4
title
Week 10, n=5, 5, 5
denoms
counts
group Id
OG000
value
5
group Id
OG001
value
5
group Id
OG002
value
5
units
Participants
description
Pulse rate was measured at the following scheduled time points: Screening, Day 1, each week from Week 1 to Week 24, and at each Follow-up Weeks 1-4. Baseline is defined as the value obtained on Day 1 of treatment. The maximum post-Baseline (MPB) visit included any scheduled and unscheduled post-Baseline assessment..
dispersion Type
Standard Deviation
groups
description
Participants aged between 12 and 17 years received a 24-week Open-label treatment of eltrombopag administered as a tablet. The starting dose of eltrombopag was 25 milligrams (mg), once daily (QD). The participants of East Asian ancestry began at 12.5mg QD. The maximum dose allowed was 75 mg daily. All participants enrolled in the study underwent individual dose titration based upon platelet response.
id
OG000
title
Part 1 (Dose-Finding Period) Cohort 1
description
Participants aged between 6 and 11 years received a 24-week Open-Label treatment of eltrombopag administered as a tablet. The starting dose of eltrombopag was based on the body weight. Participants with a weight of \<27 kilograms (kg) received 12.5 mg QD (approximately 0.5 - 0.7 mg/kg QD) and participants with a weight of \>=27 kg received 25 mg QD (approximately 0.5 - 0.8 mg/kg QD). The maximum dose allowed was 2 mg/kg and could not exceed 75 mg daily. All participants enrolled in the study underwent individual dose titration based upon platelet response.
id
OG001
title
Part 1 (Dose-Finding Period) Cohort 2
description
Participants aged between 1 and 5 years received a 24-week Open-Label treatment of eltrombopag administered as a dry powder for oral suspension. The starting dose of eltrombopag was 0.7 mg/kg QD. Participants of East Asian ancestry began at 0.5 mg/kg/day. The maximum dose allowed was 2 mg/kg, and could not exceed 75 mg daily. All participants enrolled in the study underwent individual dose titration based upon platelet response.
id
OG002
title
Part 1 (Dose-Finding Period) Cohort 3
param Type
MEAN
population Description
Safety Population. Only those participants available at the specified time points were analyzed (represented by n=X,X,X in the category titles). Different participants may have been analyzed at different time points, so the overall number of participants analyzed reflects everyone in the Safety Population
reporting Status
POSTED
time Frame
From Week 1 to Follow-up Week 4 of Part 1, up to Study Week 28
title
Mean Pulse Rate at Baseline and the Maximum Post-Baseline Visit Recorded During the Dose-Finding Period, Part 1
Triangle