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This website is intended for US Healthcare Professionals.
Request a RepINDICATIONS
REBLOZYL® (luspatercept-aamt) is indicated for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.
REBLOZYL® (luspatercept-aamt) is indicated for the treatment of anemia without previous erythropoiesis stimulating agent use (ESA-naïve) in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell (RBC) transfusions.
REBLOZYL® (luspatercept-aamt) is indicated for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell (RBC) units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).
REBLOZYL is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia.
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PATIENT OUTCOMES: REBLOZYL EFFICACY
REBLOZYL provided substantial clinical benefit by reducing RBC transfusion burden1,2
The efficacy of REBLOZYL in patients with beta-thalassemia was demonstrated in the BELIEVE trial
Study design: REBLOZYL was studied in the pivotal phase 3 BELIEVE trial of 336 adult patients with beta-thalassemia requiring regular RBC transfusions (6-20 RBC units per 24 weeks) with no transfusion-free period greater than 35 days during that period who were randomized 2:1 to REBLOZYL (n=224) or placebo (n=112). In BELIEVE, REBLOZYL was administered subcutaneously once every 3 weeks as long as a reduction in transfusion requirement was observed or until unacceptable toxicity. Patients were able to receive BSC as needed, including: RBC transfusions; iron-chelating agents; use of antibiotic, antiviral, and antifungal therapy; and nutritional support. The exclusion criteria for this trial included HbS/beta-thalassemia or alpha-thalassemia; major organ damage (liver, heart, or lung disease, or renal insufficiency); recent deep vein thrombosis or stroke; or recent use of ESA, immunosuppressant, or hydroxyurea therapy. At 48 weeks, patients could cross over to REBLOZYL as part of the long-term follow-up.1,3
ESA=erythropoiesis-stimulating agent; HbS=hemoglobin S.
REBLOZYL was studied in the multicenter, randomized, double-blind , placebo-controlled, phase 3 BELIEVE trial
ESA=erythropoiesis-stimulating agent; HbS=hemoglobin S; SC=subcutaneous injection.
Primary endpoint1
- ≥33% reduction in RBC transfusion burden of at least 2 units from Weeks 13 to 24
Key secondary endpoints1
- ≥50% reduction in RBC transfusion burden from baseline of at least 2 units during Weeks 13 to 24
- ≥33% and ≥50% reduction in RBC transfusion burden to assess the durability of response from baseline of at least 2 units during Weeks 37 to 48
Additional endpoints2
- ≥33% or ≥50% reduction from baseline in RBC transfusion burden during any consecutive 24 weeks
- Duration of reduction in transfusion burden
- Time to erythroid response
All patients in the pivotal phase 3 BELIEVE trial received regular RBC transfusions1
Baseline characteristics of patients with beta-thalassemia in the pivotal phase 3 BELIEVE trial (ITT population)1,2
| Demographic and disease characteristics | REBLOZYL + BSC (n=224) |
Placebo + BSC (n=112) |
|---|---|---|
| Age, years | ||
| Median (min, max) | 30.0 (18, 66) | 30.0 (18, 59) |
| Baseline transfusion burden 12 weeks prior to randomization, units/12 weeks | ||
| Median (min, max) | 6.12 (3, 14) | 6.27 (3, 12) |
| Beta-thalassemia gene mutation grouping, n (%) | ||
| β0/β0 | 68 (30.4) | 35 (31.3) |
| Non-β0/β0 | 155 (69.2) | 77 (68.8) |
| Missinga | 1 (0.4) | 0 |
| Baseline serum ferritin level, μg/L | ||
| N | 220 | 111 |
| Median (min, max) | 1441.25 (88, 6400) | 1301.50 (136, 6400) |
| Splenectomy, n (%) | ||
| Yes | 129 (57.6) | 65 (58) |
| No | 95 (42.4) | 47 (42) |
| Sex, n (%) | ||
| Male | 92 (41.1) | 49 (43.7) |
| Female | 132 (58.9) | 63 (56.3) |
| Beta-thalassemia diagnosis, n (%) | ||
| β-thalassemia | 174 (77.7) | 83 (74.1) |
| HbE/β-thalassemia | 31 (13.8) | 21 (18.8) |
| β-thalassemia combined with α-thalassemia | 18 (8) | 8 (7.1) |
| Missinga | 1 (0.4) | 0 |
Missing category includes patients in the population who had no result for the parameter listed.
The intent-to-treat (ITT) population consisted of all patients, regardless of whether the patient received the study drug.
BSC=best supportive care; HbE=hemoglobin E.
Primary endpoint: RBC-TI ≥33% reduction from baseline in transfusion burden from Weeks 13 to 241
CI=confidence interval; RBC-TI=red blood cell transfusion independence.
4X
greater percentage of patients receiving REBLOZYL achieved the primary endpoint vs placebo
CI=confidence interval; RBC-TI=red blood cell transfusion independence.
KEY SECONDARY ENDPOINTS
Clinically meaningful reductions in transfusion burden were seen with REBLOZYL1
≥50%
Reduction in transfusion burden from baseline of at least 2 units from Weeks 13 to 24
≥33%
Reduction in transfusion burden from baseline of at least 2 units from Weeks 37 to 48
≥50%
Reduction in transfusion burden from baseline of at least 2 units from Weeks 37 to 48
CI=confidence interval.
Additional analyses
This analysis looks at the reduction in transfusion burden over a longer period of time, which may be important in adult patients with beta-thalassemia who require regular transfusions2,3
| Endpoint | REBLOZYL (n=224) |
Placebo (n=112) |
Risk difference (95% CI) |
|---|---|---|---|
| ≥33% reduction in transfusion burden, n (%) | 92 (41.1) | 3 (2.7) | 38.4 (31.3-45.5) |
| ≥50% reduction in transfusion burden, n (%) | 37 (16.5) | 1 (0.9) | 15.6 (10.5-20.8) |
Not restricted by a specified time period.
- The estimated transfusion burden reduction from baseline per patient based on the consecutive 24-week analysis was2:
- 6.55 RBC units per 24 weeks in patients with ≥33% reduction in RBC transfusion burden
- 8.27 RBC units per 24 weeks in patients with ≥50% reduction in RBC transfusion burden
Analysis limitations3
- Patients may have experienced multiple periods of response intermittently between periods without response over the 48-week assessment period
- These exploratory analyses should not be interpreted to determine treatment difference between arms in these select endpoints because of potential selection bias, insufficient sample size, and a higher probability of making a false positive finding
Additional analysis information
- The total (cumulative) duration of RBC transfusion burden reduction was defined as the sum of individual response periods (≥33% reduction from baseline in RBC transfusion burden of at least 2 units), with overlapping response periods excluded, over the entire 48-week study3
- All patients in both arms were eligible to receive BSC as needed: RBC transfusions; iron-chelating agents; use of antibiotic, antiviral, and antifungal therapy; and nutritional support1
Time from the first dose to response and total (cumulative) duration of response during any consecutive 12-week period3
| REBLOZYL (n=224) |
Placebo (n=112) |
|
|---|---|---|
| Patients with ≥33% transfusion burden reduction from baseline of at least 2 units, n (%) | 158 (70.5) | 33 (29.5) |
| Time to response, median (min, max) | 12 days (2 days, 360 days) |
107 days (2 days, 386 days) |
| Among responders, total cumulative duration of transfusion burden reduction, median (min, max) | 298 days (84 days, 631 days) |
171 days (84 days, 533 days) |
Analysis limitations3
- Patients may have experienced multiple periods of response intermittently between periods without response over the 48-week assessment period
- These exploratory analyses should not be interpreted to determine treatment difference between arms in these select endpoints because of potential selection bias, insufficient sample size, and a higher probability of making a false positive finding
Additional analysis information
- The total (cumulative) duration of RBC transfusion burden reduction was defined as the sum of individual response periods (≥33% reduction from baseline in RBC transfusion burden of at least 2 units), with overlapping response periods excluded, over the entire 48-week study3
- The time to response was defined as the first day of the start of the ≥33% reduction from baseline in RBC transfusion burden of at least 2 units during any rolling 12-week period3
- Each response period was a continuous period in which a subject had response during any 12-week interval3
- The median treatment exposure was approximately 64 weeks in both groups3
- All patients in both arms were eligible to receive BSC as needed: RBC transfusions; iron-chelating agents; use of antibiotic, antiviral, and antifungal therapy; and nutritional support1
PATIENT OUTCOMES: REBLOZYL EFFICACY
REBLOZYL provided substantial clinical benefit by reducing RBC transfusion burden1,2
The efficacy of REBLOZYL in patients with beta-thalassemia was demonstrated in the BELIEVE trial
Study design: REBLOZYL was studied in the pivotal phase 3 BELIEVE trial of 336 adult patients with beta-thalassemia requiring regular RBC transfusions (6-20 RBC units per 24 weeks) with no transfusion-free period greater than 35 days during that period who were randomized 2:1 to REBLOZYL (n=224) or placebo (n=112). In BELIEVE, REBLOZYL was administered subcutaneously once every 3 weeks as long as a reduction in transfusion requirement was observed or until unacceptable toxicity. Patients were able to receive BSC as needed, including: RBC transfusions; iron-chelating agents; use of antibiotic, antiviral, and antifungal therapy; and nutritional support. The exclusion criteria for this trial included HbS/beta-thalassemia or alpha-thalassemia; major organ damage (liver, heart, or lung disease, or renal insufficiency); recent deep vein thrombosis or stroke; or recent use of ESA, immunosuppressant, or hydroxyurea therapy. At 48 weeks, patients could cross over to REBLOZYL as part of the long-term follow-up.1,3
ESA=erythropoiesis-stimulating agent; HbS=hemoglobin S.
REBLOZYL was studied in the multicenter, randomized, double-blind , placebo-controlled, phase 3 BELIEVE trial
ESA=erythropoiesis-stimulating agent; HbS=hemoglobin S; SC=subcutaneous injection.
Primary endpoint1
- ≥33% reduction in RBC transfusion burden of at least 2 units from Weeks 13 to 24
Key secondary endpoints1
- ≥50% reduction in RBC transfusion burden from baseline of at least 2 units during Weeks 13 to 24
- ≥33% and ≥50% reduction in RBC transfusion burden to assess the durability of response from baseline of at least 2 units during Weeks 37 to 48
Additional endpoints2
- ≥33% or ≥50% reduction from baseline in RBC transfusion burden during any consecutive 24 weeks
- Duration of reduction in transfusion burden
- Time to erythroid response
All patients in the pivotal phase 3 BELIEVE trial received regular RBC transfusions1
Baseline characteristics of patients with beta-thalassemia in the pivotal phase 3 BELIEVE trial (ITT population)1,2
| Demographic and disease characteristics | REBLOZYL + BSC (n=224) |
Placebo + BSC (n=112) |
|---|---|---|
| Age, years | ||
| Median (min, max) | 30.0 (18, 66) | 30.0 (18, 59) |
| Baseline transfusion burden 12 weeks prior to randomization, units/12 weeks | ||
| Median (min, max) | 6.12 (3, 14) | 6.27 (3, 12) |
| Beta-thalassemia gene mutation grouping, n (%) | ||
| β0/β0 | 68 (30.4) | 35 (31.3) |
| Non-β0/β0 | 155 (69.2) | 77 (68.8) |
| Missinga | 1 (0.4) | 0 |
| Baseline serum ferritin level, μg/L | ||
| N | 220 | 111 |
| Median (min, max) | 1441.25 (88, 6400) | 1301.50 (136, 6400) |
| Splenectomy, n (%) | ||
| Yes | 129 (57.6) | 65 (58) |
| No | 95 (42.4) | 47 (42) |
| Sex, n (%) | ||
| Male | 92 (41.1) | 49 (43.7) |
| Female | 132 (58.9) | 63 (56.3) |
| Beta-thalassemia diagnosis, n (%) | ||
| β-thalassemia | 174 (77.7) | 83 (74.1) |
| HbE/β-thalassemia | 31 (13.8) | 21 (18.8) |
| β-thalassemia combined with α-thalassemia | 18 (8) | 8 (7.1) |
| Missinga | 1 (0.4) | 0 |
Missing category includes patients in the population who had no result for the parameter listed.
The intent-to-treat (ITT) population consisted of all patients, regardless of whether the patient received the study drug.
BSC=best supportive care; HbE=hemoglobin E.
Primary endpoint: RBC-TI ≥33% reduction from baseline in transfusion burden from Weeks 13 to 241
CI=confidence interval; RBC-TI=red blood cell transfusion independence.
4X
greater percentage of patients receiving REBLOZYL achieved the primary endpoint vs placebo
CI=confidence interval; RBC-TI=red blood cell transfusion independence.
KEY SECONDARY ENDPOINTS
Clinically meaningful reductions in transfusion burden were seen with REBLOZYL1
≥50%
Reduction in transfusion burden from baseline of at least 2 units from Weeks 13 to 24
≥33%
Reduction in transfusion burden from baseline of at least 2 units from Weeks 37 to 48
≥50%
Reduction in transfusion burden from baseline of at least 2 units from Weeks 37 to 48
CI=confidence interval.
Additional analyses
This analysis looks at the reduction in transfusion burden over a longer period of time, which may be important in adult patients with beta-thalassemia who require regular transfusions2,3
| Endpoint | REBLOZYL (n=224) |
Placebo (n=112) |
Risk difference (95% CI) |
|---|---|---|---|
| ≥33% reduction in transfusion burden, n (%) | 92 (41.1) | 3 (2.7) | 38.4 (31.3-45.5) |
| ≥50% reduction in transfusion burden, n (%) | 37 (16.5) | 1 (0.9) | 15.6 (10.5-20.8) |
Not restricted by a specified time period.
- The estimated transfusion burden reduction from baseline per patient based on the consecutive 24-week analysis was2:
- 6.55 RBC units per 24 weeks in patients with ≥33% reduction in RBC transfusion burden
- 8.27 RBC units per 24 weeks in patients with ≥50% reduction in RBC transfusion burden
Analysis limitations3
- Patients may have experienced multiple periods of response intermittently between periods without response over the 48-week assessment period
- These exploratory analyses should not be interpreted to determine treatment difference between arms in these select endpoints because of potential selection bias, insufficient sample size, and a higher probability of making a false positive finding
Additional analysis information
- The total (cumulative) duration of RBC transfusion burden reduction was defined as the sum of individual response periods (≥33% reduction from baseline in RBC transfusion burden of at least 2 units), with overlapping response periods excluded, over the entire 48-week study3
- All patients in both arms were eligible to receive BSC as needed: RBC transfusions; iron-chelating agents; use of antibiotic, antiviral, and antifungal therapy; and nutritional support1
Time from the first dose to response and total (cumulative) duration of response during any consecutive 12-week period3
| REBLOZYL (n=224) |
Placebo (n=112) |
|
|---|---|---|
| Patients with ≥33% transfusion burden reduction from baseline of at least 2 units, n (%) | 158 (70.5) | 33 (29.5) |
| Time to response, median (min, max) | 12 days (2 days, 360 days) |
107 days (2 days, 386 days) |
| Among responders, total cumulative duration of transfusion burden reduction, median (min, max) | 298 days (84 days, 631 days) |
171 days (84 days, 533 days) |
Analysis limitations3
- Patients may have experienced multiple periods of response intermittently between periods without response over the 48-week assessment period
- These exploratory analyses should not be interpreted to determine treatment difference between arms in these select endpoints because of potential selection bias, insufficient sample size, and a higher probability of making a false positive finding
Additional analysis information
- The total (cumulative) duration of RBC transfusion burden reduction was defined as the sum of individual response periods (≥33% reduction from baseline in RBC transfusion burden of at least 2 units), with overlapping response periods excluded, over the entire 48-week study3
- The time to response was defined as the first day of the start of the ≥33% reduction from baseline in RBC transfusion burden of at least 2 units during any rolling 12-week period3
- Each response period was a continuous period in which a subject had response during any 12-week interval3
- The median treatment exposure was approximately 64 weeks in both groups3
- All patients in both arms were eligible to receive BSC as needed: RBC transfusions; iron-chelating agents; use of antibiotic, antiviral, and antifungal therapy; and nutritional support1