NCT03268954 · OUTCOME

Number of Participants With Overall Response by Cycle 6

Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML), or Low-Blast Acute Myelogenous Leukemia (AML) · Source last updated 2025-10-29

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

Measure: COUNT_OF_PARTICIPANTS
Unit: Participants
Interval / dispersion: Not reported
Time frame: Up to Cycle 6 (up to approximately Day 168)

What was measured

Responses for HR MDS/CMML are based on Modified International Working Group (IWG) Response Criteria for MDS and for low-blast AML on Revised IWG Response Criteria for AML. Overall response=CR and PR for HR MDS/CMML and CR+CR with incomplete blood count recovery(CRi)+PR for low-blast AML. CR for HR MDS/CMML:\<=5% myeloblasts with normal maturation of all bone marrow cell lines,\>=11g/dL hemoglobin (Hgb),\>=100\*10\^9/L platelet (pl),\>=1.0\*10\^9/L neutrophils, 0% blasts in peripheral blood, and PR:all CR criteria met except bone marrow blasts \>=50% decrease over pretreatment but still \>5%. For low-blast AML-CR:morphologic leukemia-free state,\>1.0\*10\^9 neutrophils, \>=100\*10\^9/L pl, transfusion independence, no residual evidence of extramedullary leukemia;CRi:fulfill CR criteria except residual neutropenia \<1.0\*10\^9/L/thrombocytopenia (pl\<100\*10\^9/L); PR:all CR hematological values but\>=50% decrease in percentage of blasts to 5%-25% in bone marrow aspirate.

Analysis population: REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 post-baseline disease assessment. Number analyzed is the number of participants with data available for analysis for the specified category.

Groups in this outcome

Azacitidine 75 mg/m^2

Participants were administered azacitidine 75 mg/m\^2 IV or SC injection on Days 1 to 5, Days 8 and 9, in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles.

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

Participants were administered azacitidine 75 mg/m\^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m\^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles.

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	206
Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2	208

Reported measurements

Overall Response for HR MDS/CMML

Analysis denominator · Participants

Group	Source count
Azacitidine 75 mg/m^2	157
Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2	163

Values in Participants · Interval/dispersion: Not reported
Group	Value	Spread	Lower	Upper	Comment
Azacitidine 75 mg/m^2	36	Not reported	Not reported	Not reported	Not reported
Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2	29	Not reported	Not reported	Not reported	Not reported

Overall Response for Low-blast AML

Analysis denominator · Participants

Group	Source count
Azacitidine 75 mg/m^2	49
Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2	45

Values in Participants · Interval/dispersion: Not reported
Group	Value	Spread	Lower	Upper	Comment
Azacitidine 75 mg/m^2	13	Not reported	Not reported	Not reported	Not reported
Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2	22	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
-13.95
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
3.68
group Description
Overall Response for HR MDS/CMML
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
=0.261
p Value Comment
P-value was obtained from a stratified Cochran-Mantel-Haenszel chi-square test stratified by IPSS-R risk groups of very high, high, or intermediate for HR MDS/CMML.
param Type
Absolute Rate Difference
param Value
-5.14
statistical Method
Cochran-Mantel-Haenszel
ci Lower Limit
3.22
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
41.49
group Description
Overall Response for Low-blast AML
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
=0.026
p Value Comment
P-value was obtained from an unstratified Cochran-Mantel-Haenszel chi-square test.
param Type
Absolute Rate Difference
param Value
22.36
statistical Method
Cochran-Mantel-Haenszel

Complete source fields

analyses

ci Lower Limit
-13.95
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
3.68
group Description
Overall Response for HR MDS/CMML
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
=0.261
p Value Comment
P-value was obtained from a stratified Cochran-Mantel-Haenszel chi-square test stratified by IPSS-R risk groups of very high, high, or intermediate for HR MDS/CMML.
param Type
Absolute Rate Difference
param Value
-5.14
statistical Method
Cochran-Mantel-Haenszel
ci Lower Limit
3.22
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
41.49
group Description
Overall Response for Low-blast AML
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
=0.026
p Value Comment
P-value was obtained from an unstratified Cochran-Mantel-Haenszel chi-square test.
param Type
Absolute Rate Difference
param Value
22.36
statistical Method
Cochran-Mantel-Haenszel

classes

categories
measurements
group Id
OG000
value
36
group Id
OG001
value
29
denoms
counts
group Id
OG000
value
157
group Id
OG001
value
163
units
Participants
title
Overall Response for HR MDS/CMML
categories
measurements
group Id
OG000
value
13
group Id
OG001
value
22
denoms
counts
group Id
OG000
value
49
group Id
OG001
value
45
units
Participants
title
Overall Response for Low-blast AML

denoms

counts
group Id
OG000
value
206
group Id
OG001
value
208
units
Participants

description

groups

description
Participants were administered azacitidine 75 mg/m\^2 IV or SC injection on Days 1 to 5, Days 8 and 9, in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles.
id
OG000
title
Azacitidine 75 mg/m^2
description
Participants were administered azacitidine 75 mg/m\^2 IV or SC injection on Days 1 to 5, Days 8 and 9 and pevonedistat 20 mg/m\^2 IV infusion, on Days 1, 3, and 5 in 28-day treatment cycles until disease progression or unacceptable toxicity or up to a maximum of 63 cycles.
id
OG001
title
Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2

param Type

COUNT_OF_PARTICIPANTS

population Description

REP included all participants who received at least 1 dose of study drug and had a Baseline and at least 1 post-baseline disease assessment. Number analyzed is the number of participants with data available for analysis for the specified category.

reporting Status

POSTED

time Frame

Up to Cycle 6 (up to approximately Day 168)

title

Number of Participants With Overall Response by Cycle 6

type

SECONDARY

unit Of Measure

Participants

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Preserved source evidence · Not medical advice