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NCT03268954 · OUTCOME

Duration of Overall Response (OR)

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
Up to approximately 42 months

What was measured

Duration of OR: response to first documentation of PD or relapse from CR for low-blast AML or relapse after CR or PR for HR MDS/CMML. Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS; for low-blast AML on Revised IWG Response Criteria for AML. Overall response=CR+PR for HR MDS/CMML and CR+Cri+ PR for low-blast AML.CR for HR MDS/CMML:\<=5% myeloblasts with normal maturation of all bone marrow cell lines,\>=11 g/dL Hgb,\>=100\*10\^9/L 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; CR with CRi: 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 % of blasts 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 postbaseline disease assessment. Data is reported for 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

GroupSource count
Azacitidine 75 mg/m^273
Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^264

Reported measurements

Source class 1
Values in months · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Azacitidine 75 mg/m^218.3Not reported8.31NAThe upper limit of 95% CI was not estimable due to low number of participants with event.
Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^217.1Not reported12.68NAThe 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
    0.542
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    1.458
    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 Overall Response (OR)
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    =0.320
    p Value Comment
    P-value comparing duration of PR or better response between treatment groups 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.
    param Type
    Hazard Ratio (HR)
    param Value
    0.889
    statistical Method
    Log Rank
Complete source fields
analyses
  1. ci Lower Limit
    0.542
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    1.458
    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 Overall Response (OR)
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    =0.320
    p Value Comment
    P-value comparing duration of PR or better response between treatment groups 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.
    param Type
    Hazard Ratio (HR)
    param Value
    0.889
    statistical Method
    Log Rank
classes
  1. categories
    1. measurements
      1. comment
        The upper limit of 95% CI was not estimable due to low number of participants with event.
        group Id
        OG000
        lower Limit
        8.31
        upper Limit
        NA
        value
        18.3
      2. comment
        The upper limit of 95% CI was not estimable due to low number of participants with event.
        group Id
        OG001
        lower Limit
        12.68
        upper Limit
        NA
        value
        17.1
denoms
  1. counts
    1. group Id
      OG000
      value
      73
    2. group Id
      OG001
      value
      64
    units
    Participants
description
Duration of OR: response to first documentation of PD or relapse from CR for low-blast AML or relapse after CR or PR for HR MDS/CMML. Disease responses for HR MDS/CMML based on Modified IWG Response Criteria for MDS; for low-blast AML on Revised IWG Response Criteria for AML. Overall response=CR+PR for HR MDS/CMML and CR+Cri+ PR for low-blast AML.CR for HR MDS/CMML:\<=5% myeloblasts with normal maturation of all bone marrow cell lines,\>=11 g/dL Hgb,\>=100\*10\^9/L 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; CR with CRi: 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 % of blasts in bone marrow aspirate.
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 responders.
reporting Status
POSTED
time Frame
Up to approximately 42 months
title
Duration of Overall Response (OR)
type
SECONDARY
unit Of Measure
months

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Preserved source evidence · Not medical advice