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

Zeidan, AM; Platzbecker, U; Bewersdorf, JP; Stahl, M; Ades, L; Borate, U; Bowen, D; Buckstein, R; Brunner, A; Carraway, HE; Daver, N; Diez-Campelo, M; de Witte, T; DeZern, AE; Efficace, F; Garcia-Manero, G; Garcia, JS; Germing, U; Giagounidis, A; Griffiths, EA; Hasserjian, RP; Hellstrom-Lindberg, E; Iastrebner, M; Komrokji, R; Kulasekararaj, AG; Malcovati, L; Miyazaki, Y; Odenike, O; Santini, V; Sanz, G; Scheinberg, P; Stauder, R; van de Loosdrecht, AA; Wei, AH; Sekeres, MA; Fenaux, P

Author(s): Zeidan, AM; Platzbecker, U; Bewersdorf, JP; Stahl, M; Ades, L; Borate, U; Bowen, D; Buckstein, R; Brunner, A; Carraway, HE; Daver, N; Diez-Campelo, M; de Witte, T; DeZern, AE; Efficace, F; Garcia-Manero, G; Garcia, JS; Germing, U; Giagounidis, A; Griffiths, EA; Hasserjian, RP; Hellstrom-Lindberg, E; Iastrebner, M; Komrokji, R; Kulasekararaj, AG; Malcovati, L; Miyazaki, Y; Odenike, O; Santini, V; Sanz, G; Scheinberg, P; Stauder, R; van de Loosdrecht, AA; Wei, AH; Sekeres, MA; Fenaux, P;
Details: Publication Year 2023-04-27,Volume 141,Issue #17,Page 2047-2061
Journal Title: Blood
Publication Type: Guideline
Abstract: 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.
Publisher: American Society of Hematology
Keywords: Humans; Treatment Outcome; Consensus; *Myelodysplastic Syndromes/diagnosis/drug therapy; *Hematology; Outcome Assessment, Health Care
Department(s): Clinical Haematology
PubMed ID: 36724453
Publisher's Version: https://doi.org/10.1182/blood.2022018604
Terms of Use/Rights Notice: Refer to copyright notice on published article.

Creation Date: 2023-08-07 07:16:26

Last Modified: 2024-07-30 01:35:12