NCT02610777 · OUTCOME

Time to Progressive Disease (PD), Relapse, or Death

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: Full Range
Time frame: From date of randomization until PD, relapse or death (up to approximately 5 years)

What was measured

Time from randomization until PD/transformation to AML/relapse/death due to any cause, whichever occurs first. Relapse after CR or PR in MDS/CMML: return to pretreatment bone marrow blast % or decrement of \>=50% from maximum remission levels in ANC or plt, reduction in Hb concentration by \>=1.5 g/dL or transfusion dependence. PD: at least 50% decrement from maximum remission in ANC or plt, or reduction in Hb by\>=2g/dL or transfusion dependence;participants with \<5% blasts: \>=50% increase (inc) in blasts to \>5%; 5%-10%: \>=50% inc to \>10%; 10%-20%: \>=50% inc to\>20%;20%-30%:\>=50% inc to \>30%. Relapse after CR in Low blast AML: reappearance of leukemic blasts in peripheral blood or \>=5% blasts in bone marrow not attributable to any cause (example, bone marrow regeneration after consolidation therapy). If there are no circulating blasts, bone marrow contains 5%-20% blasts, a repeat analysis is performed a week later.

Analysis population: ITT Population was defined as all participants who were randomized.

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

Group	Source count
Azacitidine 75 mg/m^2	62
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2	58

Reported measurements

Source class 1

Values in months · Interval/dispersion: Full Range
Group	Value	Spread	Lower	Upper	Comment
Azacitidine 75 mg/m^2	13.6	Not reported	9.40	16.53	Not reported
Azacitidine 75 mg/m^2 + Pevonedistat 20 mg/m^2	15.2	Not reported	12.39	20.83	Not reported

Source statistical analyses

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

ci Lower Limit
0.521
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
1.198
estimate Comment
HR is based on an unstratified Cox proportional hazard regression model with treatment as a factor. HR\<1for the treatment indicates a longer time to PD, Relapse, or Death in the Pevonedistat Combination arm compared to the Azacitidine only arm.
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
=0.266
p Value Comment
P-value is from an unstratified log-rank test.
param Type
Hazard Ratio (HR)
param Value
0.790
statistical Method
Log Rank

Complete source fields

analyses

ci Lower Limit
0.521
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
1.198
estimate Comment
HR is based on an unstratified Cox proportional hazard regression model with treatment as a factor. HR\<1for the treatment indicates a longer time to PD, Relapse, or Death in the Pevonedistat Combination arm compared to the Azacitidine only arm.
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
=0.266
p Value Comment
P-value is from an unstratified log-rank test.
param Type
Hazard Ratio (HR)
param Value
0.790
statistical Method
Log Rank

classes

categories
measurements
group Id
OG000
lower Limit
9.40
upper Limit
16.53
value
13.6
group Id
OG001
lower Limit
12.39
upper Limit
20.83
value
15.2

denoms

counts
group Id
OG000
value
62
group Id
OG001
value
58
units
Participants

description

dispersion Type

Full Range

groups

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
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

ITT Population was defined as all participants who were randomized.

reporting Status

POSTED

time Frame

From date of randomization until PD, relapse or death (up to approximately 5 years)

title

Time to Progressive Disease (PD), Relapse, or Death

type

SECONDARY

unit Of Measure

months

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Preserved source evidence · Not medical advice