trialsTrialPositive

MEDALIST

MEDALIST showed that luspatercept let a substantial share of people with ring sideroblast myelodysplastic syndromes stop needing blood transfusions after erythropoietin had failed, which earned the drug its first approval.

MEDALIST randomised 229 patients with very low-, low- or intermediate-risk myelodysplastic syndromes with ring sideroblasts, regular red cell transfusion needs and prior failure of, or unsuitability for, erythropoiesis-stimulating agents to luspatercept or placebo every three weeks. The primary endpoint was red cell transfusion independence for eight weeks or longer in the first 24 weeks.

Significantly more patients on luspatercept became transfusion independent, many for long periods, with modest toxicity. The drug was approved in the United States in April 2020 for this population, the first new agent for lower-risk myelodysplastic anaemia in over a decade, and COMMANDS then took it into first-line use.

Setting

Transfusion-dependent lower-risk myelodysplastic syndromes with ring sideroblasts after failure of or ineligibility for erythropoiesis-stimulating agents: luspatercept against placebo

Phase

Phase 3

Sponsor

Celgene (Bristol Myers Squibb)

Registry

NCT02631070

Headline result

Luspatercept produced transfusion independence for eight weeks or longer in significantly more patients than placebo; approved in April 2020.

Started

2016-02-09

Reported

2020

Enrolled

229 enrolled

Outcomes

3top

In plain words

What these results mean for people, not percentages

229 enrolled

Transfusion independence >=8 weeks, weeks 1-24primaryother endpoint

38 vs 13 out of 100 reached this endpoint with Luspatercept compared with Placebo; 25 more per 100.
Roughly one extra person helped for every 4 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.

Transfusion independence >=12 weeks, weeks 1-24other endpoint

28 vs 8 out of 100 reached this endpoint with Luspatercept compared with Placebo; 20 more per 100.
Roughly one extra person helped for every 5 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.

Transfusion independence >=12 weeks, weeks 1-48other endpoint

33 vs 12 out of 100 reached this endpoint with Luspatercept compared with Placebo; 21 more per 100.
Roughly one extra person helped for every 5 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.

Caveats

The p-value (<0.001) says a difference this large would rarely happen by chance; it does not say how large or how useful the difference is.
This endpoint is not one of the standard survival or response measures; read it alongside the trial's primary result.
These results apply to the people the trial enrolled: Transfusion-dependent lower-risk myelodysplastic syndromes with ring sideroblasts after failure of or ineligibility for erythropoiesis-stimulating agents: luspatercept against placebo. People in a different situation may not see the same effect.

Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.

229 enrolled.

Transfusion independence >=8 weeks, weeks 1-24primary

· p <0.001

Luspatercept38 of 100

n = 153

Placebo13 of 100

n = 76

Download

Source

Transfusion independence >=12 weeks, weeks 1-24

· p <0.001

Luspatercept28 of 100

n = 153

Placebo8 of 100

n = 76

Download

Source

Transfusion independence >=12 weeks, weeks 1-48

· p <0.001

Luspatercept33 of 100

n = 153

Placebo12 of 100

n = 76

Download

Source

Endpoint	Arm	n	Value	HR (95% CI)	p	Source
Transfusion independence >=8 weeks, weeks 1-24primary	Luspatercept	153	38%	-	<0.001	link
Transfusion independence >=8 weeks, weeks 1-24primary	Placebo	76	13%	-	<0.001	link
Transfusion independence >=12 weeks, weeks 1-24	Luspatercept	153	28%	-	<0.001	link
Transfusion independence >=12 weeks, weeks 1-24	Placebo	76	8%	-	<0.001	link
Transfusion independence >=12 weeks, weeks 1-48	Luspatercept	153	33%	-	<0.001	link
Transfusion independence >=12 weeks, weeks 1-48	Placebo	76	12%	-	<0.001	link

Connected

6top

Trial
COMMANDS
Shares Luspatercept, Lower-risk myelodysplastic syndromes, Transfusion support and anaemia management, Myelodysplastic syndromes / neoplasms (MDS) and the tag subtype-trials.
Trial
AZA-001
Shares Pierre Fenaux, Myelodysplastic syndromes / neoplasms (MDS), Bristol Myers Squibb and the tag subtype-trials.
Trial
IMerge
Shares Lower-risk myelodysplastic syndromes, Myelodysplastic syndromes / neoplasms (MDS) and the tag subtype-trials.
Trial
ATTRACTION-2
Shares Bristol Myers Squibb and the tag subtype-trials.
Trial
CheckMate 025
Shares Bristol Myers Squibb and the tag subtype-trials.
Trial
DASISION
Shares Bristol Myers Squibb and the tag subtype-trials.
Trial
VERONA
Shares Myelodysplastic syndromes / neoplasms (MDS) and the tag subtype-trials.
Trial
CheckMate 358
Shares Bristol Myers Squibb and the tag subtype-trials.

cancers

Lower-risk myelodysplastic syndromes Myelodysplastic syndromes / neoplasms (MDS)

technologies

Transfusion support and anaemia management

drugs

ApprovedErythroid maturation agent (activin receptor IIB ligand trap, Fc fusion)

Luspatercept · Reblozyl

companies

Bristol Myers Squibb

people

Pierre Fenaux

Evidence strength70/100 · Solid

How much and what kind of evidence, not how big the benefit. Click for the breakdown.

Phase 3+30
229 enrolled+12
Primary endpoint: surrogate (response, pCR, detection)+8
Result: positive+15
No independent replication recorded0
A linked product is approved+5

Formula disclosed on the evidence page.

ProvenanceLast edited 2026-09-22 · Jude Gomila · Wave 2 corpus-only joins applied · diff · file

Sources & links

ClinicalTrials.gov NCT02631070

MEDALIST

Setting

Transfusion-dependent lower-risk myelodysplastic syndromes with ring sideroblasts after failure of or ineligibility for erythropoiesis-stimulating agents: luspatercept against placebo

Phase

Phase 3

Sponsor

Celgene (Bristol Myers Squibb)

Registry

NCT02631070

Headline result

Luspatercept produced transfusion independence for eight weeks or longer in significantly more patients than placebo; approved in April 2020.

Started

2016-02-09

Reported

2020

Enrolled

229 enrolled

Outcomes

3top

In plain words

What these results mean for people, not percentages

229 enrolled

Transfusion independence >=8 weeks, weeks 1-24primaryother endpoint

38 vs 13 out of 100 reached this endpoint with Luspatercept compared with Placebo; 25 more per 100.
Roughly one extra person helped for every 4 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.

Transfusion independence >=12 weeks, weeks 1-24other endpoint

28 vs 8 out of 100 reached this endpoint with Luspatercept compared with Placebo; 20 more per 100.
Roughly one extra person helped for every 5 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.

Transfusion independence >=12 weeks, weeks 1-48other endpoint

33 vs 12 out of 100 reached this endpoint with Luspatercept compared with Placebo; 21 more per 100.
Roughly one extra person helped for every 5 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.

Caveats

The p-value (<0.001) says a difference this large would rarely happen by chance; it does not say how large or how useful the difference is.
This endpoint is not one of the standard survival or response measures; read it alongside the trial's primary result.
These results apply to the people the trial enrolled: Transfusion-dependent lower-risk myelodysplastic syndromes with ring sideroblasts after failure of or ineligibility for erythropoiesis-stimulating agents: luspatercept against placebo. People in a different situation may not see the same effect.

Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.

229 enrolled.

Transfusion independence >=8 weeks, weeks 1-24primary

· p <0.001

Luspatercept38 of 100

n = 153

Placebo13 of 100

n = 76

Download

Source

Transfusion independence >=12 weeks, weeks 1-24

· p <0.001

Luspatercept28 of 100

n = 153

Placebo8 of 100

n = 76

Download

Source

Transfusion independence >=12 weeks, weeks 1-48

· p <0.001

Luspatercept33 of 100

n = 153

Placebo12 of 100

n = 76

Download

Source

Endpoint	Arm	n	Value	HR (95% CI)	p	Source
Transfusion independence >=8 weeks, weeks 1-24primary	Luspatercept	153	38%	-	<0.001	link
Transfusion independence >=8 weeks, weeks 1-24primary	Placebo	76	13%	-	<0.001	link
Transfusion independence >=12 weeks, weeks 1-24	Luspatercept	153	28%	-	<0.001	link
Transfusion independence >=12 weeks, weeks 1-24	Placebo	76	8%	-	<0.001	link
Transfusion independence >=12 weeks, weeks 1-48	Luspatercept	153	33%	-	<0.001	link
Transfusion independence >=12 weeks, weeks 1-48	Placebo	76	12%	-	<0.001	link

Connected

6top

Trial
COMMANDS
Shares Luspatercept, Lower-risk myelodysplastic syndromes, Transfusion support and anaemia management, Myelodysplastic syndromes / neoplasms (MDS) and the tag subtype-trials.
Trial
AZA-001
Shares Pierre Fenaux, Myelodysplastic syndromes / neoplasms (MDS), Bristol Myers Squibb and the tag subtype-trials.
Trial
IMerge
Shares Lower-risk myelodysplastic syndromes, Myelodysplastic syndromes / neoplasms (MDS) and the tag subtype-trials.
Trial
ATTRACTION-2
Shares Bristol Myers Squibb and the tag subtype-trials.
Trial
CheckMate 025
Shares Bristol Myers Squibb and the tag subtype-trials.
Trial
DASISION
Shares Bristol Myers Squibb and the tag subtype-trials.
Trial
VERONA
Shares Myelodysplastic syndromes / neoplasms (MDS) and the tag subtype-trials.
Trial
CheckMate 358
Shares Bristol Myers Squibb and the tag subtype-trials.

cancers

Lower-risk myelodysplastic syndromes Myelodysplastic syndromes / neoplasms (MDS)

technologies

Transfusion support and anaemia management

drugs

ApprovedErythroid maturation agent (activin receptor IIB ligand trap, Fc fusion)

Luspatercept · Reblozyl

companies

Bristol Myers Squibb

people

Pierre Fenaux

Evidence strength70/100 · Solid

How much and what kind of evidence, not how big the benefit. Click for the breakdown.

Phase 3+30
229 enrolled+12
Primary endpoint: surrogate (response, pCR, detection)+8
Result: positive+15
No independent replication recorded0
A linked product is approved+5

Formula disclosed on the evidence page.

ProvenanceLast edited 2026-09-22 · Jude Gomila · Wave 2 corpus-only joins applied · diff · file

Sources & links

ClinicalTrials.gov NCT02631070

MEDALIST

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