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NCT02610777 · OUTCOME

Percentage of Participants With CR and PR by Cycle 4

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
NUMBER
Unit
percentage of participants
Interval / dispersion
Not reported
Time frame
From date of randomization until CR and PR, by Cycle 4 (cycle length=28 days)

What was measured

Disease responses for HR MDS/CMML per modified IWG response criteria for MDS and for low-blast AML on revised IWG response criteria for AML.CR for HR MDS/CMML: \<=5% myeloblasts with normal maturation of all cell lines in bone marrow, \>=11 g/dL Hb; \>=100\*10\^9/L plt; \>=1.0\*10\^9/L ANC and 0% blasts in peripheral blood.PR for HR MDS/CMML: considered achieved if all CR criteria is met except for bone marrow blasts decreased by\>=50% over pretreatment but still\>5%.CR for low-blast AML: morphologic leukemia-free state, ANC of\>1.0\*10\^9/L and plt of\>=100\*10\^9/L, transfusion independence, no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery for low-blast AML: fulfill all criteria for CR except for residual neutropenia (\<100\*10\^9/L)/TTP (\<100\*10\^9/L). PR for low-blast AML: all hematological values for CR but with decrease of \>=50% in percentage of blasts to 5%-25% in bone marrow aspirate. The percentages are rounded off to report the nearest whole numbers.

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

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^238
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^238

Reported measurements

Source class 1
Values in percentage of participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Azacitidine 75 mg/m^221.1Not reportedNot reportedNot reportedNot reported
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^231.6Not 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
    -9.13
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    30.18
    estimate Comment
    Absolute difference \>0 represent better response achieved on pevonedistat combination compared to azacitidine treatment alone.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    =0.301
    p Value Comment
    P-value is from an unstratified Cochran-Mantel Haenszel chi-square test.
    param Type
    Absolute Rate Difference
    param Value
    10.53
    statistical Method
    Cochran-Mantel-Haenszel
Complete source fields
analyses
  1. ci Lower Limit
    -9.13
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    30.18
    estimate Comment
    Absolute difference \>0 represent better response achieved on pevonedistat combination compared to azacitidine treatment alone.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    =0.301
    p Value Comment
    P-value is from an unstratified Cochran-Mantel Haenszel chi-square test.
    param Type
    Absolute Rate Difference
    param Value
    10.53
    statistical Method
    Cochran-Mantel-Haenszel
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        value
        21.1
      2. group Id
        OG001
        value
        31.6
denoms
  1. counts
    1. group Id
      OG000
      value
      38
    2. group Id
      OG001
      value
      38
    units
    Participants
description
Disease responses for HR MDS/CMML per modified IWG response criteria for MDS and for low-blast AML on revised IWG response criteria for AML.CR for HR MDS/CMML: \<=5% myeloblasts with normal maturation of all cell lines in bone marrow, \>=11 g/dL Hb; \>=100\*10\^9/L plt; \>=1.0\*10\^9/L ANC and 0% blasts in peripheral blood.PR for HR MDS/CMML: considered achieved if all CR criteria is met except for bone marrow blasts decreased by\>=50% over pretreatment but still\>5%.CR for low-blast AML: morphologic leukemia-free state, ANC of\>1.0\*10\^9/L and plt of\>=100\*10\^9/L, transfusion independence, no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery for low-blast AML: fulfill all criteria for CR except for residual neutropenia (\<100\*10\^9/L)/TTP (\<100\*10\^9/L). PR for low-blast AML: all hematological values for CR but with decrease of \>=50% in percentage of blasts to 5%-25% in bone marrow aspirate. The percentages are rounded off to report the nearest whole numbers.
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
NUMBER
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 date of randomization until CR and PR, by Cycle 4 (cycle length=28 days)
title
Percentage of Participants With CR and PR by Cycle 4
type
SECONDARY
unit Of Measure
percentage of participants

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Preserved source evidence · Not medical advice