Validation of the International Working Group 2023 response criteria for higher risk myelodysplastic syndromes/neoplasms using the International Consortium for Myelodysplastic Syndromes VALIDATE database and a randomized phase III trial
Abstract
Response assessment criteria in patients with higher-risk myelodysplastic syndromes/neoplasms (HR-MDS) have been updated in the 2023 International Working Group (IWG) criteria. However, validation of these criteria has been limited to date. We analyzed overall survival (OS) by best response according to IWG 2023 criteria in two separate and independent cohorts of patients with HR-MDS who were treated with hypomethylating agents (HMA)-based therapies. The first dataset was the International Consortium for MDS (icMDS) VALIDATE retrospective database, and the second dataset was the cohort of HR-MDS patients treated in the randomized, phase III PANTHER trial of azacitidine + pevonedistat or placebo. A total of 1,268 patients (VALIDATE: n=1,047; PANTHER: n=221) were evaluated in two separate sets of analyses according to the source dataset. Patients who achieved composite CR (cCR) had improved OS compared to patients not achieving cCR in both datasets and in both univariate and multivariable analyses (VALIDATE: multivariable Hazard Ratio [HR]: 0.65; 95% CI: 0.55-0.76; p.
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Authors: Jan Philipp Bewersdorf, Tariq Zuheir Kewan, Luca Lanino, Ondřej Bláha, Yiwen Liu, Jessica M. Stempel, Najla H. Al Ali, Amy Elizabeth Dezern, Mikkael Aaron Sekeres, Geoffrey L Uy, Samuel Urrutia, Hetty E. Carraway, Pinkal M. Desai, Elizabeth A. Griffiths, Eytan M. Stein, Andrew Mark Brunner, Christine M. McMahon, Rory Michael Shallis, Joshua F. Zeidner, Michael Robert Savona, Hayley Hawkins, Namrata S. Chandhok, Constantine Logothetis, Aram Bidikian, Ted M. Getz, Gail J. Roboz, Benjamin Rolles, Eunice S. Wang, Amyah C. Harris, Maria L. Amaya, Somedeb Ball, Justin E. Grenet, Zhuoer Xie, Yazan F. Madanat, Yasmin Abaza, Talha Badar, Jaclynn Campos, Torsten Haferlach, Jaroslaw Pawel Maciejewski, David Andrew Sallman, Rahul S. Vedula, H. Moses Murdock, Harrison Kwei Tsai, Evan Chris Chen, Lionel Adès, Robert Coleman Lindsley, Anoop Kumar Enjeti, Kamal Hosni Al-Rabi, Khalid Halahleh, Devendra Hiwase, María Díez‐Campelo, David Valcárcel, Claudia Haferlach, Lisa Pleyer, Ιoannis Kotsianidis, Vasiliki Pappa, Valeria Santini, Angela Consagra, Aref Al‐Kali, Seishi Ogawa, Yasuhito Nannya, Matteo Giovanni Della Porta, Rami S. Komrokji, Maximilian Stahl, Amer Methqal Zeidan
Institutions: Northwestern University, Johns Hopkins University, Harvard University, University of North Carolina at Chapel Hill, Royal Adelaide Hospital, Hebron University, The University of Tokyo, University of Miami, Azienda Ospedaliero-Universitaria Careggi, Washington University in St. Louis, Yale University, Université Paris Cité, Sidney Kimmel Comprehensive Cancer Center, Universidad de Salamanca, Assistance Publique – Hôpitaux de Paris, University of Florence, UNC Lineberger Comprehensive Cancer Center, The University of Texas Southwestern Medical Center, Dana-Farber Cancer Institute, Massachusetts General Hospital, Humanitas University, IRCCS Humanitas Research Hospital, Memorial Sloan Kettering Cancer Center, Roswell Park Comprehensive Cancer Center, Mayo Clinic, Moffitt Cancer Center, Democritus University of Thrace, National and Kapodistrian University of Athens, Tokyo Medical University, Weill Cornell Medicine, University General Hospital Attikon, Vanderbilt University Medical Center, Cleveland Clinic, Paracelsus Medical University, Mayo Clinic in Florida, King Hussein Cancer Center, Instituto de Investigación Biomédica de Salamanca, Vanderbilt-Ingram Cancer Center, Sylvester Comprehensive Cancer Center, Yale New Haven Health System, Calvary Mater Newcastle Hospital, Munich Leukemia Laboratory (Germany), Harold C. Simmons Comprehensive Cancer Center