Skip to content
Triangle logo TriangleGet started
← Full study record

NCT02631070 · OUTCOME

Percentage Of Participants Who Achieved Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 Weeks From Week 1 to Week 24

A Study of Luspatercept (ACE-536) to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes · Source last updated 2021-12-17

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure
NUMBER
Unit
Percent of Participants
Interval / dispersion
95% Confidence Interval
Time frame
From Week 1 through Week 24 of study treatment

What was measured

RBC-TI response was defined as the absence of any Red Blood Cells (RBC) transfusion during any consecutive 56-day (8-week) period (ie, Days 1 to 56, Days 2 to 57, Days 3 to 58, etc.) during the first 24 weeks of study treatment. Participants had to have at least 56 days (≥ 8 weeks) of transfusion independence prior to (and including) the Week 24 cut-off date to qualify as a responder. Participants who failed to achieve RBC-TI at least 56 days prior to or on the cut-off date were counted as non-responders.

Analysis population: All treated participants

Groups in this outcome

Luspatercept

Luspatercept 1.0 mg/Kg SC on Day 1 of each 21-day treatment cycle

Placebo

Placebo (Volume equivalent to experimental arm) SC on Day 1 of each 21-day treatment 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
Luspatercept153
Placebo76

Reported measurements

Source class 1
Values in Percent of Participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Luspatercept37.91Not reported30.2046.10Not reported
Placebo13.16Not reported6.4922.87Not reported

Source statistical analyses

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

  1. ci Lower Limit
    14.48
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    34.64
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    <0.0001
    param Type
    Common Risk Difference on Response Rate
    param Value
    24.56
    statistical Method
    Cochran-Mantel-Haenszel
  2. ci Lower Limit
    2.278
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    11.259
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    <0.0001
    param Type
    Odds Ratio (OR)
    param Value
    5.065
    statistical Method
    Cochran-Mantel-Haenszel
Complete source fields
analyses
  1. ci Lower Limit
    14.48
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    34.64
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    <0.0001
    param Type
    Common Risk Difference on Response Rate
    param Value
    24.56
    statistical Method
    Cochran-Mantel-Haenszel
  2. ci Lower Limit
    2.278
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    11.259
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    <0.0001
    param Type
    Odds Ratio (OR)
    param Value
    5.065
    statistical Method
    Cochran-Mantel-Haenszel
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        30.20
        upper Limit
        46.10
        value
        37.91
      2. group Id
        OG001
        lower Limit
        6.49
        upper Limit
        22.87
        value
        13.16
denoms
  1. counts
    1. group Id
      OG000
      value
      153
    2. group Id
      OG001
      value
      76
    units
    Participants
description
RBC-TI response was defined as the absence of any Red Blood Cells (RBC) transfusion during any consecutive 56-day (8-week) period (ie, Days 1 to 56, Days 2 to 57, Days 3 to 58, etc.) during the first 24 weeks of study treatment. Participants had to have at least 56 days (≥ 8 weeks) of transfusion independence prior to (and including) the Week 24 cut-off date to qualify as a responder. Participants who failed to achieve RBC-TI at least 56 days prior to or on the cut-off date were counted as non-responders.
dispersion Type
95% Confidence Interval
groups
  1. description
    Luspatercept 1.0 mg/Kg SC on Day 1 of each 21-day treatment cycle
    id
    OG000
    title
    Luspatercept
  2. description
    Placebo (Volume equivalent to experimental arm) SC on Day 1 of each 21-day treatment cycle
    id
    OG001
    title
    Placebo
param Type
NUMBER
population Description
All treated participants
reporting Status
POSTED
time Frame
From Week 1 through Week 24 of study treatment
title
Percentage Of Participants Who Achieved Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 Weeks From Week 1 to Week 24
type
PRIMARY
unit Of Measure
Percent of Participants

Download exact source JSON → · Snapshot ctgov-results-191f4516d4760045304b24a0

Preserved source evidence · Not medical advice