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NCT03682536 · OUTCOME

Percentage of Participants With Red Blood Cell Transfusion Independence (RBC-TI) for 12 Weeks (84 Days) With a Mean Hemoglobin Increase ≥ 1.5 g/dL

A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve · Source last updated 2024-11-20

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
Week 1 through Week 24

What was measured

Percentage of participants who are RBC transfusion-free for any 12-week period associated with a concurrent mean hemoglobin (Hgb) increase ≥ 1.5 g/dL compared to baseline. After applying below 14/3-day rule, the baseline Hgb value is defined as the lowest Hgb value from the central, local laboratory, or pre transfusion Hgb from transfusion records that is within 56 days on or prior to the first dose of treatment, or randomization date if participants were not treated. 4/3-day rule: only Hgb values that are at least 14 days after a transfusion may be used unless there is another transfusion within 3 days after the Hgb assessment. If this occurs, that Hgb value will be used despite being \< 14 days after the previous transfusion.

Analysis population: ITT Population - all randomized participants regardless of whether or not the participant received treatment.

Groups in this outcome

Luspatercept

Starting dose of 1.0 mg/kg subcutaneous injection every 3 weeks (21 days; Q3W). Dose levels can be increased in a stepwise manner beyond the starting dose to 1.33 mg/kg, and up to a maximum of 1.75 mg/kg.

Epoetin Alfa

Starting dose of 450 IU/kg (maximum total starting dose is 40,000 IU) subcutaneous injection once every week (7 days; QW). Dose levels can be increased in a stepwise manner beyond the starting dose to 787.5 IU/kg, and up to a maximum of 1,050 IU/kg (with a maximum total dose of 80,000 IU).

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

Analysis denominator · Participants

GroupSource count
Luspatercept182
Epoetin Alfa181

Reported measurements

Source class 1
Values in Percent of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Luspatercept60.4Not reported52.967.6Not reported
Epoetin Alfa34.8Not reported27.942.2Not reported

Source statistical analyses

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

  1. ci Lower Limit
    2.0
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    4.8
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    <.0001
    param Type
    Odds Ratio (OR)
    param Value
    3.1
    statistical Method
    Cochran-Mantel-Haenszel
Complete source fields
analyses
  1. ci Lower Limit
    2.0
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    4.8
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    <.0001
    param Type
    Odds Ratio (OR)
    param Value
    3.1
    statistical Method
    Cochran-Mantel-Haenszel
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        52.9
        upper Limit
        67.6
        value
        60.4
      2. group Id
        OG001
        lower Limit
        27.9
        upper Limit
        42.2
        value
        34.8
denoms
  1. counts
    1. group Id
      OG000
      value
      182
    2. group Id
      OG001
      value
      181
    units
    Participants
description
Percentage of participants who are RBC transfusion-free for any 12-week period associated with a concurrent mean hemoglobin (Hgb) increase ≥ 1.5 g/dL compared to baseline. After applying below 14/3-day rule, the baseline Hgb value is defined as the lowest Hgb value from the central, local laboratory, or pre transfusion Hgb from transfusion records that is within 56 days on or prior to the first dose of treatment, or randomization date if participants were not treated. 4/3-day rule: only Hgb values that are at least 14 days after a transfusion may be used unless there is another transfusion within 3 days after the Hgb assessment. If this occurs, that Hgb value will be used despite being \< 14 days after the previous transfusion.
dispersion Type
95% Confidence Interval
groups
  1. description
    Starting dose of 1.0 mg/kg subcutaneous injection every 3 weeks (21 days; Q3W). Dose levels can be increased in a stepwise manner beyond the starting dose to 1.33 mg/kg, and up to a maximum of 1.75 mg/kg.
    id
    OG000
    title
    Luspatercept
  2. description
    Starting dose of 450 IU/kg (maximum total starting dose is 40,000 IU) subcutaneous injection once every week (7 days; QW). Dose levels can be increased in a stepwise manner beyond the starting dose to 787.5 IU/kg, and up to a maximum of 1,050 IU/kg (with a maximum total dose of 80,000 IU).
    id
    OG001
    title
    Epoetin Alfa
param Type
NUMBER
population Description
ITT Population - all randomized participants regardless of whether or not the participant received treatment.
reporting Status
POSTED
time Frame
Week 1 through Week 24
title
Percentage of Participants With Red Blood Cell Transfusion Independence (RBC-TI) for 12 Weeks (84 Days) With a Mean Hemoglobin Increase ≥ 1.5 g/dL
type
PRIMARY
unit Of Measure
Percent of participants

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

Preserved source evidence · Not medical advice