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NCT01953692 · OUTCOME

Objective Response Rate (ORR) in Cohort 1: Myelodysplastic Syndrome (MDS)

A Trial of Pembrolizumab (MK-3475) in Participants With Blood Cancers (MK-3475-013/KEYNOTE-013) · Source last updated 2021-08-04

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
90% Confidence Interval
Time frame
Up to approximately 78.5 months

What was measured

ORR was defined as the percentage of participants with response (complete response \[CR\] or partial response \[PR\]) according to the International Working Group (IWG) response criteria in myelodysplasia per Cheson et al. 2006. CR was demonstrated by ≤5% myeloblasts with normal maturation of all cell lines in the bone marrow (persistent dysplasia will be noted) and normal findings for hemoglobin, platelet count, neutrophil count, and absence of blasts in the blood. PR was all CR criteria if abnormal before treatment except bone marrow blasts decreased by ≥50% over pre-treatment but still \>5%. Cellularity and morphology are not relevant. Cohort 1 was evaluated statistically by comparing the ORR for pembrolizumab to a fixed efficacy target of 10% using a binomial exact test. The percentage of participants with CR and PR as assessed by the investigator is presented.

Analysis population: All participants in Cohort 1 with a baseline efficacy evaluation and at least 1 post-baseline efficacy evaluation.

Groups in this outcome

Cohort 1: Myelodysplastic Syndrome (MDS)

Participants received pembrolizumab 10 mg/kg by intravenous (IV) infusion on Day 1 of each 14-day cycle.

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

GroupSource count
Cohort 1: Myelodysplastic Syndrome (MDS)27

Reported measurements

Source class 1
Values in Percentage of participants · Interval/dispersion: 90% Confidence Interval
GroupValueSpreadLowerUpperComment
Cohort 1: Myelodysplastic Syndrome (MDS)0.0Not reported0.010.5Not reported

Source statistical analyses

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

  1. group Description
    Comparison to a fixed efficacy target of 10%. H0: p ≤ 0.10 versus H1: p \> 0.10
    group Ids
    1. OG000
    non Inferiority Type
    SUPERIORITY
    p Value
    >0.9999
    p Value Comment
    one-sided p value
    statistical Method
    exact binomial distribution
Complete source fields
analyses
  1. group Description
    Comparison to a fixed efficacy target of 10%. H0: p ≤ 0.10 versus H1: p \> 0.10
    group Ids
    1. OG000
    non Inferiority Type
    SUPERIORITY
    p Value
    >0.9999
    p Value Comment
    one-sided p value
    statistical Method
    exact binomial distribution
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        0.0
        upper Limit
        10.5
        value
        0.0
denoms
  1. counts
    1. group Id
      OG000
      value
      27
    units
    Participants
description
ORR was defined as the percentage of participants with response (complete response \[CR\] or partial response \[PR\]) according to the International Working Group (IWG) response criteria in myelodysplasia per Cheson et al. 2006. CR was demonstrated by ≤5% myeloblasts with normal maturation of all cell lines in the bone marrow (persistent dysplasia will be noted) and normal findings for hemoglobin, platelet count, neutrophil count, and absence of blasts in the blood. PR was all CR criteria if abnormal before treatment except bone marrow blasts decreased by ≥50% over pre-treatment but still \>5%. Cellularity and morphology are not relevant. Cohort 1 was evaluated statistically by comparing the ORR for pembrolizumab to a fixed efficacy target of 10% using a binomial exact test. The percentage of participants with CR and PR as assessed by the investigator is presented.
dispersion Type
90% Confidence Interval
groups
  1. description
    Participants received pembrolizumab 10 mg/kg by intravenous (IV) infusion on Day 1 of each 14-day cycle.
    id
    OG000
    title
    Cohort 1: Myelodysplastic Syndrome (MDS)
param Type
NUMBER
population Description
All participants in Cohort 1 with a baseline efficacy evaluation and at least 1 post-baseline efficacy evaluation.
reporting Status
POSTED
time Frame
Up to approximately 78.5 months
title
Objective Response Rate (ORR) in Cohort 1: Myelodysplastic Syndrome (MDS)
type
PRIMARY
unit Of Measure
Percentage of participants

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

Preserved source evidence · Not medical advice