NCT02604433 · OUTCOME
Post-Baseline Transfusion Event Frequency
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
- Number of transfusions
- Interval / dispersion
- Standard Deviation
- Time frame
- From first dose through 3 weeks post last dose (up to approximately 218 weeks)
What was measured
The number of transfusion events after start of study treatment were evaluated. For the definition of transfusion events, if multiple transfusions happen on the same date, they are counted as one event; if multiple transfusions happen on two consecutive dates, they are counted as one event; if multiple transfusions happen on three consecutive dates, they are counted as two events. Results are presented in 24-week intervals, up to 96 weeks after start of study treatment
Analysis population: All randomized participants who received transfusions
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 | 210 |
|---|
| Placebo + BSC | 102 |
|---|
Reported measurements
Week 1 - 24
Analysis denominator · Participants
| Group | Source count |
|---|
| Luspatercept + BSC | 210 |
|---|
| Placebo + BSC | 102 |
|---|
Values in Number of transfusions · Interval/dispersion: Standard Deviation| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Luspatercept + BSC | 7.1 | 2.03 | Not reported | Not reported | Not reported |
|---|
| Placebo + BSC | 7.9 | 1.65 | Not reported | Not reported | Not reported |
|---|
Week 25 - 48
Analysis denominator · Participants
| Group | Source count |
|---|
| Luspatercept + BSC | 201 |
|---|
| Placebo + BSC | 96 |
|---|
Values in Number of transfusions · Interval/dispersion: Standard Deviation| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Luspatercept + BSC | 7.0 | 2.02 | Not reported | Not reported | Not reported |
|---|
| Placebo + BSC | 7.6 | 1.61 | Not reported | Not reported | Not reported |
|---|
Week 49 - 72
Analysis denominator · Participants
| Group | Source count |
|---|
| Luspatercept + BSC | 177 |
|---|
| Placebo + BSC | 62 |
|---|
Values in Number of transfusions · Interval/dispersion: Standard Deviation| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Luspatercept + BSC | 7.0 | 2.04 | Not reported | Not reported | Not reported |
|---|
| Placebo + BSC | 7.5 | 1.28 | Not reported | Not reported | Not reported |
|---|
Week 73 - 96
Analysis denominator · Participants
| Group | Source count |
|---|
| Luspatercept + BSC | 155 |
|---|
| Placebo + BSC | 3 |
|---|
Values in Number of transfusions · Interval/dispersion: Standard Deviation| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Luspatercept + BSC | 7.0 | 2.13 | Not reported | Not reported | Not reported |
|---|
| Placebo + BSC | 7.0 | 1.00 | Not reported | Not reported | Not reported |
|---|
Complete source fields
- classes
- categories
- measurements
- group Id
- OG000
- spread
- 2.03
- value
- 7.1
- group Id
- OG001
- spread
- 1.65
- value
- 7.9
- denoms
- counts
- group Id
- OG000
- value
- 210
- group Id
- OG001
- value
- 102
- units
- Participants
- title
- Week 1 - 24
- categories
- measurements
- group Id
- OG000
- spread
- 2.02
- value
- 7.0
- group Id
- OG001
- spread
- 1.61
- value
- 7.6
- denoms
- counts
- group Id
- OG000
- value
- 201
- group Id
- OG001
- value
- 96
- units
- Participants
- title
- Week 25 - 48
- categories
- measurements
- group Id
- OG000
- spread
- 2.04
- value
- 7.0
- group Id
- OG001
- spread
- 1.28
- value
- 7.5
- denoms
- counts
- group Id
- OG000
- value
- 177
- group Id
- OG001
- value
- 62
- units
- Participants
- title
- Week 49 - 72
- categories
- measurements
- group Id
- OG000
- spread
- 2.13
- value
- 7.0
- group Id
- OG001
- spread
- 1.00
- value
- 7.0
- denoms
- counts
- group Id
- OG000
- value
- 155
- group Id
- OG001
- value
- 3
- units
- Participants
- title
- Week 73 - 96
- denoms
- counts
- group Id
- OG000
- value
- 210
- group Id
- OG001
- value
- 102
- units
- Participants
- description
- The number of transfusion events after start of study treatment were evaluated. For the definition of transfusion events, if multiple transfusions happen on the same date, they are counted as one event; if multiple transfusions happen on two consecutive dates, they are counted as one event; if multiple transfusions happen on three consecutive dates, they are counted as two events. Results are presented in 24-week intervals, up to 96 weeks after start of study treatment
- 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 received transfusions
- reporting Status
- POSTED
- time Frame
- From first dose through 3 weeks post last dose (up to approximately 218 weeks)
- title
- Post-Baseline Transfusion Event Frequency
- type
- SECONDARY
- unit Of Measure
- Number of transfusions
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