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NCT03268954 · OUTCOME

Duration of Complete Remission (CR)

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
MEDIAN
Unit
months
Interval / dispersion
95% Confidence Interval
Time frame
From CR until first documentation of PD or relapse from CR or relapse after CR or PR (up to approximately 42 months)

What was measured

Duration of CR is first documented CR to the first documentation of PD or relapse from CR (participants with low-blast AML) or relapse after CR or PR (participants with HR MDS/CMML). Disease responses for HR MDS or CMML are based on the Modified IWG Response Criteria for MDS and for low-blast AML on the Revised IWG Response Criteria for AML. CR for HR MDS or CMML is defined as \<=5% myeloblasts with normal maturation of all cell lines in the bone marrow, and greater than or equal to \>=11 g/dL Hgb,\>=100\*10\^9/L pl,\>=1.0\*10\^9/L neutrophils and 0% blasts in peripheral blood. CR for low-blast AML: morphologic leukemia-free state, neutrophils of more than 1.0\*10\^9/L and pl of \>=100\*10\^9/L, transfusion independence, and no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery (CRi) for low-blast AML: participants fulfill all of the criteria for CR except for residual neutropenia (\<1.0\*10\^9/L) or thrombocytopenia (pl\<100\*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. Data is reported for participants with complete remission.

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^268
Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^257

Reported measurements

Source class 1
Values in months · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Azacitidine 75 mg/m^213.1Not reported6.9329.08Not reported
Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^217.2Not reported13.96NAThe upper limit of 95% CI was not estimable due to low number of participants with event.

Source statistical analyses

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

  1. ci Lower Limit
    0.402
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    1.146
    estimate Comment
    Hazard ratio was based on an unadjusted stratified Cox proportional hazard regression model with stratification factors (low-blast AML, IPSS-R risk groups of very high, high, or intermediate for HR MDS/CMML) and treatment as a factor in the model.
    group Description
    Duration of CR
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    =0.072
    param Type
    Hazard Ratio (HR)
    param Value
    0.679
    statistical Comment
    P-value was based on 1-sided log-rank test stratified by low-blast AML,IPSS-R risk groups of very high,high,or intermediate for HR MDS/CMML.
    statistical Method
    Log Rank
Complete source fields
analyses
  1. ci Lower Limit
    0.402
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    1.146
    estimate Comment
    Hazard ratio was based on an unadjusted stratified Cox proportional hazard regression model with stratification factors (low-blast AML, IPSS-R risk groups of very high, high, or intermediate for HR MDS/CMML) and treatment as a factor in the model.
    group Description
    Duration of CR
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    =0.072
    param Type
    Hazard Ratio (HR)
    param Value
    0.679
    statistical Comment
    P-value was based on 1-sided log-rank test stratified by low-blast AML,IPSS-R risk groups of very high,high,or intermediate for HR MDS/CMML.
    statistical Method
    Log Rank
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        6.93
        upper Limit
        29.08
        value
        13.1
      2. comment
        The upper limit of 95% CI was not estimable due to low number of participants with event.
        group Id
        OG001
        lower Limit
        13.96
        upper Limit
        NA
        value
        17.2
denoms
  1. counts
    1. group Id
      OG000
      value
      68
    2. group Id
      OG001
      value
      57
    units
    Participants
description
Duration of CR is first documented CR to the first documentation of PD or relapse from CR (participants with low-blast AML) or relapse after CR or PR (participants with HR MDS/CMML). Disease responses for HR MDS or CMML are based on the Modified IWG Response Criteria for MDS and for low-blast AML on the Revised IWG Response Criteria for AML. CR for HR MDS or CMML is defined as \<=5% myeloblasts with normal maturation of all cell lines in the bone marrow, and greater than or equal to \>=11 g/dL Hgb,\>=100\*10\^9/L pl,\>=1.0\*10\^9/L neutrophils and 0% blasts in peripheral blood. CR for low-blast AML: morphologic leukemia-free state, neutrophils of more than 1.0\*10\^9/L and pl of \>=100\*10\^9/L, transfusion independence, and no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery (CRi) for low-blast AML: participants fulfill all of the criteria for CR except for residual neutropenia (\<1.0\*10\^9/L) or thrombocytopenia (pl\<100\*10\^9/L).
dispersion Type
95% Confidence Interval
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
MEDIAN
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. Data is reported for participants with complete remission.
reporting Status
POSTED
time Frame
From CR until first documentation of PD or relapse from CR or relapse after CR or PR (up to approximately 42 months)
title
Duration of Complete Remission (CR)
type
SECONDARY
unit Of Measure
months

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Preserved source evidence · Not medical advice