EFFICACY

Reduce transfusion burden

Erythroid maturation for significant red blood cell (RBC) transfusion reduction

The efficacy of Reblozyl in β-thalassemia is based on the double-blind, randomized, placebo-controlled, Phase 3 BELIEVE trial (with 224 patients treated with Reblozyl and 112 patients receiving placebo).1

Reblozyl significantly reduces RBC transfusion burden.1

Primary Endpoint:

  • ≥33% reduction (of at least 2 units) from baseline in RBC transfusion burden (Weeks 13-24)

Primary Endpoint: ≥33% reduction in red blood cell (RBC)
transfusion burden • Weeks 13-24*1

21.4% Patients With ß-Thalassemia Experienced ≥33% Transfusion Reduction With Reblozyl Vs 4.5% Placebo During Weeks 13-24

aP-value from the Cochran Mantel-Haenszel test stratified by the geographical region.1

*Reduction from baseline in RBC transfusion burden of at least 2 units for 12 consecutive weeks compared to the 12-week interval prior to treatment.1

Reblozyl reduced the RBC transfusion burden by ≥33% during any 12- or 24-week interval.1

Reblozyl reduces RBC transfusion burden over any consecutive 12- or 24-week interval, a better reflection of assessment in real-world practice than a fixed interval analysis.1,2

Exploratory Endpoint: ≥33% reduction
in any consecutive 12- or 24-week interval*1

Any consecutive 12 weeks
70.5% Patients Receiving Reblozyl (29.5% Placebo) Experienced ≥33% Transfusion Reduction Over Any Consecutive 12 Weeks
Any consecutive 24 weeks
41.1% Patients Receiving Reblozyl (2.7% Placebo) Experienced ≥33% Transfusion Reduction Over Any Consecutive 24 Weeks

*Reduction from baseline in RBC transfusion burden of at least 2 units for 12 or 24 weeks compared to the 12- or 24-week interval prior to treatment.1

≥80% of Reblozyl responders who achieved ≥33% reduction in transfusion burden achieved

2 or more episodes of response.11

Response assessed during any consecutive 12 week interval.

Reblozyl provided a durable reduction in RBC transfusion burden.1,2

A greater proportion of Reblozyl-treated patients achieved clinically meaningful reductions in transfusion burden of ≥50% during a fixed 12-week period and any consecutive 12- or 24-week interval.1

Patients receiving Reblozyl had 4.67 fewer RBC units transfused from Weeks 1-48 versus baseline and 5.66 fewer RBC units transfused from Weeks 49-96, whereas patients under placebo experienced an increase in the RBC units transfused (+1.16 and +2.19, respectively).*1

*Least square mean change from baseline (RBC units/48 weeks).

Secondary Endpoint: ≥50% reduction in RBC transfusion
burden for a fixed 12-week period*1

7.6% Patients Receiving Reblozyl (1.8% Placebo) Experienced ≥50% Transfusion Reduction During Weeks 13-24

a P-value from the Cochran Mantel-Haenszel test stratified by the geographical region.1

*Reduction from baseline in RBC transfusion burden of at least 2 units for 12 consecutive weeks compared to the 12-week interval prior to treatment.1

Exploratory Endpoints: ≥50% transfusion reduction
for any consecutive interval*1

Any consecutive 12 weeks
Any consecutive 24 weeks

*Reduction from baseline in RBC transfusion burden of at least 2 units for 12 or 24 weeks compared to the 12- or 24-week  interval prior to treatment.1

Reduce transfusion burden

Erythroid maturation for significant red blood cell (RBC) transfusion reduction

Reblozyl significantly reduces RBC transfusion burden1

Primary Endpoint: ≥33% reduction in RBC transfusion burden • (Weeks 13-24)*1

21.4% Patients With ß-Thalassemia Experienced ≥33% Transfusion Reduction With Reblozyl Vs 4.5% Placebo During Weeks 13-24

a P-value from the Cochran Mantel-Haenszel test stratified by the geographical region.1

*Reduction from baseline in RBC transfusion burden of at least 2 units for 12 consecutive weeks compared to the 12-week interval prior to treatment.1

The efficacy of Reblozyl in β-thalassemia is based on the double-blind, randomized, placebo-controlled, Phase 3 BELIEVE trial (with 224 patients treated with Reblozyl and 112 patients receiving placebo).1

Exploratory Endpoint: ≥33% reduction in any consecutive 12- or 24-week interval*1

Any consecutive 12 weeks

70.5% Patients Receiving Reblozyl (29.5% Placebo) Experienced ≥33% Transfusion Reduction Over Any Consecutive 12 Weeks

Any consecutive 24 weeks

41.1% Patients Receiving Reblozyl (2.7% Placebo) Experienced ≥33% Transfusion Reduction Over Any Consecutive 24 Weeks

* Reduction from baseline in RBC transfusion burden of at least 2 units for 12 or 24 weeks compared to the 12- or 24-week interval prior to treatment.1

Reblozyl reduces RBC transfusion burden over any consecutive 12- or 24-week interval, a better reflection of assessment in real-world practice than a fixed interval analysis.1,2

≥80% of Reblozyl responders who achieved ≥33% reduction in transfusion burden achieved

2 or more episodes of response.11

Response assessed during
any consecutive 12-week interval.

Secondary Endpoint

Secondary Endpoint: ≥50% reduction in RBC transfusion burden for a fixed 12-week period*1

Reblozyl provided a durable reduction in RBC transfusion burden.1

Weeks 13-24

7.6% Patients Receiving Reblozyl (1.8% Placebo) Experienced ≥50% Transfusion Reduction During Weeks 13-24

a P-value from the Cochran Mantel-Haenszel test stratified by the geographical region.1

*Reduction from baseline in RBC transfusion burden of at least 2 units for 12 consecutive weeks compared to the 12-week interval prior to treatment.1

A greater proportion of Reblozyl-treated patients achieved clinically meaningful reductions in transfusion burden of ≥50% during a fixed 12-week period and any consecutive 12- or 24-week period1.

Exploratory Endpoint

Exploratory Endpoints: ≥50% transfusion reduction for any consecutive interval*1

Any consecutive 12 weeks

Any consecutive 24 weeks

*Reduction from baseline in RBC transfusion burden of at least 2 units for 12 or 24 weeks compared to the 12- or 24-week  interval prior to treatment.1

Patients receiving Reblozyl had 4.67 fewer RBC units transfused from Weeks 1-48 versus baseline and 5.66 fewer RBC units transfused from Weeks 49-96, whereas patients under placebo experienced an increase in the RBC units transfused (+1.16 and +2.19, respectively).*1

*Least square mean change from baseline (RBC units/48 weeks).

References:

1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020.

2. Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion-dependent ß-thalassemia. N Engl J Med. 2020;382:1219-1231. doi:10.1056/NEJMoa1910182.

The efficacy and safety outcomes for Reblozyl in β-thalassemia were evaluated in the pivotal Phase 3 BELIEVE trial1-3

 

BSC=best supportive therapy; RBC=red blood cell.
aBSC included RBC transfusions, iron-chelating agents, use of antibiotic, antiviral and antifungal therapy, and nutritional support, as needed.

BELIEVE (ACE-536-B-THAL-001) was a global, randomized, double-blind, placebo-controlled, Phase 3 trial involving adult patients with beta-thalassemia–associated anemia who require RBC transfusions (6-20 RBC units/24 weeks) with no transfusion-free period >35 days during that period.1

Primary endpoint: ≥33% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units from Weeks 13-24.1

References: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020. 2. Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion-dependent β-thalassemia. N Engl J Med. 2020;382(13):1219-1231. 3. Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion dependent β-thalassemia [supplemental appendix]. N Engl J Med. 2020;382(13):1219-1231.

The efficacy and safety outcomes for Reblozyl in ß-thalassemia were evaluated in the pivotal BELIEVE trial1

 

BSC=best supportive therapy; RBC=red blood cell.
aBSC included RBC transfusions, iron-chelating agents, use of antibiotic, antiviral and antifungal therapy, and nutritional support, as needed.

BELIEVE (ACE-536-B-THAL-001) was a global, randomized, double-blind, placebo-controlled, Phase 3 trial involving adult patients with beta-thalassemia–associated anemia who require RBC transfusions (6-20 RBC units/24 weeks) with no transfusion-free period >35 days during that period.1

Primary endpoint: ≥33% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units from Weeks 13-24.1

References: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020. 2. Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion-dependent β-thalassemia. N Engl J Med. 2020;382(13):1219-1231. 3. Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion dependent β-thalassemia [supplemental appendix]. N Engl J Med. 2020;382(13):1219-1231.

BASELINE DISEASE CHARACTERISTICS OF PATIENTS WITH β-THALASSEMIA IN THE PHASE 3 BELIEVE TRIAL1
DISEASE CHARACTERISTICS REBLOZYL
(N=224)
PLACEBO
(N=112)
Pretransfusion Hb thresholda, 12-week run-in (g/dL)
Median (min, max) 9.30 (4.6, 11.4) 9.16 (6.2, 11.5)
Baseline transfusion burden 12 weeks prior to trial
Median (min, max)
(units/12 weeks) (Week–12 to Day 1)
6.12 (3.0, 14.0) 6.27 (3.0, 12.0)
β-thalassemia gene mutation grouping, n (%)
β00 68 (30.4) 35 (31.3)
Non-β00 155 (69.2) 77 (68.8)
Missingb 1 (0.4) 0

 

aThe 12-week pretransfusion threshold was defined as mean of a subject’s all documented pretansfusion Hb values during 12 weeks prior to Dose 1, Day 1.
b“Missing” category includes patients in the population who had no result for the parameter listed.1

 

Reference: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020.

Baseline disease characteristics of patients with β-thalassemia enrolled in the Phase 3 BELIEVE trial1

DISEASE CHARACTERISTICS REBLOZYL
(N=224)
PLACEBO
(N=112)
Pretransfusion Hb thresholda, 12-week run-in (g/dL)
Median (min, max) 9.30
(4.6, 11.4)
9.16
(6.2, 11.5)
Baseline transfusion burden 12 weeks prior to trial
Median (min, max)
(units/12 weeks) (Week–12 to Day 1)
6.12
(3.0, 14.0)
6.27
(3.0, 12.0)
β-thalassemia gene mutation grouping, n (%)
β00 68
(30.4)
35
(31.3)
Non-β00 155
(69.2)
77
(68.8)
Missingb 1
(0.4)
0

 

aThe 12-week pretransfusion threshold was defined as mean of a subject’s all documented pretansfusion Hb values during 12 weeks prior to Dose 1, Day 1.
b“Missing” category includes patients in the population who had no result for the parameter listed.1

 

Reference: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020.

Reduce serum ferritin levels

Patients treated with Reblozyl experienced significant reductions in mean serum ferritin levels at Week 48 vs an increase with placebo1
  • A significant reduction in mean serum ferritin levels from baseline vs an increase with placebo was seen with Reblozyl at Week 48 (‑233.51 μg/L and +114.28 μg/L, respectively, which resulted in a least square mean difference of -347.8 μg/L)

Impact on serum ferritin levels at Week 481

At Weeks 48, Mean Serum Ferritin Had Decreased 248.02 μg/L With Reblozyl vs Increase Of 106.62 μg/L With Placebo

Reduce serum ferritin levels

Patients treated with Reblozyl experienced significant reductions in mean serum ferritin levels at Week 48 vs an increase with placebo1

Impact on serum ferritin levels at Week 481

At Weeks 48, Mean Serum Ferritin Had Decreased 248.02 μg/L With Reblozyl vs Increase Of 106.62 μg/L With Placebo
  • A significant reduction in mean serum ferritin levels from baseline vs an increase with placebo was seen with Reblozyl at Week 48 (‑233.51 μg/L and +114.28 μg/L, respectively, which resulted in a least square mean difference of -347.8 μg/L)

References:

1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020.

2. Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion-dependent ß-thalassemia. N Engl J Med. 2020;382:1219-1231. doi:10.1056/NEJMoa1910182.

The efficacy and safety outcomes for Reblozyl in β-thalassemia were evaluated in the pivotal Phase 3 BELIEVE trial1-3

 

BSC=best supportive therapy; RBC=red blood cell.
aBSC included RBC transfusions, iron-chelating agents, use of antibiotic, antiviral and antifungal therapy, and nutritional support, as needed.

BELIEVE (ACE-536-B-THAL-001) was a global, randomized, double-blind, placebo-controlled, Phase 3 trial involving adult patients with beta-thalassemia–associated anemia who require RBC transfusions (6-20 RBC units/24 weeks) with no transfusion-free period >35 days during that period.1

Primary endpoint: ≥33% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units from Weeks 13-24.1

References: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020. 2. Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion-dependent β-thalassemia. N Engl J Med. 2020;382(13):1219-1231. 3. Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion dependent β-thalassemia [supplemental appendix]. N Engl J Med. 2020;382(13):1219-1231.

The efficacy and safety outcomes for Reblozyl in ß-thalassemia were evaluated in the pivotal BELIEVE trial1

 

BSC=best supportive therapy; RBC=red blood cell.
aBSC included RBC transfusions, iron-chelating agents, use of antibiotic, antiviral and antifungal therapy, and nutritional support, as needed.

BELIEVE (ACE-536-B-THAL-001) was a global, randomized, double-blind, placebo-controlled, Phase 3 trial involving adult patients with beta-thalassemia–associated anemia who require RBC transfusions (6-20 RBC units/24 weeks) with no transfusion-free period >35 days during that period.1

Primary endpoint: ≥33% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units from Weeks 13-24.1

References: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020. 2. Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion-dependent β-thalassemia. N Engl J Med. 2020;382(13):1219-1231. 3. Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion dependent β-thalassemia [supplemental appendix]. N Engl J Med. 2020;382(13):1219-1231.

BASELINE DISEASE CHARACTERISTICS OF PATIENTS WITH β-THALASSEMIA IN THE PHASE 3 BELIEVE TRIAL1
DISEASE CHARACTERISTICS REBLOZYL
(N=224)
PLACEBO
(N=112)
Pretransfusion Hb thresholda, 12-week run-in (g/dL)
Median (min, max) 9.30 (4.6, 11.4) 9.16 (6.2, 11.5)
Baseline transfusion burden 12 weeks prior to trial
Median (min, max)
(units/12 weeks) (Week–12 to Day 1)
6.12 (3.0, 14.0) 6.27 (3.0, 12.0)
β-thalassemia gene mutation grouping, n (%)
β00 68 (30.4) 35 (31.3)
Non-β00 155 (69.2) 77 (68.8)
Missingb 1 (0.4) 0

 

aThe 12-week pretransfusion threshold was defined as mean of a subject’s all documented pretansfusion Hb values during 12 weeks prior to Dose 1, Day 1.
b“Missing” category includes patients in the population who had no result for the parameter listed.1

 

Reference: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020.

Baseline disease characteristics of patients with β-thalassemia enrolled in the Phase 3 BELIEVE trial1

DISEASE CHARACTERISTICS REBLOZYL
(N=224)
PLACEBO
(N=112)
Pretransfusion Hb thresholda, 12-week run-in (g/dL)
Median (min, max) 9.30
(4.6, 11.4)
9.16
(6.2, 11.5)
Baseline transfusion burden 12 weeks prior to trial
Median (min, max)
(units/12 weeks) (Week–12 to Day 1)
6.12
(3.0, 14.0)
6.27
(3.0, 12.0)
β-thalassemia gene mutation grouping, n (%)
β00 68
(30.4)
35
(31.3)
Non-β00 155
(69.2)
77
(68.8)
Missingb 1
(0.4)
0

 

aThe 12-week pretransfusion threshold was defined as mean of a subject’s all documented pretansfusion Hb values during 12 weeks prior to Dose 1, Day 1.
b“Missing” category includes patients in the population who had no result for the parameter listed.1

 

Reference: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020.

 

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IMPORTANT: The information on this website is based on the European Summary of Product Characteristics (EU SmPC).
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This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. See Section 4.8 in the EU Summary of Product Characteristics for more information. Before prescribing Reblozyl, please refer to the
EU Summary of Product Characteristics.