Safety and Efficacy Study of Romiplostim to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients · Source last updated 2017-02-09
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 the first dose of study drug until 4 weeks after last dose; 28 weeks.
What was measured
A serious adverse event is defined as an adverse event that meets at least 1 of the following serious criteria:
* fatal,
* life threatening (places the subject at immediate risk of death),
* requires in-patient hospitalization or prolongation of existing hospitalization,
* results in persistent or significant disability/incapacity,
* congenital anomaly/birth defect, and/or
* other significant medical hazard. Adverse events were graded for severity according to the CTCAE version 3.0 grading scale, where Grade 3 = moderate, Grade 4 = life-threatening and Grade 5 = fatal.
Treatment-related adverse events (TRAEs) were those assessed by the investigator as possibly related to study drug. This relationship was determined by a "yes" or "no" response to the question: "Is there a reasonable possibility that the event may have been caused by study drug?"
Analysis population: Safety analysis set (all participants who received at least one dose of study drug)
Groups in this outcome
Placebo
Participants received weekly subcutaneous placebo for 24 weeks.
Romiplostim
Participants received once weekly subcutaneous romiplostim for 24 weeks at a starting dose of 1 µg/kg; weekly dose increases continued in increments of 1 µg/kg/week to a maximum dose of 10 µg/kg in an attempt to reach a target platelet count of ≥ 50 x 10\^9/L.
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
Placebo
19
Romiplostim
42
Reported measurements
Any adverse event (AE)
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Placebo
19
Not reported
Not reported
Not reported
Not reported
Romiplostim
41
Not reported
Not reported
Not reported
Not reported
Serious adverse events (SAE)
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Placebo
1
Not reported
Not reported
Not reported
Not reported
Romiplostim
10
Not reported
Not reported
Not reported
Not reported
AE leading to discontinuation of study drug
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Placebo
0
Not reported
Not reported
Not reported
Not reported
Romiplostim
0
Not reported
Not reported
Not reported
Not reported
AE leading to discontinuation from study
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Placebo
0
Not reported
Not reported
Not reported
Not reported
Romiplostim
0
Not reported
Not reported
Not reported
Not reported
Grade 3 AE
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Placebo
3
Not reported
Not reported
Not reported
Not reported
Romiplostim
6
Not reported
Not reported
Not reported
Not reported
Grade 4 AE
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Placebo
1
Not reported
Not reported
Not reported
Not reported
Romiplostim
2
Not reported
Not reported
Not reported
Not reported
Grade 5 AE
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Placebo
0
Not reported
Not reported
Not reported
Not reported
Romiplostim
0
Not reported
Not reported
Not reported
Not reported
Treatment-related adverse events (TRAE)
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Placebo
5
Not reported
Not reported
Not reported
Not reported
Romiplostim
11
Not reported
Not reported
Not reported
Not reported
Treatment-related serious adverse events
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Placebo
0
Not reported
Not reported
Not reported
Not reported
Romiplostim
1
Not reported
Not reported
Not reported
Not reported
TRAE leading to discontinuation of study drug
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Placebo
0
Not reported
Not reported
Not reported
Not reported
Romiplostim
0
Not reported
Not reported
Not reported
Not reported
TRAE leading to discontinuation from study
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Placebo
0
Not reported
Not reported
Not reported
Not reported
Romiplostim
0
Not reported
Not reported
Not reported
Not reported
TRAE Grade 3
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Placebo
0
Not reported
Not reported
Not reported
Not reported
Romiplostim
1
Not reported
Not reported
Not reported
Not reported
TRAE Grade 4
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Placebo
0
Not reported
Not reported
Not reported
Not reported
Romiplostim
0
Not reported
Not reported
Not reported
Not reported
TRAE Grade 5
Values in participants · Interval/dispersion: Not reported
Group
Value
Spread
Lower
Upper
Comment
Placebo
0
Not reported
Not reported
Not reported
Not reported
Romiplostim
0
Not reported
Not reported
Not reported
Not reported
Complete source fields
classes
categories
measurements
group Id
OG000
value
19
group Id
OG001
value
41
title
Any adverse event (AE)
categories
measurements
group Id
OG000
value
1
group Id
OG001
value
10
title
Serious adverse events (SAE)
categories
measurements
group Id
OG000
value
0
group Id
OG001
value
0
title
AE leading to discontinuation of study drug
categories
measurements
group Id
OG000
value
0
group Id
OG001
value
0
title
AE leading to discontinuation from study
categories
measurements
group Id
OG000
value
3
group Id
OG001
value
6
title
Grade 3 AE
categories
measurements
group Id
OG000
value
1
group Id
OG001
value
2
title
Grade 4 AE
categories
measurements
group Id
OG000
value
0
group Id
OG001
value
0
title
Grade 5 AE
categories
measurements
group Id
OG000
value
5
group Id
OG001
value
11
title
Treatment-related adverse events (TRAE)
categories
measurements
group Id
OG000
value
0
group Id
OG001
value
1
title
Treatment-related serious adverse events
categories
measurements
group Id
OG000
value
0
group Id
OG001
value
0
title
TRAE leading to discontinuation of study drug
categories
measurements
group Id
OG000
value
0
group Id
OG001
value
0
title
TRAE leading to discontinuation from study
categories
measurements
group Id
OG000
value
0
group Id
OG001
value
1
title
TRAE Grade 3
denoms
counts
group Id
OG000
value
19
group Id
OG001
value
42
units
Participants
description
A serious adverse event is defined as an adverse event that meets at least 1 of the following serious criteria:
* fatal,
* life threatening (places the subject at immediate risk of death),
* requires in-patient hospitalization or prolongation of existing hospitalization,
* results in persistent or significant disability/incapacity,
* congenital anomaly/birth defect, and/or
* other significant medical hazard. Adverse events were graded for severity according to the CTCAE version 3.0 grading scale, where Grade 3 = moderate, Grade 4 = life-threatening and Grade 5 = fatal.
Treatment-related adverse events (TRAEs) were those assessed by the investigator as possibly related to study drug. This relationship was determined by a "yes" or "no" response to the question: "Is there a reasonable possibility that the event may have been caused by study drug?"
groups
description
Participants received weekly subcutaneous placebo for 24 weeks.
id
OG000
title
Placebo
description
Participants received once weekly subcutaneous romiplostim for 24 weeks at a starting dose of 1 µg/kg; weekly dose increases continued in increments of 1 µg/kg/week to a maximum dose of 10 µg/kg in an attempt to reach a target platelet count of ≥ 50 x 10\^9/L.
id
OG001
title
Romiplostim
param Type
NUMBER
population Description
Safety analysis set (all participants who received at least one dose of study drug)
reporting Status
POSTED
time Frame
From the first dose of study drug until 4 weeks after last dose; 28 weeks.
Triangle