Evidence map›Paper›PMID 41140451›Full record

ArticleEClinicalMedicine2025

Omacetaxine and azacitidine for untreated patients with myelodysplastic syndromes and excess blasts: a phase I/II clinical trial.

Daniel A Pollyea, Brett M Stevens, Diana Abbott, Maria Amaya, Jonathan A Gutman, Andrew Kent, Christine McMahon, Marc Schwartz, Clayton Smith, Jessie Dell-Martin and 10 more

Registry-linked trialAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in EClinicalMedicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03564873 (Concomitant Omacetaxine Mepesuccinate and Azacitidine for Patients With Previously Untreated High Grade Myelodysplastic Syndromes), which is not on this map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–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.

NCT03564873 phase1 / phase2active not recruitingnot on this map

Concomitant Omacetaxine Mepesuccinate and Azacitidine for Patients With Previously Untreated High Grade Myelodysplastic Syndromes

TypeinterventionalSponsorUniversity of Colorado, DenverRan2018 to 2027Enrolled28ConditionsHigh Grade Myelodysplastic SyndromesArmsOmacetaxine, Azacitidine
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

20 authors.

Daniel A PollyeaDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Brett M StevensDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Diana AbbottDepartment of Biostatistics and Informatics, Center for Innovative Design and Analysis, University of Colorado, Aurora, CO, USA.
Maria AmayaDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Jonathan A GutmanDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Andrew KentDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Christine McMahonDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Marc SchwartzDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Clayton SmithDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Jessie Dell-MartinDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Connor SohalskiDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Alexandra EllisDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Mary HaagDepartment of Pathology, University of Colorado, Aurora, CO, USA.
Jeffrey SchowinskyDepartment of Pathology, University of Colorado, Aurora, CO, USA.
Zenggang PanDepartment of Pathology, University of Colorado, Aurora, CO, USA.
Sweta B PatelDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Monica RansomDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Austin GillenDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Craig T JordanDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.
Eric M PietrasDivision of Hematology, Department of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.

Funding

Metabolic targeting of heterogenous myelodysplastic syndrome stem cellsR01CA286717 · NCI · UNIVERSITY OF COLORADO DENVER · PI Eric M Pietras, Brett M Stevens · 2023 to 2026
$1.7M
NCI NIH HHS R01 CA286717
6 · The paper itself

Abstract

Background: Patients with myelodysplastic syndromes (MDS) with excess blasts (MDS-EB) have poor long-term outcomes. Our preclinical studies showed MDS-EB stem cells are dependent upon protein synthesis. We designed a phase 1/2 clinical trial to examine the safety/efficacy of the protein synthesis inhibitor omacetaxine mepesuccinate (oma) with the hypomethylating agent (HMA) azacitidine (aza) for patients with untreated MDS-EB. Methods: Enrollment occurred from September 2018 to March 2024 and the study was registered at clinicaltrials.gov (NCT03564873). The phase 1 primary endpoint was to determine the maximum tolerated dose (MTD) and the phase 2 primary endpoint was to determine the overall response rate. Aza 75 mg/m Findings: The MTD of oma was 0.5 mg/m Interpretation: The MTD of oma in MDS-EB has been established. Responses, including CRs, occurred rapidly. This therapeutic combination, conceived based on data that it targets the malignant stem cell population, could be further studied for patients with MDS-EB but high toxicity needs to be taken into account. Funding: HYPERLINCI, Edward P. Evans Foundation, Leukemia and Lymphoma Society Career Development Program, VA Merit, V-Foundation.

Indexed as

AzacitidineClinical trialMyelodysplastic syndromesOmacetaxinePhase 1/2

Identifiers

PMID41140451
PMCPMC12547833

What Socratic holds

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LicenceCC BY-NC-ND
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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 Socratic graph.