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 (during weeks 13 to 24)

Reblozyl® reduced the RBC transfusion burden by ≥33% and ≥50% 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

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

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

bCollected over 16 weeks prior to randomization.

Exploratory Endpoint: ≥33% and ≥50% reduction in any consecutive 12- or 24-week interval*1,3

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

Exploratory Endpoints: ≥50% transfusion reduction for any consecutive 12- or 24 week interval*1,3

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 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

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

Reblozyl® significantly reduces RBC transfusion burden.1

Primary Endpoint: ≥33% reduction in 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

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% and ≥50% reduction in any consecutive 12- or 24-week interval*1,3

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

†Response assessed during any
consecutive 12-week interval

Exploratory Endpoints: ≥50% transfusion reduction for any consecutive 12- or 24 week interval*1,3

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).

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)