Consensus proposal for revised International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes

Author(s): Amer M. Zeidan1;Uwe Platzbecker2;Jan Philipp Bewersdorf3;Maximilian Stahl4;Lionel Adès5;Uma Borate6;David Bowen7;Rena Buckstein8;Andrew Brunner9;Hetty E. Carraway10;Naval Daver11;Maria Díez-Campelo12;Theo de Witte13;Amy E. DeZern14;Fabio Efficace15;Guillermo Garcia-Manero11;Jacqueline S. Garcia4;Ulrich Germing16;Aristoteles Giagounidis17;Elizabeth A. Griffiths18;Robert P. Hasserjian19;Eva Hellström-Lindberg20;Marcelo Iastrebner21;Rami Komrokji22;Austin G. Kulasekararaj23;Luca Malcovati24;Yasushi Miyazaki25;Olatoyosi Odenike26;Valeria Santini27;Guillermo Sanz28;Phillip Scheinberg29;Reinhard Stauder30;Arjan A. van de Loosdrecht31;Andrew H. Wei32;Mikkael A. Sekeres33;Pierre Fenaux5
Source: Blood (2023) 141 (17): 2047–2061
Maem Hussein MD

Dr. Maen Hussein's Thoughts

New criteria for response to therapy in high grade MDS, focuses on pt centered outcomes.


Myelodysplastic syndromes/myelodysplastic neoplasms (MDS) are associated with variable clinical presentations and outcomes. The initial response criteria developed by the International Working Group (IWG) in 2000 have been used in clinical practice, clinical trials, regulatory reviews, and drug labels. Although the IWG criteria were revised in 2006 and 2018 (the latter focusing on lower-risk disease), limitations persist in their application to higher-risk MDS (HR-MDS) and their ability to fully capture the clinical benefits of novel investigational drugs or serve as valid surrogates for longer-term clinical end points (eg, overall survival). Further, issues related to the ambiguity and practicality of some criteria lead to variability in interpretation and interobserver inconsistency in reporting results from the same sets of data. Thus, we convened an international panel of 36 MDS experts and used an established modified Delphi process to develop consensus recommendations for updated response criteria that would be more reflective of patient-centered and clinically relevant outcomes in HR-MDS. Among others, the IWG 2023 criteria include changes in the hemoglobin threshold for complete remission (CR), the introduction of CR with limited count recovery and CR with partial hematologic recovery as provisional response criteria, the elimination of marrow CR, and specific recommendations for the standardization of time-to-event end points and the derivation and reporting of responses. The updated criteria should lead to a better correlation between patient-centered outcomes and clinical trial results in an era of multiple emerging new agents with novel mechanisms of action.

Author Affiliations

1Section of Hematology, Department of Internal Medicine, Yale School of Medicine and Yale Cancer Center, Yale University, New Haven, CT2Leipzig University Hospital, Leipzig, Germany3Department of Medicine, Leukemia Service, Memorial Sloan Kettering Cancer Center, New York, NY4Department of Medical Oncology, Dana-Farber Cancer Institute and Harvard Medical School, Harvard University, Boston, MA5Service d’hématologie, Hôpital Saint-Louis (Assistance Publique Hôpitaux de Paris) and Université de Paris-Cité, Paris, France6Division of Hematology, Department of Internal Medicine, James Cancer Center, The Ohio State University, Columbus, OH7St James’s Institute of Oncology, Leeds, United Kingdom8Division of Oncology and Malignant Hematology, Odette Cancer Centre, Sunnybrook Health Sciences Centre, Toronto, Canada9Leukemia Program, Massachusetts General Hospital Cancer Center, Harvard Medical School, Harvard University, Boston, MA10Leukemia Program, Hematology and Medical Oncology, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH11Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX12Hematology Department, University Hospital of Salamanca, Institute for Biomedical Research of Salamanca, Universidad de Salamanca, Salamanca, Spain13Department of Tumor Immunology, Radboud Institute of Molecular Life Sciences, Radboud University Medical Center, Nijmegen, The Netherlands14Johns Hopkins Sidney Kimmel Comprehensive Cancer Centre, Baltimore, MD15Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy16Klinik für Hämatologie, Onkologie und klinische Immunologie, Universitätsklinikum Düsseldorf, Düsseldorf, Germany17St Mary’s Hospital, Düsseldorf, Germany18Roswell Park Comprehensive Cancer Center, Buffalo, NY19Department of Pathology, Massachusetts General Hospital, Boston, MA20Karolinska Institutet, Center for Hematology and Regenerative Medicine, Department of Medicine Huddinge, Karolinska University Hospital, Stockholm, Sweden21Sanatorio Universitario Sagrado Corazón, Ciudad Autónoma de Buenos Aires, Argentina22Department of Malignant Hematology, H. Lee Moffitt Cancer Center, Tampa, FL23Department of Haematological Medicine, King’s College Hospital, National Institute of Health Research/Wellcome King’s Clinical Research Facility and King’s College London, London, United Kingdom24Department of Hematology, University of Pavia & IRCCS S. Matteo Hospital Foundation, Pavia, Italy25Department of Hematology, Atomic Bomb Disease Institute, Nagasaki University Graduate School of Biomedical Sciences, Nagasaki, Japan26Leukemia Program, University of Chicago Medicine and University of Chicago Comprehensive Cancer Center, Chicago, IL27MDS Unit, AOUC, University of Florence, Florence, Italy28Hematology Department, Hospital Universitario y Politécnico La Fe, Valencia, Spain, Health Research Institute La Fe, Valencia, Spain; and CIBERONC, Instituto de Salud Carlos III, Madrid, Spain29Division of Hematology, Hospital A Beneficência Portuguesa, São Paulo, Brazil30Department of Internal Medicine V, Innsbruck Medical University, Innsbruck, Austria31Department of Hematology, Amsterdam University Medical Center, Vrije Universiteit Medical Center, Amsterdam Cancer Center, Amsterdam, The Netherlands32Peter MacCallum Cancer Centre, Royal Melbourne Hospital, University of Melbourne and Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia33Division of Hematology, Sylvester Comprehensive Cancer Center, Miami Miller School of Medicine, University of Miami, Miami, FL

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