Evidence map›Paper›PMID 35011768›Full record

ArticleJournal of clinical medicine2021

Health-Related Quality of Life Outcomes in Patients with Myelodysplastic Syndromes with Ring Sideroblasts Treated with Luspatercept in the MEDALIST Phase 3 Trial.

Esther Natalie Oliva, Uwe Platzbecker, Guillermo Garcia-Manero, Ghulam J Mufti, Valeria Santini, Mikkael A Sekeres, Rami S Komrokji, Jeevan K Shetty, Derek Tang, Shien Guo and 7 more

Registry-linked trialAbstract read
In one paragraph

Article in Journal of clinical medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02631070 (A Phase 3, Double-blind, Randomized Study to Compare the Efficacy and Safety of Luspatercept), which is not on this map. Cited by 18 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed, 1 pooled it
–field-weighted citation impact
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.

NCT02631070 phase3completednot on this map

A Phase 3, Double-blind, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo for the Treatment of Anemia Due to the IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in Subjects With Ring Sideroblasts Who Require Red Blood Cell Transfusions.

TypeinterventionalSponsorCelgeneRan2016 to 2020Enrolled229ConditionsMyelodysplastic SyndromesArmsLuspatercept, Placebo
3 · Its place in the literature

Who cites it

18 citing papers in PubMed, 1 synthesis or guideline pooled it.

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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

17 authors.

Esther Natalie OlivaGrande Ospedale Metropolitano Bianchi Melacrino Morelli, 89124 Reggio Calabria, Italy.ORCID 0000-0001-9238-5734
Uwe PlatzbeckerMedical Clinic and Policlinic 1, Hematology and Cellular Therapy, Leipzig University Hospital, 04103 Leipzig, Germany.ORCID 0000-0003-1863-3239
Guillermo Garcia-ManeroThe University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Ghulam J MuftiDepartment of Haemato-Oncology, King's College London, London SE5 9RS, UK.
Valeria SantiniMDS Unit, Azienda Ospedaliero Universitaria Careggi, University of Florence, 50134 Florence, Italy.
Mikkael A SekeresSylvester Comprehensive Cancer Center, University of Miami, Miami, FL 33136, USA.
Rami S KomrokjiMoffitt Cancer Center, Tampa, FL 33612, USA.
Jeevan K ShettyBristol Myers Squibb, Princeton, NJ 08540, USA.
Derek TangBristol Myers Squibb, Princeton, NJ 08540, USA.
Shien GuoEvidera, Waltham, MA 02451, USA.
Weiqin LiaoEvidera, Waltham, MA 02451, USA.
George ZhangBristol Myers Squibb, Princeton, NJ 08540, USA.
Xianwei HaBristol Myers Squibb, Princeton, NJ 08540, USA.
Rodrigo ItoBristol Myers Squibb, Princeton, NJ 08540, USA.
Jennifer Lord-BessenBristol Myers Squibb, Princeton, NJ 08540, USA.
Jay T BackstromAcceleron Pharma, Cambridge, MA 02139, USA.
Pierre FenauxService d'Hématologie Séniors, Hôpital Saint-Louis, Assistance Publique des Hôpitaux de Paris (AP-HP), 75010 Paris, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Patients with myelodysplastic syndromes (MDS) often experience chronic anemia and long-term red blood cell transfusion dependence associated with significant burden on clinical and health-related quality of life (HRQoL) outcomes. In the MEDALIST trial (NCT02631070), luspatercept significantly reduced transfusion burden in patients with lower-risk MDS who had ring sideroblasts and were refractory to, intolerant to, or ineligible for prior treatment with erythropoiesis-stimulating agents. We evaluated the effect of luspatercept on HRQoL in patients enrolled in MEDALIST using the EORTC QLQ-C30 and the QOL-E questionnaire. Change in HRQoL was assessed every 6 weeks in patients receiving luspatercept with best supportive care (+ BSC) and placebo + BSC from baseline through week 25. No clinically meaningful within-group changes and between-group differences across all domains of the EORTC QLQ-C30 and QOL-E were observed. On one item of the QOL-E MDS-specific disturbances domain, patients treated with luspatercept reported marked improvements in their daily life owing to the reduced transfusion burden, relative to placebo. Taken together with previous reports of luspatercept + BSC reducing transfusion burden in patients from baseline through week 25 in MEDALIST, these results suggest luspatercept may offer a treatment option for patients that reduces transfusion burden while providing stability in HRQoL.

Indexed as

luspaterceptmyelodysplastic syndromesquality of lifetransfusion dependence

Identifiers

PMID35011768
PMCPMC8745777

What OpenQuestion holds

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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.