NCT02610777 · OUTCOME

Percentage of Participants With Red Blood Cells (RBCs) and Platelet-transfusion Independence

An Efficacy and Safety Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML) and Low-Blast Acute Myelogenous Leukemia (AML) · Source last updated 2022-09-19

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure: NUMBER
Unit: percentage of participants
Interval / dispersion: Not reported
Time frame: 8 weeks before randomization through 30 days after last dose of any study drug (up to approximately 5 years and 3 months)

What was measured

A participant was defined as RBC or platelet-transfusion independent if he/she received no RBC or platelet transfusions for a period of at least 8 weeks before the first dose of study drug through 30 days after the last dose of any study drug. Rate of transfusion independence was defined as number of participants who became transfusion independent divided by the number of participants who were transfusion dependent at Baseline.

Analysis population: ITT Population included all participants who were randomized. Overall number analyzed are the number of participants from a subset of the ITT Population who were transfusion dependent at Baseline. Number analyzed is the number of participants who were transfusion dependent at Baseline for the specified category.

Groups in this outcome

Azacitidine 75 mg/m^2

Azacitidine 75 mg/m\^2, infusion, intravenously or subcutaneously, on Day 1 through Day 5, Days 8 and 9 in 28-day treatment cycles until unacceptable toxicity, relapse, transformation to AML (for participants with HR MDS or CMML), or progressive disease (for participants with low-blast AML).

Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2

Azacitidine 75 mg/m\^2, infusion, intravenously or subcutaneously, on Day 1 through Day 5, Days 8 and 9 and pevonedistat 20 mg/m\^2, infusion, intravenously, on Days 1, 3, and 5 in 28-day treatment cycles until unacceptable toxicity, relapse, transformation to AML (for participants with HR MDS or CMML), or progressive disease (for participants with low-blast AML).

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
Azacitidine 75 mg/m^2	26
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2	26

Reported measurements

RBCs-transfusion Independence

Analysis denominator · Participants

Group	Source count
Azacitidine 75 mg/m^2	26
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2	26

Values in percentage of participants · Interval/dispersion: Not reported
Group	Value	Spread	Lower	Upper	Comment
Azacitidine 75 mg/m^2	50.0	Not reported	Not reported	Not reported	Not reported
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2	69.2	Not reported	Not reported	Not reported	Not reported

Platelet-transfusion Independence

Analysis denominator · Participants

Group	Source count
Azacitidine 75 mg/m^2	10
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2	5

Values in percentage of participants · Interval/dispersion: Not reported
Group	Value	Spread	Lower	Upper	Comment
Azacitidine 75 mg/m^2	60.0	Not reported	Not reported	Not reported	Not reported
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2	80.0	Not reported	Not reported	Not reported	Not reported

Source statistical analyses

Group order, estimate direction, methods and comments are retained. No treatment ranking is inferred.

ci Lower Limit
-6.925
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
45.386
group Description
Percentage of Participants With RBCs-transfusion Independence
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
=0.162
p Value Comment
P-value is from an unstratified Cochran-Mantel Haenszel chi-square test.
param Type
Absolute Rate Difference
param Value
19.231
statistical Method
Cochran-Mantel-Haenszel
ci Lower Limit
-26.381
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
66.381
group Description
Percentage of Participants With Platelet-transfusion Independence
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
=0.454
p Value Comment
P-value is from an unstratified Cochran-Mantel Haenszel chi-square test.
param Type
Absolute Rate Difference
param Value
20.000
statistical Method
Cochran-Mantel-Haenszel

Complete source fields

analyses

ci Lower Limit
-6.925
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
45.386
group Description
Percentage of Participants With RBCs-transfusion Independence
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
=0.162
p Value Comment
P-value is from an unstratified Cochran-Mantel Haenszel chi-square test.
param Type
Absolute Rate Difference
param Value
19.231
statistical Method
Cochran-Mantel-Haenszel
ci Lower Limit
-26.381
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
66.381
group Description
Percentage of Participants With Platelet-transfusion Independence
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
=0.454
p Value Comment
P-value is from an unstratified Cochran-Mantel Haenszel chi-square test.
param Type
Absolute Rate Difference
param Value
20.000
statistical Method
Cochran-Mantel-Haenszel

classes

categories
measurements
group Id
OG000
value
50.0
group Id
OG001
value
69.2
denoms
counts
group Id
OG000
value
26
group Id
OG001
value
26
units
Participants
title
RBCs-transfusion Independence
categories
measurements
group Id
OG000
value
60.0
group Id
OG001
value
80.0
denoms
counts
group Id
OG000
value
10
group Id
OG001
value
5
units
Participants
title
Platelet-transfusion Independence

denoms

counts
group Id
OG000
value
26
group Id
OG001
value
26
units
Participants

description

groups

description
Azacitidine 75 mg/m\^2, infusion, intravenously or subcutaneously, on Day 1 through Day 5, Days 8 and 9 in 28-day treatment cycles until unacceptable toxicity, relapse, transformation to AML (for participants with HR MDS or CMML), or progressive disease (for participants with low-blast AML).
id
OG000
title
Azacitidine 75 mg/m^2
description
Azacitidine 75 mg/m\^2, infusion, intravenously or subcutaneously, on Day 1 through Day 5, Days 8 and 9 and pevonedistat 20 mg/m\^2, infusion, intravenously, on Days 1, 3, and 5 in 28-day treatment cycles until unacceptable toxicity, relapse, transformation to AML (for participants with HR MDS or CMML), or progressive disease (for participants with low-blast AML).
id
OG001
title
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2

param Type

NUMBER

population Description

ITT Population included all participants who were randomized. Overall number analyzed are the number of participants from a subset of the ITT Population who were transfusion dependent at Baseline. Number analyzed is the number of participants who were transfusion dependent at Baseline for the specified category.

reporting Status

POSTED

time Frame

8 weeks before randomization through 30 days after last dose of any study drug (up to approximately 5 years and 3 months)

title

Percentage of Participants With Red Blood Cells (RBCs) and Platelet-transfusion Independence

type

SECONDARY

unit Of Measure

percentage of participants

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Preserved source evidence · Not medical advice