European LeukemiaNet criteria for safety and efficacy evaluation of new drugs for myelofibrosis
Year: 2025
Type: article
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.
Source: Blood Cancer Journal
Authors: 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, Ruben Mesa, Francesco Passamonti, Alessandro Rambaldi, Ayalew Tefferi, Tiziano Barbui
Institutions: Azienda Ospedaliera Nazionale SS. Antonio e Biagio e Cesare Arrigo, Azienda Ospedaliero-Universitaria Careggi, University of Florence, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, Hospital Clínic de Barcelona, The University of Texas MD Anderson Cancer Center, Wake Forest University, Imperial College London, Guy's Hospital, Guy's and St Thomas' NHS Foundation Trust, Université Paris Cité, Hôpital Saint-Louis, Universitätsklinikum Aachen, RWTH Aachen University, Wake Forest University Health Sciences, University of Milan, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Ospedale Papa Giovanni XXIII, Mayo Clinic
Language: English
Subfield: Genetics
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $5,290
Award: G803893432
OpenAlex ID: W4416591644