Number of Participants With Any Bleeding and Significant Bleeding as Assessed Using the World Health Organization (WHO) Bleeding Scale During Part 1
Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body. · Source last updated 2015-03-10
Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.
Measure
NUMBER
Unit
Participants
Interval / dispersion
Not reported
Time frame
From Baseline through Follow-up of Part 1
What was measured
The WHO Bleeding Scale is a measure of bleeding severity with the following grades: Grade 0=no bleeding; Grade 1=petechiae; Grade 2=mild blood loss; Grade 3=gross bleeding; Grade 4=debilitating blood loss. The WHO grades were dichotomized into the following categories: no bleeding=Grade 0; any bleeding=Grades 1 to 4; no clinically significant bleeding=Grades 0 to 1; clinically significant bleeding=Grades 2 to 4. Baseline was defined as the Day 1 assessment or the latest possible screening assessment.
Analysis population: ITT Population. Only those participants that did not enroll in Part 2 were analyzed during the follow-up visits. Only those participants available at the specified time points were analyzed (represented by n=X,X in the category titles).
Groups in this outcome
Part 1 (Randomized Period)-Placebo
In Part 1, participants aged between 6 and 17 years with a body weight \<27 kg received placebo 37.5 mg QD, and those with a body weight \>=27 kg received placebo 50 mg QD. Participants of East Asian ancestry received a starting dose of placebo 25 mg QD. Participants aged between 1 and 5 years received placebo 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of placebo 0.8 mg/kg/day.
Part 1 (Randomized Period) -Eltrombopag
In Part 1, participants aged between 6 and 17 years with a body weight \<27 kg received eltrombopag 37.5 mg QD, and those with a body weight \>=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. Participants aged between 1 and 5 years received eltrombopag 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day.
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 (Randomized Period)-Placebo
29
Part 1 (Randomized Period) -Eltrombopag
63
Reported measurements
Baseline, Any bleeding n=29,63
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
20
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
45
Not reported
Not reported
Not reported
Not reported
Baseline, Significant bleeding n=29,63
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
6
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
16
Not reported
Not reported
Not reported
Not reported
Week 1, Any bleeding n=29,63
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
19
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
41
Not reported
Not reported
Not reported
Not reported
Week 1, Significant bleeding n=29,63
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
3
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
8
Not reported
Not reported
Not reported
Not reported
Week 2, Any bleeding n=29,63
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
19
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
33
Not reported
Not reported
Not reported
Not reported
Week 2, Significant bleeding n=29,63
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
6
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
6
Not reported
Not reported
Not reported
Not reported
Week 3, Any bleeding n=28,62
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
18
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
32
Not reported
Not reported
Not reported
Not reported
Week 3, Significant bleeding n=28,62
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
3
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
7
Not reported
Not reported
Not reported
Not reported
Week 4, Any bleeding n=29, 62
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
20
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
27
Not reported
Not reported
Not reported
Not reported
Week 4, Significant bleeding n=29, 62
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
4
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
4
Not reported
Not reported
Not reported
Not reported
Week 5, Any bleeding n=27, 62
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
18
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
22
Not reported
Not reported
Not reported
Not reported
Week 5, Significant bleeding n=27, 62
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
2
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
5
Not reported
Not reported
Not reported
Not reported
Week 6, Any bleeding n=28, 62
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
17
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
24
Not reported
Not reported
Not reported
Not reported
Week 6, Significant bleeding n=28, 62
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
5
Not reported
Not reported
Not reported
Not reported
Week 7, Any bleeding n=28, 63
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
14
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
20
Not reported
Not reported
Not reported
Not reported
Week 7, Significant bleeding n=28, 63
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
1
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
1
Not reported
Not reported
Not reported
Not reported
Week 8, Any bleeding n=28, 63
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
17
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
24
Not reported
Not reported
Not reported
Not reported
Week 8,Significant bleeding n=28, 63
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
1
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
4
Not reported
Not reported
Not reported
Not reported
Week 9, Any bleeding n=27, 61
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
18
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
24
Not reported
Not reported
Not reported
Not reported
Week 9, Significant bleeding n=27, 61
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
3
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
4
Not reported
Not reported
Not reported
Not reported
Week 10, Any bleeding n=28, 62
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
17
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
20
Not reported
Not reported
Not reported
Not reported
Week 10, Significant bleeding n=28, 62
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
2
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
4
Not reported
Not reported
Not reported
Not reported
Week 11, Any bleeding n=28, 61
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
16
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
26
Not reported
Not reported
Not reported
Not reported
Week 11, Significant bleeding n=28, 61
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
3
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
8
Not reported
Not reported
Not reported
Not reported
Week 12, Any bleeding n=28, 61
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
16
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
23
Not reported
Not reported
Not reported
Not reported
Week 12, Significant bleeding n=28, 61
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
2
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
3
Not reported
Not reported
Not reported
Not reported
Follow-up Week 1, Any bleeding n=0,2
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
1
Not reported
Not reported
Not reported
Not reported
Follow-up Week 1, Significant bleeding n=0,2
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
0
Not reported
Not reported
Not reported
Not reported
Follow-up Week 2, Any bleeding n=0,2
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
1
Not reported
Not reported
Not reported
Not reported
Follow-up Week 2, Significant bleeding n=0,2
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
0
Not reported
Not reported
Not reported
Not reported
Follow-up Week 3, Any bleeding n=0,3
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
2
Not reported
Not reported
Not reported
Not reported
Follow-up Week 3, Significant bleeding n=0,3
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
1
Not reported
Not reported
Not reported
Not reported
Follow-up Week 4, Any bleeding n=0,1
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
1
Not reported
Not reported
Not reported
Not reported
Follow-up Week 4, Significant bleeding n=0,1
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
1
Not reported
Not reported
Not reported
Not reported
Any follow-up Week, Any bleeding n=0,3
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
2
Not reported
Not reported
Not reported
Not reported
Any Follow-up Week, Significant bleeding n=0,3
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Randomized Period)-Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 1 (Randomized Period) -Eltrombopag
1
Not reported
Not reported
Not reported
Not reported
Complete source fields
classes
categories
measurements
group Id
OG000
value
20
group Id
OG001
value
45
title
Baseline, Any bleeding n=29,63
categories
measurements
group Id
OG000
value
6
group Id
OG001
value
16
title
Baseline, Significant bleeding n=29,63
categories
measurements
group Id
OG000
value
19
group Id
OG001
value
41
title
Week 1, Any bleeding n=29,63
categories
measurements
group Id
OG000
value
3
group Id
OG001
value
8
title
Week 1, Significant bleeding n=29,63
categories
measurements
group Id
OG000
value
19
group Id
OG001
value
33
title
Week 2, Any bleeding n=29,63
categories
measurements
group Id
OG000
value
6
group Id
OG001
value
6
title
Week 2, Significant bleeding n=29,63
categories
measurements
group Id
OG000
value
18
group Id
OG001
value
32
title
Week 3, Any bleeding n=28,62
categories
measurements
group Id
OG000
value
3
group Id
OG001
value
7
title
Week 3, Significant bleeding n=28,62
categories
measurements
group Id
OG000
value
20
group Id
OG001
value
27
title
Week 4, Any bleeding n=29, 62
categories
measurements
group Id
OG000
value
4
group Id
OG001
value
4
title
Week 4, Significant bleeding n=29, 62
categories
measurements
group Id
OG000
value
18
group Id
OG001
value
22
title
Week 5, Any bleeding n=27, 62
categories
measurements
group Id
OG000
value
2
group Id
OG001
value
5
title
Week 5, Significant bleeding n=27, 62
denoms
counts
group Id
OG000
value
29
group Id
OG001
value
63
units
Participants
description
The WHO Bleeding Scale is a measure of bleeding severity with the following grades: Grade 0=no bleeding; Grade 1=petechiae; Grade 2=mild blood loss; Grade 3=gross bleeding; Grade 4=debilitating blood loss. The WHO grades were dichotomized into the following categories: no bleeding=Grade 0; any bleeding=Grades 1 to 4; no clinically significant bleeding=Grades 0 to 1; clinically significant bleeding=Grades 2 to 4. Baseline was defined as the Day 1 assessment or the latest possible screening assessment.
groups
description
In Part 1, participants aged between 6 and 17 years with a body weight \<27 kg received placebo 37.5 mg QD, and those with a body weight \>=27 kg received placebo 50 mg QD. Participants of East Asian ancestry received a starting dose of placebo 25 mg QD. Participants aged between 1 and 5 years received placebo 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of placebo 0.8 mg/kg/day.
id
OG000
title
Part 1 (Randomized Period)-Placebo
description
In Part 1, participants aged between 6 and 17 years with a body weight \<27 kg received eltrombopag 37.5 mg QD, and those with a body weight \>=27 kg received eltrombopag 50 mg QD. Participants of East Asian ancestry received a starting dose of eltrombopag 25 mg QD. Participants aged between 1 and 5 years received eltrombopag 1.2 mg/kg QD; participants of East Asian ancestry received a starting dose of eltrombopag 0.8 mg/kg/day.
id
OG001
title
Part 1 (Randomized Period) -Eltrombopag
param Type
NUMBER
population Description
ITT Population. Only those participants that did not enroll in Part 2 were analyzed during the follow-up visits. Only those participants available at the specified time points were analyzed (represented by n=X,X in the category titles).
reporting Status
POSTED
time Frame
From Baseline through Follow-up of Part 1
title
Number of Participants With Any Bleeding and Significant Bleeding as Assessed Using the World Health Organization (WHO) Bleeding Scale During Part 1
Triangle