EFFICACY

Reduce or eliminate transfusions

The efficacy of Reblozyl in MDS is based on the double-blind, randomized, placebo-controlled, phase 3 MEDALIST trial (with 153 patients treated with Reblozyl and 76 patients receiving placebo).1

Significantly more patients experienced RBC transfusion independence (TI) with Reblozyl vs placebo.1

  • 37.9% achieved TI for at least 8 weeks (vs placebo 13.2%, P<0.0001) during Weeks 1-241

Primary Endpoint: RBC transfusion independence (TI)
≥8 weeks • Weeks 1-241

Significantly more patients experienced RBC-TI with Reblozyl vs placebo1
Patients With MDS Experienced Significant RBC Transfusion Independence With Reblozyl For ≥8 Weeks (Weeks 1-24)

Significantly more patients on Reblozyl experienced a longer RBC-TI period vs placebo.1
  • 28.1% achieved TI for at least 12 weeks (vs placebo 7.9%, P=0.0002) during Weeks 1-241
  • 33.3% achieved TI for at least 12 weeks (vs placebo 11.8%, P=0.0003) during Weeks 1-481

Secondary Endpoint: RBC-TI 12 weeks • Weeks 1-481

Significantly more patients experienced a longer RBC-TI period with Reblozyl vs placebo1
Patients With MDS Experienced Significant RBC Transfusion Independence With Reblozyl For ≥12 Weeks (Weeks 1-48)
Patients receiving Reblozyl had fewer RBC transfusion events vs placebo during Weeks 1-24 and Weeks 25-481

During Weeks 1-24:

  • With Reblozyl, patients had 6.26 transfusion events vs 9.20 with placebo (0.68 relative risk vs placebo)1

During Weeks 25-48:

  • With Reblozyl, patients had 6.27 transfusion events vs 8.72 with placebo (0.72 relative risk vs placebo)1

Secondary Endpoint: RBC transfusion event frequency Weeks 1-241

Fewer events with Reblozly vs placebo
Patients receiving Reblozyl required fewer RBC transfusion units vs placebo during Weeks 1-24 and Weeks 25-48 1

During Weeks 1-24:

  • Of patients with a low baseline transfusion burden (<6 units/8 weeks), those receiving Reblozyl received 7.2 units vs 12.8 units with placebo
  • Of patients with a high baseline transfusion burden (≥6 units/8 weeks), those receiving Reblozyl received 18.9 units vs 23.7 units with placebo

During Weeks 25-48:

  • Of patients with a low baseline transfusion burden (<6 units/8 weeks), those receiving Reblozyl received 7.5 units vs 11.8 units with placebo
  • Of patients with a high baseline transfusion burden (≥6 units/8 weeks), those receiving Reblozyl received 19.6 units vs 22.9 units with placebo

Secondary Endpoint: RBC transfusion units • Weeks 1-241

Fewer RBC transfusion units with Reblozyl vs placebo

Low baseline transfusion
burden (<6 units/8 weeks)

High baseline transfusion
burden (≥6 units/8 weeks)

Reduce or eliminate transfusions

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

Primary Endpoint: RBC transfusion independence (TI) ≥8 weeks • Weeks 1-241

Significantly more patients experienced RBC-TI with Reblozyl vs placebo1
Patients With MDS Experienced Significant RBC Transfusion Independence With Reblozyl For ≥8 Weeks (Weeks 1-24)

The efficacy of Reblozyl in MDS is based on the double-blind, randomized, placebo-controlled, phase 3 MEDALIST trial (with 153 patients treated with Reblozyl and 76 patients receiving placebo).1

Secondary Endpoints

Secondary Endpoint: RBC-TI 12 weeks • Weeks 1-481

Significantly more patients experienced a longer RBC-TI period with Reblozyl vs placebo1
Patients With MDS Experienced Significant RBC Transfusion Independence With Reblozyl For ≥12 Weeks (Weeks 1-48)
  • 28.1% achieved TI for at least 12 weeks (vs placebo 7.9%, P=0.0002) during Weeks 1-241
  • 33.3% achieved TI for at least 12 weeks (vs placebo 11.8%, P=0.0003) during Weeks 1-481

Secondary Endpoint: RBC transfusion event frequency • Weeks 1-241

Patients receiving Reblozyl had fewer RBC transfusion events vs placebo during Weeks 1-24 and Weeks 25-481

During Weeks 1-24:

  • With Reblozyl, patients had 6.26 transfusion events vs 9.20 with placebo (0.68 relative risk vs placebo)1

During Weeks 25-48:

  • With Reblozyl, patients had 6.27 transfusion events vs 8.72 with placebo (0.72 relative risk vs placebo)1

Secondary Endpoint: RBC transfusion units • Weeks 1-241

Patients receiving Reblozyl required fewer RBC transfusion units vs placebo during Weeks 1-24 and Weeks 25-48 1

Low baseline transfusion burden (<6 units/8 weeks)

High baseline transfusion burden (≥6 units/8 weeks)

During Weeks 1-24:

  • Of patients with a low baseline transfusion burden (<6 units/8 weeks), those receiving Reblozyl received 7.2 units vs 12.8 units with placebo
  • Of patients with a high baseline transfusion burden (≥6 units/8 weeks), those receiving Reblozyl received 18.9 units vs 23.7 units with placebo

During Weeks 25-48:

  • Of patients with a low baseline transfusion burden (<6 units/8 weeks), those receiving Reblozyl received 7.5 units vs 11.8 units with placebo
  • Of patients with a high baseline transfusion burden (≥6 units/8 weeks), those receiving Reblozyl received 19.6 units vs 22.9 units with placebo

References:

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

2. Fenaux P, Platzbecker U, Mufti GJ, et al. Luspatercept in patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:140-151.

The efficacy and safety outcomes for Reblozyl were evaluated
in the pivotal phase 3 MEDALIST trial1-3

MEDALIST (ACE-536-MDS-001) was a global, randomized, double-blind, placebo-controlled Phase 3 trial involving adult patients with anemia requiring RBC transfusions (≥2 units/8 weeks) due to IPSS-R very low-, low-, or intermediate-risk MDS, who have RS (with either ≥15% ring sideroblasts or ≥5% ring sideroblasts if an SF3B1 mutation was present). Patients were required to have either received prior treatment with an ESA with inadequate response, to be ineligible for ESAs (determined to be unlikely to respond to ESA treatment with serum EPO >200 U/L), or intolerant to ESA treatment.1

Primary endpoint: RBC transfusion independence (RBC-TI) ≥8 weeks (Weeks 1-24)1

References: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020. 2. Fenaux P, Haase D, Sanz GF, Santini V, Buske C; on behalf of the ESMO Guidelines Working Group. Myelodysplastic syndromes: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2014;25(suppl 3):iii57-iii69. 3. Fenaux P, Platzbecker U, Mufti GJ, et al. Luspatercept in patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:140-151.

The efficacy and safety outcomes for Reblozyl were evaluated in the pivotal Phase 3 MEDALIST trial1,2

 

MEDALIST (ACE-536-MDS-001) was a global, randomized, double-blind, placebo-controlled Phase 3 trial involving adult patients with anemia requiring RBC transfusions (≥2 units/8 weeks) due to IPSS-R very low-, low-, or intermediate-risk MDS, who have RS (with either ≥15% ring sideroblasts or ≥5% ring sideroblasts if an SF3B1 mutation was present). Patients were required to have either received prior treatment with an ESA with inadequate response, to be ineligible for ESAs (determined to be unlikely to respond to ESA treatment with serum EPO >200 U/L), or intolerant to ESA treatment.1

Primary endpoint: RBC transfusion independence (RBC-TI) ≥8 weeks (Weeks 1-24)1

References:

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

2. Fenaux P, Platzbecker, GJ, Mufti G et al. Luspatercept patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:149-151.

BASELINE DISEASE CHARACTERISTICS OF MDS PATIENTS
ENROLLED IN THE PHASE 3 MEDALIST TRIAL1
DISEASE CHARACTERISTICS REBLOZYL
(N=153)
PLACEBO
(N=76)
Serum EPO (U/L) categories,a n (%)
<200 88 (57.5) 50 (65.8)
200 to 500 43 (28.1) 15 (19.7)
>500 21 (13.7) 11 (14.5)
Missing 1 (0.7) 0
IPSS-R classification risk category, n (%)
Very low 18 (11.8) 6 (7.9)
Low 109 (71.2)  57 (75.0)
Intermediate 25 (16.3) 13 (17.1)
Other 1 (0.7) 0
Baseline RBC transfusion burden/last 8 weeks,b n (%)
≥6 units 66 (43.1) 33 (43.4)
≥6 and <8 units 35 (22.9) 15 (20.2)
≥8 and <12 units 24 (15.7) 17 (22.4)
≥12 units 7 (4.6) 1 (1.3)
<6 units 87 (56.9) 43 (56.6)
≥4 units and <6 units 41 (26.8) 23 (30.3)
<4 units 46 (30.1) 20 (26.3)
Hemoglobinc (g/dL)
Median (min, max) 7.6 (6,10) 7.6 (5,9)
SF3B1, n (%)
Mutated 149 (92.2) 65 (85.5)
Unmutated 12 (7.8) 10 (13.2)
Missing 0 1 (1.3)

 

EPO=erythropoietin; IPSS-R=International Prognostic Scoring System-Revised.
a Baseline EPO was defined as the highest EPO value within 35 days of the first dose of study drug.
b Collected over 16 weeks prior to randomization.
c Baseline hemoglobin was defined as the last value measured on or before the date of the first dose of investigational product.

References: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020. 2. Fenaux P, Haase D, Sanz GF, Santini V, Buske C; on behalf of the ESMO Guidelines Working Group. Myelodysplastic syndromes: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2014;25(suppl 3):iii57-iii69.

Improve hemoglobin levels

More patients on Reblozyl achieved a hematologic improvement, according to the International Working Group (IWG) 2006 response criteria, during Weeks 1-24.1,2

During Weeks 1-24:

  • 52.9% of patients achieved modified hematologic improvement-erythroid (mHI-E) vs 11.8% for placebo1,c
    • 63.0% achieved a mean hemoglobin (Hb) increase of at least 1.5 g/dL for 8 weeks vs 5.0% for placebo1,c
    • 48.6% achieved a RBC transfusion reduction of 4 units/8 weeks vs 14.3% for placebo1,c

Exploratory Endpoint: Modified hematologic improvement-erythroid (mHI-E)a,b,cWeeks 1-241

More patients achieved mHI-E at Week 24 with Reblozyl vs placebo1
52.9% Of Patients With MDS Achieved A Hematologic Improvement vs 11.8% Receiving Placebo Through Week 24

aResponse criteria were developed by the IWG.

bThe proportion of patients meeting the HI-E criteria as per IWG 2006 criteria sustained over a consecutive 56-day period during the indicated treatment period.

cIn patients with a baseline transfusion burden of ≥4 units/8 weeks, mHI-E was defined as a reduction in RBC transfusions of at least 4 units/8 weeks. For patients with a baseline RBC burden of <4 units/8 weeks, mHI-E was defined as a mean increase in Hb of ≥1.5 g/dL for 8 weeks in the absence of transfusions.

More patients receiving Reblozyl achieved a hematologic improvement through Week 48 vs placebo1

During Weeks 1-48:

  • 58.8% of patients achieved mHI-E vs 17.1% for placebo1.
    • 69.6% achieved a mean Hb increase of at least 1.5 g/dL for 8 weeks vs 5.0% for placebo1,c.
    • 54.2% achieved a RBC transfusion reduction of 4 units/8 weeks vs 21.4% for placebo1,c.

Exploratory Endpoint: Modified hematologic improvement-erythroid (mHI-E)a,b,c  Weeks 1-481

More patients achieved mHI-E at Week 48 with Reblozyl vs placebo
58.8% Of Patients With MDS Achieved A Hematologic Improvement vs 17.1% Receiving Placebo Through Week 48

aResponse criteria were developed by the IWG.

bThe proportion of patients meeting the HI-E criteria as per IWG 2006 criteria sustained over a consecutive 56-day period during the indicated treatment period.

cIn patients with a baseline transfusion burden of ≥4 units/8 weeks, mHI-E was defined as a reduction in RBC transfusions of at least 4 units/8 weeks. For patients with a baseline RBC burden of <4 units/8 weeks, mHI-E was defined as a mean increase in Hb of ≥1.5 g/dL for 8 weeks in the absence of transfusions.

Improve hemoglobin levels

Erythroid maturation for achieving hematologic improvement

More patients on Reblozyl achieved a hematologic improvement, according to the International Working Group (IWG) 2006 response criteria, during Weeks 1-24.1

Exploratory Endpoint: Modified hematologic
improvement-erythroid (mHI-E)a,b,c  Weeks 1-241

More patients achieved mHI-E at Week 24 with Reblozyl vs placebo1
52.9% Of Patients With MDS Achieved A Hematologic Improvement vs 11.8% Receiving Placebo Through Week 24
  • 63.0% achieved a mean hemoglobin (Hb) increase of at least 1.5 g/dL for 8 weeks vs 5.0% for placebo1,c
  • 48.6% achieved a RBC transfusion reduction of 4 units/8 weeks vs 14.3% for placebo1,c

aResponse criteria were developed by the IWG.

bThe proportion of patients meeting the HI-E criteria as per IWG 2006 criteria sustained over a consecutive 56-day period during the indicated treatment period.

cIn patients with a baseline transfusion burden of ≥4 units/8 weeks, mHI-E was defined as a reduction in RBC transfusions of at least 4 units/8 weeks. For patients with a baseline RBC burden of <4 units/8 weeks, mHI-E was defined as a mean increase in hemoglobin of ≥1.5 g/dL for 8 weeks in the absence of transfusions.

Exploratory endpoint: mHI-E improvement up to Week 48 with Reblozyl

Exploratory Endpoint: mHI-Ea,b,c  Weeks 1-481

More patients achieved mHI-E at Week 48 with Reblozyl vs placebo1
58.8% Of Patients With MDS Achieved A Hematologic Improvement vs 17.1% Receiving Placebo Through Week 48
  • 69.6% achieved a mean Hb increase of at least 1.5 g/dL for 8 weeks vs 5.0% for placebo1,c.
  • 54.2% achieved a RBC transfusion reduction of 4 units/8 weeks vs 21.4% for placebo1,c.

aResponse criteria were developed by the IWG.

bThe proportion of patients meeting the HI-E criteria as per IWG 2006 criteria sustained over a consecutive 56-day period during the indicated treatment period.

cIn patients with a baseline transfusion burden of ≥4 units/8 weeks, mHI-E was defined as a reduction in RBC transfusions of at least 4 units/8 weeks. For patients with a baseline RBC burden of <4 units/8 weeks, mHI-E was defined as a mean increase in hemoglobin of ≥1.5 g/dL for 8 weeks in the absence of transfusions.1

References:

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

2. Fenaux P, Platzbecker U, Mufti GJ, et al. Luspatercept in patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:140-151.

The efficacy and safety outcomes for Reblozyl were evaluated
in the pivotal phase 3 MEDALIST trial1-3

MEDALIST (ACE-536-MDS-001) was a global, randomized, double-blind, placebo-controlled Phase 3 trial involving adult patients with anemia requiring RBC transfusions (≥2 units/8 weeks) due to IPSS-R very low-, low-, or intermediate-risk MDS, who have RS (with either ≥15% ring sideroblasts or ≥5% ring sideroblasts if an SF3B1 mutation was present). Patients were required to have either received prior treatment with an ESA with inadequate response, to be ineligible for ESAs (determined to be unlikely to respond to ESA treatment with serum EPO >200 U/L), or intolerant to ESA treatment.1

Primary endpoint: RBC transfusion independence (RBC-TI) ≥8 weeks (Weeks 1-24)1

References: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020. 2. Fenaux P, Haase D, Sanz GF, Santini V, Buske C; on behalf of the ESMO Guidelines Working Group. Myelodysplastic syndromes: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2014;25(suppl 3):iii57-iii69. 3. Fenaux P, Platzbecker U, Mufti GJ, et al. Luspatercept in patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:140-151.

The efficacy and safety outcomes for Reblozyl were evaluated in the pivotal Phase 3 MEDALIST trial1,2

 

MEDALIST (ACE-536-MDS-001) was a global, randomized, double-blind, placebo-controlled Phase 3 trial involving adult patients with anemia requiring RBC transfusions (≥2 units/8 weeks) due to IPSS-R very low-, low-, or intermediate-risk MDS, who have RS (with either ≥15% ring sideroblasts or ≥5% ring sideroblasts if an SF3B1 mutation was present). Patients were required to have either received prior treatment with an ESA with inadequate response, to be ineligible for ESAs (determined to be unlikely to respond to ESA treatment with serum EPO >200 U/L), or intolerant to ESA treatment.1

Primary endpoint: RBC transfusion independence (RBC-TI) ≥8 weeks (Weeks 1-24)1

References:

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

2. Fenaux P, Platzbecker, GJ, Mufti G et al. Luspatercept patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:149-151.

BASELINE DISEASE CHARACTERISTICS OF MDS PATIENTS
ENROLLED IN THE PHASE 3 MEDALIST TRIAL1
DISEASE CHARACTERISTICS REBLOZYL
(N=153)
PLACEBO
(N=76)
Serum EPO (U/L) categories,a n (%)
<200 88 (57.5) 50 (65.8)
200 to 500 43 (28.1) 15 (19.7)
>500 21 (13.7) 11 (14.5)
Missing 1 (0.7) 0
IPSS-R classification risk category, n (%)
Very low 18 (11.8) 6 (7.9)
Low 109 (71.2)  57 (75.0)
Intermediate 25 (16.3) 13 (17.1)
Other 1 (0.7) 0
Baseline RBC transfusion burden/last 8 weeks,b n (%)
≥6 units 66 (43.1) 33 (43.4)
≥6 and <8 units 35 (22.9) 15 (20.2)
≥8 and <12 units 24 (15.7) 17 (22.4)
≥12 units 7 (4.6) 1 (1.3)
<6 units 87 (56.9) 43 (56.6)
≥4 units and <6 units 41 (26.8) 23 (30.3)
<4 units 46 (30.1) 20 (26.3)
Hemoglobinc (g/dL)
Median (min, max) 7.6 (6,10) 7.6 (5,9)
SF3B1, n (%)
Mutated 149 (92.2) 65 (85.5)
Unmutated 12 (7.8) 10 (13.2)
Missing 0 1 (1.3)

 

EPO=erythropoietin; IPSS-R=International Prognostic Scoring System-Revised.
a Baseline EPO was defined as the highest EPO value within 35 days of the first dose of study drug.
b Collected over 16 weeks prior to randomization.
c Baseline hemoglobin was defined as the last value measured on or before the date of the first dose of investigational product.

References: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020. 2. Fenaux P, Haase D, Sanz GF, Santini V, Buske C; on behalf of the ESMO Guidelines Working Group. Myelodysplastic syndromes: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2014;25(suppl 3):iii57-iii69.

Rapid and sustained response

Response to Reblozyl treatment was rapid and sustained.1
  • The median duration of the longest RBC-TI period among responders in the Reblozyl treatment arm was 30.6 weeks1
  • With Reblozyl, hemoglobin began to increase within 7 days from treatment initiation in patients who received <4 units of RBC transfusions within 8 weeks prior to the study1

Reblozyl treatment response1,2

With Reblozyl, Hemoglobin Increased Within Just 7 Days and the Median Duration of Response Was 30.6 Weeks

aFor patients with baseline RBC transfusion burden of <4 units within 8 weeks prior to the study.1

62.1% of Reblozyl responders had 2 or more episodes of RBC-T1.1

Rapid and sustained response

Response to Reblozyl treatment was rapid and sustained.1

Reblozyl treatment response1,2

With Reblozyl, Hemoglobin Increased Within Just 7 Days and the Median Duration of Response Was 30.6 Weeks

aFor patients with baseline RBC transfusion burden of <4 units within 8 weeks prior to the study.1

  • The median duration of the longest RBC-TI period among responders in the Reblozyl treatment arm was 30.6 weeks1
  • With Reblozyl, hemoglobin began to increase within 7 days from treatment initiation in patients who received <4 units of RBC transfusions within 8 weeks prior to the study 1

62.1% of Reblozyl responders had 2 or more episodes of RBC-T1.1

References:

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

2. Fenaux P, Platzbecker U, Mufti GJ, et al. Luspatercept in patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:140-151.

The efficacy and safety outcomes for Reblozyl were evaluated
in the pivotal phase 3 MEDALIST trial1-3

MEDALIST (ACE-536-MDS-001) was a global, randomized, double-blind, placebo-controlled Phase 3 trial involving adult patients with anemia requiring RBC transfusions (≥2 units/8 weeks) due to IPSS-R very low-, low-, or intermediate-risk MDS, who have RS (with either ≥15% ring sideroblasts or ≥5% ring sideroblasts if an SF3B1 mutation was present). Patients were required to have either received prior treatment with an ESA with inadequate response, to be ineligible for ESAs (determined to be unlikely to respond to ESA treatment with serum EPO >200 U/L), or intolerant to ESA treatment.1

Primary endpoint: RBC transfusion independence (RBC-TI) ≥8 weeks (Weeks 1-24)1

References: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020. 2. Fenaux P, Haase D, Sanz GF, Santini V, Buske C; on behalf of the ESMO Guidelines Working Group. Myelodysplastic syndromes: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2014;25(suppl 3):iii57-iii69. 3. Fenaux P, Platzbecker U, Mufti GJ, et al. Luspatercept in patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:140-151.

The efficacy and safety outcomes for Reblozyl were evaluated in the pivotal Phase 3 MEDALIST trial1,2

 

MEDALIST (ACE-536-MDS-001) was a global, randomized, double-blind, placebo-controlled Phase 3 trial involving adult patients with anemia requiring RBC transfusions (≥2 units/8 weeks) due to IPSS-R very low-, low-, or intermediate-risk MDS, who have RS (with either ≥15% ring sideroblasts or ≥5% ring sideroblasts if an SF3B1 mutation was present). Patients were required to have either received prior treatment with an ESA with inadequate response, to be ineligible for ESAs (determined to be unlikely to respond to ESA treatment with serum EPO >200 U/L), or intolerant to ESA treatment.1

Primary endpoint: RBC transfusion independence (RBC-TI) ≥8 weeks (Weeks 1-24)1

References:

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

2. Fenaux P, Platzbecker, GJ, Mufti G et al. Luspatercept patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:149-151.

BASELINE DISEASE CHARACTERISTICS OF MDS PATIENTS
ENROLLED IN THE PHASE 3 MEDALIST TRIAL1
DISEASE CHARACTERISTICS REBLOZYL
(N=153)
PLACEBO
(N=76)
Serum EPO (U/L) categories,a n (%)
<200 88 (57.5) 50 (65.8)
200 to 500 43 (28.1) 15 (19.7)
>500 21 (13.7) 11 (14.5)
Missing 1 (0.7) 0
IPSS-R classification risk category, n (%)
Very low 18 (11.8) 6 (7.9)
Low 109 (71.2)  57 (75.0)
Intermediate 25 (16.3) 13 (17.1)
Other 1 (0.7) 0
Baseline RBC transfusion burden/last 8 weeks,b n (%)
≥6 units 66 (43.1) 33 (43.4)
≥6 and <8 units 35 (22.9) 15 (20.2)
≥8 and <12 units 24 (15.7) 17 (22.4)
≥12 units 7 (4.6) 1 (1.3)
<6 units 87 (56.9) 43 (56.6)
≥4 units and <6 units 41 (26.8) 23 (30.3)
<4 units 46 (30.1) 20 (26.3)
Hemoglobinc (g/dL)
Median (min, max) 7.6 (6,10) 7.6 (5,9)
SF3B1, n (%)
Mutated 149 (92.2) 65 (85.5)
Unmutated 12 (7.8) 10 (13.2)
Missing 0 1 (1.3)

 

EPO=erythropoietin; IPSS-R=International Prognostic Scoring System-Revised.
a Baseline EPO was defined as the highest EPO value within 35 days of the first dose of study drug.
b Collected over 16 weeks prior to randomization.
c Baseline hemoglobin was defined as the last value measured on or before the date of the first dose of investigational product.

References: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020. 2. Fenaux P, Haase D, Sanz GF, Santini V, Buske C; on behalf of the ESMO Guidelines Working Group. Myelodysplastic syndromes: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2014;25(suppl 3):iii57-iii69.

Impact serum ferritin levels

Lower mean serum ferritin levels were observed from baseline with Reblozyl compared with placebo.1
  • At Weeks 9-24, mean serum ferritin had increased 9.9 μg/L with Reblozyl vs an increase of 190 μg/L with placebo1
    • The least square mean difference (SE) was –180.1 μg/L

Exploratory Endpoint: Impact on serum ferritin levels with Reblozyl1

At Weeks 9-24, Mean Serum Ferritin Had Decreased 2.7 μg/L With Reblozyl vs Increase of 226.5 μg/L With Placebo

Impact Serum Ferritin Levels

Lower mean serum ferritin levels were observed from baseline with Reblozyl compared with placebo.1

Exploratory Endpoint: Impact on serum ferritin levels with Reblozyl1

At Weeks 9-24, Mean Serum Ferritin Had Decreased 2.7 μg/L With Reblozyl vs Increase of 226.5 μg/L With Placebo
  • At Weeks 9-24, mean serum ferritin had increased 9.9 μg/L  with Reblozyl vs an increase of 190 μg/L  with placebo1
    • The least square mean difference (SE) was -180.1 μg/L

References:

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

2. Fenaux P, Platzbecker U, Mufti GJ, et al. Luspatercept in patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:140-151.

The efficacy and safety outcomes for Reblozyl were evaluated
in the pivotal phase 3 MEDALIST trial1-3

MEDALIST (ACE-536-MDS-001) was a global, randomized, double-blind, placebo-controlled Phase 3 trial involving adult patients with anemia requiring RBC transfusions (≥2 units/8 weeks) due to IPSS-R very low-, low-, or intermediate-risk MDS, who have RS (with either ≥15% ring sideroblasts or ≥5% ring sideroblasts if an SF3B1 mutation was present). Patients were required to have either received prior treatment with an ESA with inadequate response, to be ineligible for ESAs (determined to be unlikely to respond to ESA treatment with serum EPO >200 U/L), or intolerant to ESA treatment.1

Primary endpoint: RBC transfusion independence (RBC-TI) ≥8 weeks (Weeks 1-24)1

References: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020. 2. Fenaux P, Haase D, Sanz GF, Santini V, Buske C; on behalf of the ESMO Guidelines Working Group. Myelodysplastic syndromes: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2014;25(suppl 3):iii57-iii69. 3. Fenaux P, Platzbecker U, Mufti GJ, et al. Luspatercept in patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:140-151.

The efficacy and safety outcomes for Reblozyl were evaluated in the pivotal Phase 3 MEDALIST trial1,2

 

MEDALIST (ACE-536-MDS-001) was a global, randomized, double-blind, placebo-controlled Phase 3 trial involving adult patients with anemia requiring RBC transfusions (≥2 units/8 weeks) due to IPSS-R very low-, low-, or intermediate-risk MDS, who have RS (with either ≥15% ring sideroblasts or ≥5% ring sideroblasts if an SF3B1 mutation was present). Patients were required to have either received prior treatment with an ESA with inadequate response, to be ineligible for ESAs (determined to be unlikely to respond to ESA treatment with serum EPO >200 U/L), or intolerant to ESA treatment.1

Primary endpoint: RBC transfusion independence (RBC-TI) ≥8 weeks (Weeks 1-24)1

References:

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

2. Fenaux P, Platzbecker, GJ, Mufti G et al. Luspatercept patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:149-151.

BASELINE DISEASE CHARACTERISTICS OF MDS PATIENTS
ENROLLED IN THE PHASE 3 MEDALIST TRIAL1
DISEASE CHARACTERISTICS REBLOZYL
(N=153)
PLACEBO
(N=76)
Serum EPO (U/L) categories,a n (%)
<200 88 (57.5) 50 (65.8)
200 to 500 43 (28.1) 15 (19.7)
>500 21 (13.7) 11 (14.5)
Missing 1 (0.7) 0
IPSS-R classification risk category, n (%)
Very low 18 (11.8) 6 (7.9)
Low 109 (71.2)  57 (75.0)
Intermediate 25 (16.3) 13 (17.1)
Other 1 (0.7) 0
Baseline RBC transfusion burden/last 8 weeks,b n (%)
≥6 units 66 (43.1) 33 (43.4)
≥6 and <8 units 35 (22.9) 15 (20.2)
≥8 and <12 units 24 (15.7) 17 (22.4)
≥12 units 7 (4.6) 1 (1.3)
<6 units 87 (56.9) 43 (56.6)
≥4 units and <6 units 41 (26.8) 23 (30.3)
<4 units 46 (30.1) 20 (26.3)
Hemoglobinc (g/dL)
Median (min, max) 7.6 (6,10) 7.6 (5,9)
SF3B1, n (%)
Mutated 149 (92.2) 65 (85.5)
Unmutated 12 (7.8) 10 (13.2)
Missing 0 1 (1.3)

 

EPO=erythropoietin; IPSS-R=International Prognostic Scoring System-Revised.
a Baseline EPO was defined as the highest EPO value within 35 days of the first dose of study drug.
b Collected over 16 weeks prior to randomization.
c Baseline hemoglobin was defined as the last value measured on or before the date of the first dose of investigational product.

References: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020. 2. Fenaux P, Haase D, Sanz GF, Santini V, Buske C; on behalf of the ESMO Guidelines Working Group. Myelodysplastic syndromes: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2014;25(suppl 3):iii57-iii69.

Various patient groups benefit

A treatment effect in favor of Reblozyl over placebo was observed in most subgroups analyzed using transfusion independence ≥12 weeks (during week 1 to week 24).1

Reblozyl demonstrated greater transfusion independence
≥12 weeks in most subgroups analyzed, including*1:

Reblozyl Demonstrated A Reduction In RBC Transfusions In Patients With  High Baseline EPO Levels (≥200 U/L)
Patients with high baseline ENDOGENOUS EPO levels (200-500 U/L) (23.3% vs 0%, explorative analysis)

*Analyzed using transfusion independence ≥12 weeks (during Week 1 to Week 24).1

Various patient groups benefit

Reblozyl demonstrated efficacy across various patient groups1

A treatment effect in favor of Reblozyl over placebo was observed in most subgroups analyzed using transfusion independence ≥12 weeks (during week 1 to week 24).1

Reblozyl demonstrated greater transfusion independence ≥12 weeks in most subgroups analyzed, including*1:

Reblozyl Demonstrated A Reduction In RBC Transfusions In Patients With  High Baseline EPO Levels (≥200 U/L)
Patients with high baseline ENDOGENOUS EPO levels (200-500 U/L) (23.3% vs 0%, explorative analysis)

*Analyzed using transfusion independence ≥12 weeks (during Week 1 to Week 24).1

References:

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

2. Fenaux P, Platzbecker U, Mufti GJ, et al. Luspatercept in patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:140-151.

The efficacy and safety outcomes for Reblozyl were evaluated
in the pivotal phase 3 MEDALIST trial1-3

MEDALIST (ACE-536-MDS-001) was a global, randomized, double-blind, placebo-controlled Phase 3 trial involving adult patients with anemia requiring RBC transfusions (≥2 units/8 weeks) due to IPSS-R very low-, low-, or intermediate-risk MDS, who have RS (with either ≥15% ring sideroblasts or ≥5% ring sideroblasts if an SF3B1 mutation was present). Patients were required to have either received prior treatment with an ESA with inadequate response, to be ineligible for ESAs (determined to be unlikely to respond to ESA treatment with serum EPO >200 U/L), or intolerant to ESA treatment.1

Primary endpoint: RBC transfusion independence (RBC-TI) ≥8 weeks (Weeks 1-24)1

References: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020. 2. Fenaux P, Haase D, Sanz GF, Santini V, Buske C; on behalf of the ESMO Guidelines Working Group. Myelodysplastic syndromes: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2014;25(suppl 3):iii57-iii69. 3. Fenaux P, Platzbecker U, Mufti GJ, et al. Luspatercept in patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:140-151.

The efficacy and safety outcomes for Reblozyl were evaluated in the pivotal Phase 3 MEDALIST trial1,2

 

MEDALIST (ACE-536-MDS-001) was a global, randomized, double-blind, placebo-controlled Phase 3 trial involving adult patients with anemia requiring RBC transfusions (≥2 units/8 weeks) due to IPSS-R very low-, low-, or intermediate-risk MDS, who have RS (with either ≥15% ring sideroblasts or ≥5% ring sideroblasts if an SF3B1 mutation was present). Patients were required to have either received prior treatment with an ESA with inadequate response, to be ineligible for ESAs (determined to be unlikely to respond to ESA treatment with serum EPO >200 U/L), or intolerant to ESA treatment.1

Primary endpoint: RBC transfusion independence (RBC-TI) ≥8 weeks (Weeks 1-24)1

References:

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

2. Fenaux P, Platzbecker, GJ, Mufti G et al. Luspatercept patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020;382:149-151.

BASELINE DISEASE CHARACTERISTICS OF MDS PATIENTS
ENROLLED IN THE PHASE 3 MEDALIST TRIAL1
DISEASE CHARACTERISTICS REBLOZYL
(N=153)
PLACEBO
(N=76)
Serum EPO (U/L) categories,a n (%)
<200 88 (57.5) 50 (65.8)
200 to 500 43 (28.1) 15 (19.7)
>500 21 (13.7) 11 (14.5)
Missing 1 (0.7) 0
IPSS-R classification risk category, n (%)
Very low 18 (11.8) 6 (7.9)
Low 109 (71.2)  57 (75.0)
Intermediate 25 (16.3) 13 (17.1)
Other 1 (0.7) 0
Baseline RBC transfusion burden/last 8 weeks,b n (%)
≥6 units 66 (43.1) 33 (43.4)
≥6 and <8 units 35 (22.9) 15 (20.2)
≥8 and <12 units 24 (15.7) 17 (22.4)
≥12 units 7 (4.6) 1 (1.3)
<6 units 87 (56.9) 43 (56.6)
≥4 units and <6 units 41 (26.8) 23 (30.3)
<4 units 46 (30.1) 20 (26.3)
Hemoglobinc (g/dL)
Median (min, max) 7.6 (6,10) 7.6 (5,9)
SF3B1, n (%)
Mutated 149 (92.2) 65 (85.5)
Unmutated 12 (7.8) 10 (13.2)
Missing 0 1 (1.3)

 

EPO=erythropoietin; IPSS-R=International Prognostic Scoring System-Revised.
a Baseline EPO was defined as the highest EPO value within 35 days of the first dose of study drug.
b Collected over 16 weeks prior to randomization.
c Baseline hemoglobin was defined as the last value measured on or before the date of the first dose of investigational product.

References: 1. Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). Celgene 2020. 2. Fenaux P, Haase D, Sanz GF, Santini V, Buske C; on behalf of the ESMO Guidelines Working Group. Myelodysplastic syndromes: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2014;25(suppl 3):iii57-iii69.

 

This site is not intended for US or UK healthcare professionals.

The product has received European Medicines Agency approval. The registration status and approved product labels of Reblozyl may vary from country to country. The information on this website is not country-specific and may not be applicable to your country. For more information, please refer to your local Prescribing Information.
IMPORTANT: The information on this website is based on the European Summary of Product Characteristics (EU SmPC).
This site is not intended for US or UK healthcare professionals. View the US site.
The information provided on this website is intended for use by a healthcare professional qualified to prescribe and supply medications, thus requiring specific scientific knowledge and training to interpret it correctly.
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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.