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NCT02610777 · OUTCOME

Time to First CR or PR

An Efficacy and Safety Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML) and Low-Blast Acute Myelogenous Leukemia (AML) · Source last updated 2022-09-19

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 date of randomization until CR or PR (up to approximately 5 years)

What was measured

Time to first CR or PR: time from randomization to first documented CR or PR, whichever occurs first. Disease responses (HR MDS/CMML) based on modified IWG response criteria for MDS; low-blast AML on revised IWG response criteria for AML. HR MDS/CMML-CR: \<=5% myeloblasts with normal maturation of all bone marrow cell lines, \>=11 g/dL Hb, \>=100\*10\^9/L plt,\>=1.0\*10\^9/L ANC, 0% blasts in peripheral blood; 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/L ANC, plt \>=100\*10\^9/L, 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/TTP \<100\*10\^9/L; PR: all CR hematological values but with a decrease of \>=50% in the percentage of blasts to 5% to 25% in the 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.

Groups in this outcome

Azacitidine 75 mg/m^2

Azacitidine 75 mg/m\^2, infusion, intravenously or subcutaneously, on Day 1 through Day 5, Days 8 and 9 in 28-day treatment cycles until unacceptable toxicity, relapse, transformation to AML (for participants with HR MDS or CMML), or progressive disease (for participants with low-blast AML).

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

Azacitidine 75 mg/m\^2, infusion, intravenously or subcutaneously, on Day 1 through Day 5, Days 8 and 9 and pevonedistat 20 mg/m\^2, infusion, intravenously, on Days 1, 3, and 5 in 28-day treatment cycles until unacceptable toxicity, relapse, transformation to AML (for participants with HR MDS or CMML), or progressive disease (for participants with low-blast AML).

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^253
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^255

Reported measurements

Source class 1
Values in months · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Azacitidine 75 mg/m^213.2Not reported6.4NAThe upper limit of 95% CI was not estimable due to low number of participants with the event.
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^28.3Not reported4.5NAThe upper limit of 95% CI was not estimable due to low number of participants with the event.

Source statistical analyses

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

  1. ci Lower Limit
    0.699
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    2.081
    estimate Comment
    HR is based on an unstratified Cox proportional hazard regression model with treatment as a factor. HR\>1 for the treatment indicates a shorter time to first CR, CRi or PR in the Pevonedistat Combination arm compared to the Azacitidine only arm.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    =0.498
    p Value Comment
    P-value is from an unstratified log-rank test.
    param Type
    Hazard Ratio (HR)
    param Value
    1.206
    statistical Method
    Log Rank
Complete source fields
analyses
  1. ci Lower Limit
    0.699
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    2.081
    estimate Comment
    HR is based on an unstratified Cox proportional hazard regression model with treatment as a factor. HR\>1 for the treatment indicates a shorter time to first CR, CRi or PR in the Pevonedistat Combination arm compared to the Azacitidine only arm.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    =0.498
    p Value Comment
    P-value is from an unstratified log-rank test.
    param Type
    Hazard Ratio (HR)
    param Value
    1.206
    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 the event.
        group Id
        OG000
        lower Limit
        6.4
        upper Limit
        NA
        value
        13.2
      2. comment
        The upper limit of 95% CI was not estimable due to low number of participants with the event.
        group Id
        OG001
        lower Limit
        4.5
        upper Limit
        NA
        value
        8.3
denoms
  1. counts
    1. group Id
      OG000
      value
      53
    2. group Id
      OG001
      value
      55
    units
    Participants
description
Time to first CR or PR: time from randomization to first documented CR or PR, whichever occurs first. Disease responses (HR MDS/CMML) based on modified IWG response criteria for MDS; low-blast AML on revised IWG response criteria for AML. HR MDS/CMML-CR: \<=5% myeloblasts with normal maturation of all bone marrow cell lines, \>=11 g/dL Hb, \>=100\*10\^9/L plt,\>=1.0\*10\^9/L ANC, 0% blasts in peripheral blood; 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/L ANC, plt \>=100\*10\^9/L, 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/TTP \<100\*10\^9/L; PR: all CR hematological values but with a decrease of \>=50% in the percentage of blasts to 5% to 25% in the bone marrow aspirate.
dispersion Type
95% Confidence Interval
groups
  1. description
    Azacitidine 75 mg/m\^2, infusion, intravenously or subcutaneously, on Day 1 through Day 5, Days 8 and 9 in 28-day treatment cycles until unacceptable toxicity, relapse, transformation to AML (for participants with HR MDS or CMML), or progressive disease (for participants with low-blast AML).
    id
    OG000
    title
    Azacitidine 75 mg/m^2
  2. description
    Azacitidine 75 mg/m\^2, infusion, intravenously or subcutaneously, on Day 1 through Day 5, Days 8 and 9 and pevonedistat 20 mg/m\^2, infusion, intravenously, on Days 1, 3, and 5 in 28-day treatment cycles until unacceptable toxicity, relapse, transformation to AML (for participants with HR MDS or CMML), or progressive disease (for participants with low-blast AML).
    id
    OG001
    title
    Azacitidine 75 mg/m^2 + Pevonedistat 20 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.
reporting Status
POSTED
time Frame
From date of randomization until CR or PR (up to approximately 5 years)
title
Time to First CR or PR
type
SECONDARY
unit Of Measure
months

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Preserved source evidence · Not medical advice