Evidence map›Paper›PMID 36153475›Full record

Trial reportBMC cancer2022

The ABNL-MARRO 001 study: a phase 1-2 study of randomly allocated active myeloid target compound combinations in MDS/MPN overlap syndromes.

Tamara K Moyo, Jason H Mendler, Raphael Itzykson, Ashwin Kishtagari, Eric Solary, Adam C Seegmiller, Aaron T Gerds, Gregory D Ayers, Amy E Dezern, Aziz Nazha and 27 more

Registry-linked trialOpen access · goldAbstract readClinical Trial, Phase IClinical Trial, Phase IIRandomized Controlled Trial
In one paragraph

Trial report in BMC cancer, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04061421 (The ABNL-MARRO 001 Study), which is not on this map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
2.7field-weighted citation impact, top 8% 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.

NCT04061421 phase1 / phase2recruitingnot on this map

The ABNL-MARRO 001 Study: A Phase 1/2 Study of Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes.

TypeinterventionalSponsorMichael SavonaRan2021 to 2027Enrolled94ConditionsMDS/MPNArmsASTX727, Itacitinib, Ruxolitinib
3 · Its place in the literature

Who cites it

10 citing papers in PubMed, 17 citations in OpenAlex.

  1. Review
  2. Review
  3. Clinical management of CMML-State of the art.British journal of haematology · 2025
    Review
  4. Contemporary CMML Risk Stratification and Management.Current hematologic malignancy reports · 2025
    Review
  5. Review
  6. Review
  7. Article
  8. Atypical CML: diagnosis and treatment.Hematology. American Society of Hematology. Education Program · 2023
    Article
  9. Proposals for Clinical Trials in Chronic Myelomonocytic Leukemia.Current treatment options in oncology · 2023
    Review
  10. 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

37 authors at 20 institutions in 8 countries.

Tamara K MoyoVanderbilt University School of Medicine, Vanderbilt-Ingram Cancer Center, 2220 Pierce Avenue, Nashville, TN, 777 PRB, USA.
Jason H MendlerJames P. Wilmot Cancer Institute, University of Rochester Medical Center, Rochester, NY, USA.
Raphael ItzyksonParis Diderot University, Paris, France.
Ashwin KishtagariVanderbilt University School of Medicine, Vanderbilt-Ingram Cancer Center, 2220 Pierce Avenue, Nashville, TN, 777 PRB, USA.
Eric SolaryInstitut Gustave Roussy, Université Paris-Saclay, Villejuif, France.
Adam C SeegmillerVanderbilt University School of Medicine, Vanderbilt-Ingram Cancer Center, 2220 Pierce Avenue, Nashville, TN, 777 PRB, USA.
Aaron T GerdsCleveland Clinic, Cleveland, OH, USA.
Gregory D AyersVanderbilt University School of Medicine, Vanderbilt-Ingram Cancer Center, 2220 Pierce Avenue, Nashville, TN, 777 PRB, USA.
Amy E DezernJohns Hopkins University, Baltimore, MD, USA.
Aziz NazhaCleveland Clinic, Cleveland, OH, USA.
Peter ValentDepartment of Internal Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.
Arjan A van de LoosdrechtVU University Medical Center, Amsterdam, Netherlands.
Francesco OnidaFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy.
Lisa PleyerThird Medical Department With Hematology, Medical Oncology, Rheumatology and Infectiology, Paracelsus Medical University, Salzburg, Austria.
Blanca Xicoy CiriciInstitut Català d'Oncologia-Hospital Germans Trias i Pujol, Josep Carreras Leukemia Research Institute, Universitat Autònoma de Barcelona, Bellaterr, Spain.
Raoul TibesMayo Clinic, Scottsdale, AZ, USA.
Klaus GeisslerSigmund Freud University, Vienna, Austria.
Rami S KomrokjiH. Lee Moffitt Cancer Center, Tampa, FL, USA.
Jing ZhangUniversity of Wisconsin-Madison, Madison, WI, USA.
Ulrich GermingDepartment of Hematology, Oncology, and Clinical Immunology, University of Duesseldorf, Duesseldorf, Germany.
David P SteensmaDana-Farber Cancer Institute, Boston, MA, USA.
Daniel H WisemanUniversity of Manchester, Manchester, UK.
Michael PfeilstöeckerHanusch Hospital and Ludwig Boltzmann Institute for Hematology and Oncology, Vienna, Austria.
Chiara ElenaUniversity of Pavia, Pavia, Italy.
Nicholas C P CrossSchool of Medicine, University of Southampton, Southampton, UK.
Jean-Jacques KiladjianUniversité de Paris, APHP, Hôpital Saint-Louis, Centre d'Investigations Cliniques, INSERM CIC 1427, Paris, France.
Michael LuebbertUniversity of Freiburg, Freiburg, Germany.
Ruben A MesaMays Cancer Center at UT Health San Antonio MD Anderson, San Antonio, TX, USA.
Guillermo Montalban-BravoMD Anderson Cancer Center, Houston, TX, USA.
Guillermo F SanzHospital Universitario Y Politécnico La Fe, Valencia, Spain.
Uwe PlatzbeckerUniversity Hospital Leipzig, Leipzig, Germany.
Mrinal M PatnaikMayo Clinic, Rochester, MN, USA.
Eric PadronH. Lee Moffitt Cancer Center, Tampa, FL, USA.
Valeria SantiniMDS UNIT, University of Florence, HematologyFlorence, Italy.
Pierre FenauxParis Diderot University, Paris, France.
Michael R SavonaVanderbilt University School of Medicine, Vanderbilt-Ingram Cancer Center, 2220 Pierce Avenue, Nashville, TN, 777 PRB, USA. michael.savona@vanderbilt.edu.ORCID http://orcid.org/0000-0003-3763-5504
MDS/MPN International Working Group
Vanderbilt University · USCleveland Clinic · USDélégation Paris 7 · FRMayo Clinic in Arizona · USMoffitt Cancer Center · USThe University of Texas MD Anderson Cancer Center · USAmsterdam UMC Location Vrije Universiteit Amsterdam · NLDana-Farber Cancer Institute · USHanusch Hospital · ATHeinrich Heine University Düsseldorf · DEHospital Universitari i Politècnic La Fe · ESInserm · FRJohns Hopkins University · USMedical University of Vienna · ATParacelsus Medical University · ATSigmund Freud Privatuniversität Wien · ATUniversitat Autònoma de Barcelona · ESUniversité Paris-Saclay · FRUniversity Hospital Leipzig · DEUniversity of Florence · IT

Funding

The role of TCOF1 in germline predisposition to and in leukemic transformation of chronic myelomonocytic leukemiaR01CA152108 · NCI · UNIVERSITY OF WISCONSIN-MADISON · PI Jing Zhang · 2011 to 2026
$5.4M
NCI NIH HHS R01 CA152108
6 · The paper itself

Abstract

backgroundMyelodysplastic/myeloproliferative neoplasms (MDS/MPN) comprise several rare hematologic malignancies with shared concomitant dysplastic and proliferative clinicopathologic features of bone marrow failure and propensity of acute leukemic transformation, and have significant impact on patient quality of life. The only approved disease-modifying therapies for any of the MDS/MPN are DNA methyltransferase inhibitors (DNMTi) for patients with dysplastic CMML, and still, outcomes are generally poor, making this an important area of unmet clinical need. Due to both the rarity and the heterogeneous nature of MDS/MPN, they have been challenging to study in dedicated prospective studies. Thus, refining first-line treatment strategies has been difficult, and optimal salvage treatments following DNMTi failure have also not been rigorously studied. ABNL-MARRO (A Basket study of Novel therapy for untreated MDS/MPN and Relapsed/Refractory Overlap Syndromes) is an international cooperation that leverages the expertise of the MDS/MPN International Working Group (IWG) and provides the framework for collaborative studies to advance treatment of MDS/MPN and to explore clinical and pathologic markers of disease severity, prognosis, and treatment response.

methodsABNL MARRO 001 (AM-001) is an open label, randomly allocated phase 1/2 study that will test novel treatment combinations in MDS/MPNs, beginning with the novel targeted agent itacitinib, a selective JAK1 inhibitor, combined with ASTX727, a fixed dose oral combination of the DNMTi decitabine and the cytidine deaminase inhibitor cedazuridine to improve decitabine bioavailability. DISCUSSION: Beyond the primary objectives of the study to evaluate the safety and efficacy of novel treatment combinations in MDS/MPN, the study will (i) Establish the ABNL MARRO infrastructure for future prospective studies, (ii) Forge innovative scientific research that will improve our understanding of pathogenetic mechanisms of disease, and (iii) Inform the clinical application of diagnostic criteria, risk stratification and prognostication tools, as well as response assessments in this heterogeneous patient population.

trial registrationThis trial was registered with ClinicalTrials.gov on August 19, 2019 (Registration No. NCT04061421).

Indexed as

Myelodysplastic-Myeloproliferative DiseasesQuality of LifeAcetonitrilesCytidine DeaminaseDecitabineDNAHumansMethyltransferasesProspective StudiesPyrazolesPyrimidinesPyrrolesSyndromeAcetonitrilesCytidine DeaminaseDecitabineDNAitacitinibMethyltransferasesPyrazolesPyrimidinesPyrrolesABNL MARROASTX727ItacitinibMDS/MPNPhase 1b/2

Identifiers

PMID36153475
PMCPMC9509596
OpenAlexW4296970967

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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.