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NCT02604433 · OUTCOME

Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia · Source last updated 2023-04-18

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
Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48

What was measured

Percentage of participants who achieved a red blood cell (RBC) transfusion burden reduction from baseline ≥ 50% with a reduction of at least 2 units during Week 37 to Week 48 compared to the 12-week interval on or prior to Dose 1 Day 1.

Analysis population: All randomized participants

Groups in this outcome

Luspatercept + BSC

Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care

Placebo + BSC

Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care

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

Analysis denominator · Participants

GroupSource count
Luspatercept + BSC224
Placebo + BSC112

Reported measurements

Source class 1
Values in Percentage of participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Luspatercept + BSC10.3Not reportedNot reportedNot reportedNot reported
Placebo + BSC0.9Not 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
    1.65
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    86.29
    group Description
    Odds ratio, 95% CIs, and p-value were estimated from the Cochran-Mantel-Haenszel (CMH) test stratified by the geographical regions defined at randomization. To control the overall Type 1 error rate, the testing procedure was done strictly in order: the test for this outcome was only conducted when there was evidence showing erythroid response was achieved in the luspatercept group for the primary endpoint, and achievement of objective in the luspatercept group in outcomes 2+3.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.0017
    p Value Comment
    Significance level of 0.050 for 2-sided tests.
    param Type
    Odds Ratio (OR)
    param Value
    11.92
    statistical Method
    Cochran-Mantel-Haenszel
Complete source fields
analyses
  1. ci Lower Limit
    1.65
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    86.29
    group Description
    Odds ratio, 95% CIs, and p-value were estimated from the Cochran-Mantel-Haenszel (CMH) test stratified by the geographical regions defined at randomization. To control the overall Type 1 error rate, the testing procedure was done strictly in order: the test for this outcome was only conducted when there was evidence showing erythroid response was achieved in the luspatercept group for the primary endpoint, and achievement of objective in the luspatercept group in outcomes 2+3.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY
    p Value
    0.0017
    p Value Comment
    Significance level of 0.050 for 2-sided tests.
    param Type
    Odds Ratio (OR)
    param Value
    11.92
    statistical Method
    Cochran-Mantel-Haenszel
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        value
        10.3
      2. group Id
        OG001
        value
        0.9
denoms
  1. counts
    1. group Id
      OG000
      value
      224
    2. group Id
      OG001
      value
      112
    units
    Participants
description
Percentage of participants who achieved a red blood cell (RBC) transfusion burden reduction from baseline ≥ 50% with a reduction of at least 2 units during Week 37 to Week 48 compared to the 12-week interval on or prior to Dose 1 Day 1.
groups
  1. description
    Luspatercept (ACE-536) was administered subcutaneously (SC) at a starting dose level of 1 mg/kg once every 21 days. Participants could be dose-titrated to 1.25 mg/kg, but the maximum total dose should not exceed 120 mg, total dose per administration during the Treatment Period, the Long-term Treatment Period, and Open-Label Treatment Period. BSC = Best Supportive Care
    id
    OG000
    title
    Luspatercept + BSC
  2. description
    Placebo (normal saline) was administered subcutaneously (SC) in volumes to match active treatment once every 21 days. BSC = Best Supportive Care
    id
    OG001
    title
    Placebo + BSC
param Type
NUMBER
population Description
All randomized participants
reporting Status
POSTED
time Frame
Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48
title
Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48
type
SECONDARY
unit Of Measure
Percentage of participants

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Preserved source evidence · Not medical advice