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NCT03268954 · OUTCOME

Number of Participants With Hematologic Improvement (HI)

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 HI (up to approximately 42 months)

What was measured

Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS. HI: Hgb increase \>=1.5 g/dL if baseline \<11 g/dL; pl increase \>=30\*10\^9/L if baseline \>20\*10\^9/L or increase from \<20\*10\^9/L to \>20\*10\^9/L by at least 100%; neutrophil increase by 100% and absolute increase of \>0.5\*10\^9/L if baseline \<1.0\*10\^9/L.

Analysis population: REP included all participants who received at least 1 dose of study drug and had a baseline and at least 1 postbaseline disease assessment. Overall number analyzed is the number of participants with data available for analyses.

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

GroupSource count
Azacitidine 75 mg/m^2157
Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2163

Reported measurements

Source class 1
Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Azacitidine 75 mg/m^276Not reportedNot reportedNot reportedNot reported
Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^270Not reportedNot reportedNot reportedNot reported

Source statistical analyses

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

  1. ci Lower Limit
    -16.36
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    5.44
    group Description
    Number of Participants With HI
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    =0.320
    p Value Comment
    P-value was obtained from a stratified Cochran-Mantel-Haenszel chi-square test.
    param Type
    Absolute Rate Difference
    param Value
    -5.46
    statistical Method
    Cochran-Mantel-Haenszel
Complete source fields
analyses
  1. ci Lower Limit
    -16.36
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    5.44
    group Description
    Number of Participants With HI
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    =0.320
    p Value Comment
    P-value was obtained from a stratified Cochran-Mantel-Haenszel chi-square test.
    param Type
    Absolute Rate Difference
    param Value
    -5.46
    statistical Method
    Cochran-Mantel-Haenszel
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        value
        76
      2. group Id
        OG001
        value
        70
denoms
  1. counts
    1. group Id
      OG000
      value
      157
    2. group Id
      OG001
      value
      163
    units
    Participants
description
Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS. HI: Hgb increase \>=1.5 g/dL if baseline \<11 g/dL; pl increase \>=30\*10\^9/L if baseline \>20\*10\^9/L or increase from \<20\*10\^9/L to \>20\*10\^9/L by at least 100%; neutrophil increase by 100% and absolute increase of \>0.5\*10\^9/L if baseline \<1.0\*10\^9/L.
groups
  1. 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
  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 postbaseline disease assessment. Overall number analyzed is the number of participants with data available for analyses.
reporting Status
POSTED
time Frame
From randomization until HI (up to approximately 42 months)
title
Number of Participants With Hematologic Improvement (HI)
type
SECONDARY
unit Of Measure
Participants

Download exact source JSON → · Snapshot ctgov-results-25a395029948b04d52b1253f

Preserved source evidence · Not medical advice