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
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
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 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
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
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
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
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
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).
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.
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
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
- 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)
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.