Evidence map›Paper›PMID 33687432›Full record

ArticleBlood advances2021

Development of luspatercept to treat ineffective erythropoiesis.

Anne Sophie Kubasch, Pierre Fenaux, Uwe Platzbecker

2 registry-linked trialsOpen access · goldAbstract read
In one paragraph

Article in Blood advances, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 48 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
48citing papers in PubMed, 2 pooled it
7.7field-weighted citation impact, top 2% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT06006949 phase4unknown statusnot on this mapstarted 2023, after this paper: background citation

Roxadustat Combined With Luspatercept Versus Luspatercept Monotherapy in the Treatment of Refractory Myelodysplastic Syndrome With Ring Sideroblasts (MDS-RS): A Prospective Randomized Controlled Study

TypeinterventionalSponsorPeking Union Medical College HospitalRan2023 to 2025Enrolled62ConditionsMyelodysplastic SyndromesArmsRoxadustat, Luspatercept
NCT06424639 phase4withdrawnnot on this mapstarted 2024, after this paper: background citation

A Randomized, Controlled Trial Comparing the Safety and Efficacy of Luspatercept Plus Cyclosporine Versus Cyclosporine Alone for the Treatment of Newly Diagnosed Non-transfusion-dependent Non-severe Aplastic Anemia (NSAA)

TypeinterventionalSponsorPeking Union Medical College HospitalRan2024 to 2025Enrolled0ConditionsAplastic AnemiaArmsLuspatercept, cyclosporine
3 · Its place in the literature

Who cites it

48 citing papers in PubMed, 2 syntheses or guidelines pooled it, 62 citations in OpenAlex.

  1. Pooled it
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  6. Long-Term Efficacy and Safety of Luspatercept for Anemia Treatment in Patients With Lower-Risk Myelodysplastic Syndromes: The Phase II PACE-MDS Study.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2022
    Trial
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  8. Article
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  13. Molecular and therapeutic frontiers in anemia therapy.The Journal of clinical investigation · 2026
    Review
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  19. Hereditary disorders of ineffective erythropoiesis.Blood cells, molecules & diseases · 2025
    Review
  20. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

3 authors at 2 institutions in 2 countries.

Anne Sophie KubaschDepartment of Hematology, Cellular Therapy and Hemostaseology, University Hospital Leipzig, Leipzig, Germany.
Pierre FenauxThe European Myelodysplastic Syndromes Cooperative Group (EMSCO), Leipzig, Germany.
Uwe PlatzbeckerDepartment of Hematology, Cellular Therapy and Hemostaseology, University Hospital Leipzig, Leipzig, Germany.
University Hospital Leipzig · DEGroupe Francophone des Myélodysplasies · FR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Luspatercept (Reblozyl) was recently approved for treating patients with transfusion-dependent lower-risk myelodysplastic syndrome (MDS) with ring sideroblasts (RS) and/or SF3B1 mutation who were not eligible for erythropoiesis-stimulating agents (ESAs) or patients for whom those agents failed. Luspatercept acts as an activin receptor type IIB fusion protein ligand trap that targets the altered transforming growth factor beta pathway in MDS, which is associated with impaired terminal erythroid maturation. Treatment with luspatercept results in decreased SMAD signaling, which enables erythroid maturation by means of late-stage erythroblast differentiation and thus improves anemia. ESAs, the current standard first-line therapeutic option for anemic lower-risk patients with MDS, also improve red cell parameters mainly by expanding proliferation of early erythroid progenitor cells. However, erythropoietin (EPO) and its receptor (EPO-R) are also required for survival of late-stage definitive erythroid cells, and they play an essential role in promoting proliferation, survival, and appropriate timing of terminal maturation of primitive erythroid precursors. Thus, luspatercept joins the mechanism of ESAs in promoting erythroid maturation. Especially in the subgroup of MDS patients with RS, luspatercept showed high clinical activity for the treatment of anemia in the phase 2 (PACE-MDS) trial and subsequently in the phase 3 (MEDALIST) trial, which resulted in approval by both the US Food and Drug Administration and the European Medicines Agency in April 2020. Additional studies are needed to better understand the mechanism of action and pharmacodynamics of this novel agent in MDS.

Indexed as

ErythropoiesisMyelodysplastic SyndromesActivin Receptors, Type IIHumansImmunoglobulin Fc FragmentsRecombinant Fusion ProteinsUnited StatesActivin Receptors, Type IIImmunoglobulin Fc FragmentsluspaterceptRecombinant Fusion Proteins

Identifiers

PMID33687432
PMCPMC7948289
OpenAlexW3134802607

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.