Number of Participants With the Indicated Hematology Parameters Falling Outside of the Reference Range at Any Time Post-Baseline During Part 1, Part 2, and Part 2/3
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
NUMBER
Unit
Participants
Interval / dispersion
Not reported
Time frame
Post-Baseline from Week 1 through Follow-up up to Study Week 35
What was measured
Hematology parameters included: erythrocytes (RR: 4.2 - 6.1 teragrams per liter \[TI/L\]), hemoglobin (RR: 125 - 165 g/L), hematocrit (RR: 0.36 - 0.46), platelets (RR: 170 - 430 gigagrams per liter \[GI/L\]), mean platelet volume (MPV, RR: 4 - 14 femotoliter \[fL\]), leukocytes (RR: 3.4 - 11.2 GI/L), total neutrophils (RR: 2.1 - 4.9 GI/L), lymphocytes (RR: 1.4 - 2.9 GI/L), monocytes (RR: 0.2 - 0.9 GI/L), eosinophils (RR: 0.2 - 0.7 GI/L), and basophils (RR: 0.02 - 0.12 GI/L). Baseline values were obtained at Day 1. The number of participants with the indicated hematology parameters data outside of the reference range (with high and low) any time post-baseline are presented. Anytime post-Baseline assesments 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,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)
Participants (par) aged between 1 and 17 years (Cohort 1 age group: 12 to 17 years, Cohort 2: 6 to 11 years and Cohort 3:1 to 5 years) received eltrombopag for 24 weeks. The starting dose for Cohort 1 was 25 mg, and par of East Asian ancestry received 12.5mg QD. For cohort 2 starting dose was based on the body weight. Par with a bodyweight of \<27 kg received 12.5 mg QD, par with a body weight of \>=27 kg received 25 mg QD; par of east Asian ancestry with a body weight \<27 kg received 12.5 mg QD, and with a body weight of \>=27 kg received 25 mg QD. For Cohort 3, the starting dose was 0.7 mg/kg QD and 0.5 mg/kg/day for par of East Asian ancestry and the dose calculations were based on the body weight. The maximum dose allowed for all Cohorts was 75mg daily. For all par, individual dose titration was allowed based upon platelet response.
Part 2 (Randomized Period) -Placebo
Par aged between 1 and 17 years (Cohort 1 age group: 12 to 17 years, Cohort 2: 6 to 11 years and Cohort 3:1 to 5 years) received eltrombopag matching placebo for 7 weeks.
Part 2 (Randomized Period) - Eltrombopag
Par aged between 1 and 17 years (Cohort 1 age group: 12 to 17 years, Cohort 2: 6 to 11 years and Cohort 3:1 to 5 years) received eltrombopag for 7 weeks. The starting dose for Cohort 1 was 37.5 mg QD. For Cohort 2, starting dose was based on the body weight. Par with a body weight of \<27 kg received 25 mg QD, and par with a body weight of \>=27 kg received 50 mg QD. Par of East Asian ancestry with a body weight of \<27 kg received 12.5 mg QD, and with a body weight of \>=27 kg received 25 mg QD. For Cohort 3, the starting dose was 1.5 mg/kg QD and 0.8 mg/kg/day for par of East Asian ancestry. The maximum dose allowed was 2mg/kg and could not exceed 75 mg daily. For all par, individual dose titration was allowed based upon platelet response.
Part 2/ 3 (Eltrombopag Open-Label Period)
Par aged between 1 and 17 years (Cohort 1 age group: 12 to 17 years, Cohort 2: 6 to 11 years and Cohort 3: 1 to 5 years), completing Part 2 of the study received an Open -Label treatment of eltrombopag administered as a tablet or dry powder for oral suspension in Part 2/3. Par who received eltrombopag during the Randomized Period continued on the same dose, unless adjustments were warranted according to the dosing guidelines, for 17 additional weeks (for a total of 24 weeks of treatment). Par who received placebo during the Randomized Period followed the starting doses for each age Cohort specified for Part 2, and received a total of 24 weeks of eltrombopag treatment.
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)
15
Part 2 (Randomized Period) -Placebo
21
Part 2 (Randomized Period) - Eltrombopag
44
Part 2/ 3 (Eltrombopag Open-Label Period)
65
Reported measurements
Erythrocytes high, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
5
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
7
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
15
Not reported
Not reported
Not reported
Not reported
Erythrocytes low, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
2
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
8
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
13
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
24
Not reported
Not reported
Not reported
Not reported
Hemoglobin, high, n= 15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
2
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
2
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
2
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
8
Not reported
Not reported
Not reported
Not reported
Hemoglobin low, n= 15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
10
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
5
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
15
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
32
Not reported
Not reported
Not reported
Not reported
Hematocrit, high, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
1
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
0
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
5
Not reported
Not reported
Not reported
Not reported
Hematocrit, low, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
10
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
11
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
22
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
39
Not reported
Not reported
Not reported
Not reported
Platelets, high, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
9
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
6
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
14
Not reported
Not reported
Not reported
Not reported
Platelets, low, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
15
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
21
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
44
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
65
Not reported
Not reported
Not reported
Not reported
Leukocytes, high, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
6
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
5
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
8
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
27
Not reported
Not reported
Not reported
Not reported
Leukocytes, low, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
4
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
5
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
10
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
19
Not reported
Not reported
Not reported
Not reported
Neutrophils, high, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
7
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
5
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
11
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
33
Not reported
Not reported
Not reported
Not reported
Neutrophils, low, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
3
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
4
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
10
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
21
Not reported
Not reported
Not reported
Not reported
Lymphocytes, high, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
1
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
3
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
5
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
11
Not reported
Not reported
Not reported
Not reported
Lymphocytes, low, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
5
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
6
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
10
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
25
Not reported
Not reported
Not reported
Not reported
Monocytes, high, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
3
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
3
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
7
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
15
Not reported
Not reported
Not reported
Not reported
Monocytes, low, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
4
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
7
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
10
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
23
Not reported
Not reported
Not reported
Not reported
Eosinophils, high, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
5
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
3
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
11
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
24
Not reported
Not reported
Not reported
Not reported
Eosinophils, low, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
4
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
2
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
5
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
9
Not reported
Not reported
Not reported
Not reported
Basophils, high, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
6
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
5
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
10
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
20
Not reported
Not reported
Not reported
Not reported
Basophils, low, n=15, 21, 44, 65
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
2
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
0
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
1
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
1
Not reported
Not reported
Not reported
Not reported
Mean Platelet Volume, high, n=12, 17, 40, 59
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
10
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
10
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
23
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
47
Not reported
Not reported
Not reported
Not reported
Mean Platelet Volume, low, n=12, 17, 40, 59
Values in Participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Part 1 (Dose-Finding Period)
4
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) -Placebo
6
Not reported
Not reported
Not reported
Not reported
Part 2 (Randomized Period) - Eltrombopag
10
Not reported
Not reported
Not reported
Not reported
Part 2/ 3 (Eltrombopag Open-Label Period)
16
Not reported
Not reported
Not reported
Not reported
Complete source fields
classes
categories
measurements
group Id
OG000
value
5
group Id
OG001
value
0
group Id
OG002
value
7
group Id
OG003
value
15
title
Erythrocytes high, n=15, 21, 44, 65
categories
measurements
group Id
OG000
value
2
group Id
OG001
value
8
group Id
OG002
value
13
group Id
OG003
value
24
title
Erythrocytes low, n=15, 21, 44, 65
categories
measurements
group Id
OG000
value
2
group Id
OG001
value
2
group Id
OG002
value
2
group Id
OG003
value
8
title
Hemoglobin, high, n= 15, 21, 44, 65
categories
measurements
group Id
OG000
value
10
group Id
OG001
value
5
group Id
OG002
value
15
group Id
OG003
value
32
title
Hemoglobin low, n= 15, 21, 44, 65
categories
measurements
group Id
OG000
value
1
group Id
OG001
value
0
group Id
OG002
value
0
group Id
OG003
value
5
title
Hematocrit, high, n=15, 21, 44, 65
categories
measurements
group Id
OG000
value
10
group Id
OG001
value
11
group Id
OG002
value
22
group Id
OG003
value
39
title
Hematocrit, low, n=15, 21, 44, 65
categories
measurements
group Id
OG000
value
9
group Id
OG001
value
0
group Id
OG002
value
6
group Id
OG003
value
14
title
Platelets, high, n=15, 21, 44, 65
categories
measurements
group Id
OG000
value
15
group Id
OG001
value
21
group Id
OG002
value
44
group Id
OG003
value
65
title
Platelets, low, n=15, 21, 44, 65
categories
measurements
group Id
OG000
value
6
group Id
OG001
value
5
group Id
OG002
value
8
group Id
OG003
value
27
title
Leukocytes, high, n=15, 21, 44, 65
categories
measurements
group Id
OG000
value
4
group Id
OG001
value
5
group Id
OG002
value
10
group Id
OG003
value
19
title
Leukocytes, low, n=15, 21, 44, 65
categories
measurements
group Id
OG000
value
7
group Id
OG001
value
5
group Id
OG002
value
11
group Id
OG003
value
33
title
Neutrophils, high, n=15, 21, 44, 65
categories
measurements
group Id
OG000
value
3
group Id
OG001
value
4
group Id
OG002
value
10
group Id
OG003
value
21
title
Neutrophils, low, n=15, 21, 44, 65
denoms
counts
group Id
OG000
value
15
group Id
OG001
value
21
group Id
OG002
value
44
group Id
OG003
value
65
units
Participants
description
Hematology parameters included: erythrocytes (RR: 4.2 - 6.1 teragrams per liter \[TI/L\]), hemoglobin (RR: 125 - 165 g/L), hematocrit (RR: 0.36 - 0.46), platelets (RR: 170 - 430 gigagrams per liter \[GI/L\]), mean platelet volume (MPV, RR: 4 - 14 femotoliter \[fL\]), leukocytes (RR: 3.4 - 11.2 GI/L), total neutrophils (RR: 2.1 - 4.9 GI/L), lymphocytes (RR: 1.4 - 2.9 GI/L), monocytes (RR: 0.2 - 0.9 GI/L), eosinophils (RR: 0.2 - 0.7 GI/L), and basophils (RR: 0.02 - 0.12 GI/L). Baseline values were obtained at Day 1. The number of participants with the indicated hematology parameters data outside of the reference range (with high and low) any time post-baseline are presented. Anytime post-Baseline assesments included any scheduled and unscheduled post-Baseline assessment
groups
description
Participants (par) aged between 1 and 17 years (Cohort 1 age group: 12 to 17 years, Cohort 2: 6 to 11 years and Cohort 3:1 to 5 years) received eltrombopag for 24 weeks. The starting dose for Cohort 1 was 25 mg, and par of East Asian ancestry received 12.5mg QD. For cohort 2 starting dose was based on the body weight. Par with a bodyweight of \<27 kg received 12.5 mg QD, par with a body weight of \>=27 kg received 25 mg QD; par of east Asian ancestry with a body weight \<27 kg received 12.5 mg QD, and with a body weight of \>=27 kg received 25 mg QD. For Cohort 3, the starting dose was 0.7 mg/kg QD and 0.5 mg/kg/day for par of East Asian ancestry and the dose calculations were based on the body weight. The maximum dose allowed for all Cohorts was 75mg daily. For all par, individual dose titration was allowed based upon platelet response.
id
OG000
title
Part 1 (Dose-Finding Period)
description
Par aged between 1 and 17 years (Cohort 1 age group: 12 to 17 years, Cohort 2: 6 to 11 years and Cohort 3:1 to 5 years) received eltrombopag matching placebo for 7 weeks.
id
OG001
title
Part 2 (Randomized Period) -Placebo
description
Par aged between 1 and 17 years (Cohort 1 age group: 12 to 17 years, Cohort 2: 6 to 11 years and Cohort 3:1 to 5 years) received eltrombopag for 7 weeks. The starting dose for Cohort 1 was 37.5 mg QD. For Cohort 2, starting dose was based on the body weight. Par with a body weight of \<27 kg received 25 mg QD, and par with a body weight of \>=27 kg received 50 mg QD. Par of East Asian ancestry with a body weight of \<27 kg received 12.5 mg QD, and with a body weight of \>=27 kg received 25 mg QD. For Cohort 3, the starting dose was 1.5 mg/kg QD and 0.8 mg/kg/day for par of East Asian ancestry. The maximum dose allowed was 2mg/kg and could not exceed 75 mg daily. For all par, individual dose titration was allowed based upon platelet response.
id
OG002
title
Part 2 (Randomized Period) - Eltrombopag
description
Par aged between 1 and 17 years (Cohort 1 age group: 12 to 17 years, Cohort 2: 6 to 11 years and Cohort 3: 1 to 5 years), completing Part 2 of the study received an Open -Label treatment of eltrombopag administered as a tablet or dry powder for oral suspension in Part 2/3. Par who received eltrombopag during the Randomized Period continued on the same dose, unless adjustments were warranted according to the dosing guidelines, for 17 additional weeks (for a total of 24 weeks of treatment). Par who received placebo during the Randomized Period followed the starting doses for each age Cohort specified for Part 2, and received a total of 24 weeks of eltrombopag treatment.
id
OG003
title
Part 2/ 3 (Eltrombopag Open-Label Period)
param Type
NUMBER
population Description
Safety Population. Only those participants available at the specified time points were analyzed (represented by n=X,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
Post-Baseline from Week 1 through Follow-up up to Study Week 35
title
Number of Participants With the Indicated Hematology Parameters Falling Outside of the Reference Range at Any Time Post-Baseline During Part 1, Part 2, and Part 2/3
Triangle