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NCT02610777 · OUTCOME

Percentage of Participants With Complete Remission (CR)

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 (up to approximately 5 years)

What was measured

Disease responses for HR MDS or CMML is based on the modified International Working Group (IWG) response criteria for MDS and for low-blast AML on the revised IWG response criteria for AML. CR for HR MDS or CMML: \<= 5% myeloblasts with normal maturation of all cell lines in the bone marrow, and \>= 11 gram/deciliter (g/dL) hemoglobin (Hb), \>=100\*10\^9/liter (/L) platelets (plt), \>=1.0\*10\^9/L absolute neutrophil count (ANC) and 0% blasts in peripheral blood. CR for low-blast AML: morphologic leukemia-free state, ANC of more than 1.0\*10\^9/L and plt of \>=1.0\*10\^9/L, transfusion independence, and no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery for low-blast AML: some participants fulfill all of the criteria for CR except for residual neutropenia (\<1.0\*10\^9/L) or thrombocytopenia (TTP) (\<100\*10\^9/L). The percentages are rounded off to report the nearest whole numbers.

Analysis population: Response-Evaluable 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 percentage of participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Azacitidine 75 mg/m^236Not reportedNot reportedNot reportedNot reported
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^245Not 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
    -8.83
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    28.04
    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
    =0.312
    p Value Comment
    P-value is from an unstratified Cochran-Mantel Haenszel chi-square test.
    param Type
    Absolute Rate Difference
    param Value
    9.61
    statistical Method
    Cochran-Mantel-Haenszel
Complete source fields
analyses
  1. ci Lower Limit
    -8.83
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    28.04
    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
    =0.312
    p Value Comment
    P-value is from an unstratified Cochran-Mantel Haenszel chi-square test.
    param Type
    Absolute Rate Difference
    param Value
    9.61
    statistical Method
    Cochran-Mantel-Haenszel
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        value
        36
      2. group Id
        OG001
        value
        45
denoms
  1. counts
    1. group Id
      OG000
      value
      53
    2. group Id
      OG001
      value
      55
    units
    Participants
description
Disease responses for HR MDS or CMML is based on the modified International Working Group (IWG) response criteria for MDS and for low-blast AML on the revised IWG response criteria for AML. CR for HR MDS or CMML: \<= 5% myeloblasts with normal maturation of all cell lines in the bone marrow, and \>= 11 gram/deciliter (g/dL) hemoglobin (Hb), \>=100\*10\^9/liter (/L) platelets (plt), \>=1.0\*10\^9/L absolute neutrophil count (ANC) and 0% blasts in peripheral blood. CR for low-blast AML: morphologic leukemia-free state, ANC of more than 1.0\*10\^9/L and plt of \>=1.0\*10\^9/L, transfusion independence, and no residual evidence of extramedullary leukemia. CR with incomplete blood count recovery for low-blast AML: some participants fulfill all of the criteria for CR except for residual neutropenia (\<1.0\*10\^9/L) or thrombocytopenia (TTP) (\<100\*10\^9/L). 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
Response-Evaluable 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.
reporting Status
POSTED
time Frame
From date of randomization until CR (up to approximately 5 years)
title
Percentage of Participants With Complete Remission (CR)
type
SECONDARY
unit Of Measure
percentage of participants

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

Preserved source evidence · Not medical advice