Evidence map›Paper›PMID 41285724›Full record

ArticleBlood cancer journal2025

European LeukemiaNet criteria for safety and efficacy evaluation of new drugs for myelofibrosis.

Monia Marchetti, Alessandro Maria Vannucchi, John O Mascarenhas, Alberto Alvarez-Larran, Prithviraj Bose, Robert Peter Gale, Claire Harrison, Rűdiger Hehlmann, Jean-Jacques Kiladjian, Steffen Koschmieder and 5 more

Abstract read
In one paragraph

Article in Blood cancer journal, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Review
  2. Article
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

15 authors.

Monia MarchettiHematology and Transplant Unit, University Hospital SS Antonio e Biagio e Cesare Arrigo, Alessandria, Italy. monia.marchetti@uniupo.it.ORCID 0000-0001-7615-0572
Alessandro Maria Vannucchi *Dept Hematology, University of Florence, Azienda Ospedaliera Universitaria Careggi, Florence, Italy.ORCID 0000-0001-5755-0730
John O Mascarenhas *Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.ORCID 0000-0002-8400-0483
Alberto Alvarez-LarranHematology Department, Hospital Clinic, Barcelona, Spain.ORCID 0000-0001-6387-4619
Prithviraj BoseDepartment of Leukemia, University of Texas MD Anderson Cancer Center, 1515 Holcombe Blvd, Houston, TX, USA.ORCID 0000-0002-4343-5712
Robert Peter GaleImperial College London, London, UK.ORCID 0000-0002-9156-1676
Claire HarrisonDepartment of Clinical Hematology, Guy's and St Thomas' NHS Foundation Trust, London, UK.ORCID 0000-0002-3212-920X
Rűdiger HehlmannELN Foundation, Weinheim, Germany.
Jean-Jacques KiladjianHospital Saint Louis and University Paris Cité, Paris, France.
Steffen KoschmiederDepartment of Hematology, Oncology, Hemostaseology, and Stem Cell Transplantation (Dept. Of Medicine IV) University of Aachen, Aachen, Germany.ORCID 0000-0002-1011-8171
Ruben MesaWake Forest University School of Medicine, Winston-Salem, NC, USA.
Francesco PassamontiUniversità degli Studi di Milano, Fondazione IRCSS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Alessandro RambaldiDepartment of Oncology-Hematology, Azienda Socio-Sanitaria Territoriale Papa Giovanni XXIII, Bergamo, Italy.ORCID 0000-0002-3739-7502
Ayalew TefferiDivision of Hematology, Mayo Clinic, Rochester, MN, USA.ORCID 0000-0003-4605-3821
Tiziano BarbuiFROM Foundation ASST Papa Giovanni XXIII, Bergamo, Italy.ORCID 0000-0003-2747-6327

Funding

Tissue BankP01CA108671 · NCI · UNIVERSITY OF ILLINOIS AT CHICAGO · PI Ronald Hoffman · 2006 to 2026
$82.0M
NCI NIH HHS P01 CA108671
6 · The paper itself

Abstract

Most myelofibrosis (MF) drug approvals by Health Authorities are based on 1 or 2 outcome measures such as spleen volume reduction and/or improved quality of life. This 1-2 dimensional approach fails to capture the clinical complexity of MF. A European LeukemiaNet (ELN) task force developed a composite endpoint that allows multiple outcomes to be simultaneously measured. The panel used the Desirability Of Outcome Ranking (DOOR) method to assign 25 outcomes into 4 quartiles based on a multi-dimensional desirability score and build a 5-layer composite outcome named Desirability of Myelofibrosis Outcomes (DEMYO). Outcome operational definitions were tested by Delphi consensus, and correlations with survival were validated by a literature review. The outcomes assigned to the first two quartiles were negatively correlated with leukemia-free survival and survival. DEMYO comprehensively captures relevant MF clinical features and meaningful endpoints. Despite DEMYO requiring validation based on available trial data, it is expected to improve the quality of Health Authority approvals for new drugs for MF.

Indexed as

Primary MyelofibrosisEuropeHumansQuality of LifeTreatment Outcome

Identifiers

PMID41285724
PMCPMC12645036

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

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.