NCT02604433 · OUTCOME

Time to Erythroid Response

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: MEAN
Unit: Days
Interval / dispersion: Standard Deviation
Time frame: From first dose to 48 weeks following first dose

What was measured

Time to erythroid response was defined as the time from first dose of the study drug to first erythroid response. This is reported for participants with a ≥ 33% reduction from baseline in RBC transfusion burden (with a reduction of at least 2 units) for any 12-week interval., as well as participants with a ≥ 50% reduction from baseline in RBC transfusion burden (with a reduction of at least 2 units) for any 12-week interval.

Analysis population: All randomized participants who achieved erythroid response

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

Group	Source count
Luspatercept + BSC	173
Placebo + BSC	39

Reported measurements

≥ 33% Transfusion Burden Reduction

Analysis denominator · Participants

Group	Source count
Luspatercept + BSC	173
Placebo + BSC	39

Values in Days · Interval/dispersion: Standard Deviation
Group	Value	Spread	Lower	Upper	Comment
Luspatercept + BSC	96.3	163.92	Not reported	Not reported	Not reported
Placebo + BSC	163.5	148.78	Not reported	Not reported	Not reported

≥ 50% Transfusion Burden Reduction

Analysis denominator · Participants

Group	Source count
Luspatercept + BSC	112
Placebo + BSC	9

Values in Days · Interval/dispersion: Standard Deviation
Group	Value	Spread	Lower	Upper	Comment
Luspatercept + BSC	189.1	257.69	Not reported	Not reported	Not reported
Placebo + BSC	160.9	160.17	Not reported	Not reported	Not reported

Source statistical analyses

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

ci Lower Limit
-123.63
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
-10.91
estimate Comment
luspatercept - placebo
group Description
≥ 33% Transfusion Burden Reduction
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
0.0195
p Value Comment
Significance level of 0.050 for 2-sided tests.
param Type
Mean Difference (Final Values)
param Value
-67.27
statistical Method
t-test, 2 sided
ci Lower Limit
-144.87
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
201.34
estimate Comment
luspatercept - placebo
group Description
≥ 50% Transfusion Burden Reduction
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
0.7473
p Value Comment
Significance level of 0.050 for 2-sided tests.
param Type
Mean Difference (Final Values)
param Value
28.24
statistical Method
t-test, 2 sided

Complete source fields

analyses

ci Lower Limit
-123.63
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
-10.91
estimate Comment
luspatercept - placebo
group Description
≥ 33% Transfusion Burden Reduction
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
0.0195
p Value Comment
Significance level of 0.050 for 2-sided tests.
param Type
Mean Difference (Final Values)
param Value
-67.27
statistical Method
t-test, 2 sided
ci Lower Limit
-144.87
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
201.34
estimate Comment
luspatercept - placebo
group Description
≥ 50% Transfusion Burden Reduction
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY
p Value
0.7473
p Value Comment
Significance level of 0.050 for 2-sided tests.
param Type
Mean Difference (Final Values)
param Value
28.24
statistical Method
t-test, 2 sided

classes

categories
measurements
group Id
OG000
spread
163.92
value
96.3
group Id
OG001
spread
148.78
value
163.5
denoms
counts
group Id
OG000
value
173
group Id
OG001
value
39
units
Participants
title
≥ 33% Transfusion Burden Reduction
categories
measurements
group Id
OG000
spread
257.69
value
189.1
group Id
OG001
spread
160.17
value
160.9
denoms
counts
group Id
OG000
value
112
group Id
OG001
value
9
units
Participants
title
≥ 50% Transfusion Burden Reduction

denoms

counts
group Id
OG000
value
173
group Id
OG001
value
39
units
Participants

description

dispersion Type

Standard Deviation

groups

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
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

MEAN

population Description

All randomized participants who achieved erythroid response

reporting Status

POSTED

time Frame

From first dose to 48 weeks following first dose

title

Time to Erythroid Response

type

SECONDARY

unit Of Measure

Days

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Preserved source evidence · Not medical advice