NCT03268954 · OUTCOME

Number of Participants With Overall Response 2 (OR2)

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: From randomization until, CR, PR or HI or CR, CRi or PR (up to approximately 42 months)

What was measured

OR2=participant with best overall response of CR+PR+HI in HR MDS/CMML participants,or of CR+CRi+PR in low-blast AML participants.CR:≤5% myeloblasts with normal maturation of all bone marrow(BM)cell lines,≥11g/dL Hgb,≥100\*10\^9/L pl,≥1.0\*10\^9/L neutrophils,0% blasts in peripheral blood;PR:all CR criteria met except BM blasts ≥50% decrease over pretreatment but still \>5%;HI:Hgb increase(inc) ≥1.5g/dL if baseline(BL)\<11 g/dL;pl inc≥30\*10\^9/L if BL\>20\*10\^9/L or inc from \<20\*10\^9/L to \>20\*10\^9/L and by 100%;neutrophil inc by 100%;absolute inc of \>0.5\*10\^9/L if BL\<100\*10\^9/L.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;CR with incomplete blood count recovery:fulfill CR criteria except residual neutropenia \<1.0\*10\^9/L or pl\<100\*10\^9/L;PR:all CR hematological values but ≥50% decrease in BM aspirate.Number of responders determined by independent review committee(IRC) assessment.

Analysis population: REP: all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. Data is reported for responders. Three participants were not counted as responders for OR2 as the IRC assessed these participants as non-responders.

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

Source class 1

Values in Participants · Interval/dispersion: Not reported
Group	Value	Spread	Lower	Upper	Comment
Azacitidine 75 mg/m^2	94	Not reported	Not reported	Not reported	Not reported
Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2	94	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
-10.03
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
9.15
group Description
Number of Participants with OR2
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
=0.891
p Value Comment
P-value was obtained from a stratified Cochran-Mantel-Haenszel chi-square test stratified by low-blast AML, IPSS-R risk groups of very high, high, or intermediate for HR MDS/CMML.
param Type
Absolute Rate Difference
param Value
-0.44
statistical Method
Cochran-Mantel-Haenszel

Complete source fields

analyses

ci Lower Limit
-10.03
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
9.15
group Description
Number of Participants with OR2
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
=0.891
p Value Comment
P-value was obtained from a stratified Cochran-Mantel-Haenszel chi-square test stratified by low-blast AML, IPSS-R risk groups of very high, high, or intermediate for HR MDS/CMML.
param Type
Absolute Rate Difference
param Value
-0.44
statistical Method
Cochran-Mantel-Haenszel

classes

categories
measurements
group Id
OG000
value
94
group Id
OG001
value
94

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: all participants who received at least 1 dose of study drug and had a Baseline and at least 1 postbaseline disease assessment. Data is reported for responders. Three participants were not counted as responders for OR2 as the IRC assessed these participants as non-responders.

reporting Status

POSTED

time Frame

From randomization until, CR, PR or HI or CR, CRi or PR (up to approximately 42 months)

title

Number of Participants With Overall Response 2 (OR2)

type

SECONDARY

unit Of Measure

Participants

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Preserved source evidence · Not medical advice