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
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.
Authors
- Vasiliki Pappa (ORCID: https://orcid.org/0000-0003-0421-9424)
- Kamal Hosni Al-Rabi (ORCID: https://orcid.org/0009-0007-7492-6374)
- Samuel Urrutia (ORCID: https://orcid.org/0000-0003-4634-2279)
- Lionel Adès (ORCID: https://orcid.org/0000-0002-9020-8766)
- Aref Al‐Kali (ORCID: https://orcid.org/0000-0002-0824-3715)
- Andrew Mark Brunner (ORCID: https://orcid.org/0000-0002-2169-4115)
- Rami S. Komrokji (ORCID: https://orcid.org/0000-0002-1876-5269)
- Seishi Ogawa (ORCID: https://orcid.org/0000-0002-7778-5374)
- Aram Bidikian (ORCID: https://orcid.org/0000-0001-5856-5926)
- Yasmin Abaza (ORCID: https://orcid.org/0000-0002-9156-7912)
- Gail J. Roboz (ORCID: https://orcid.org/0000-0002-0384-3658)
- Ιoannis Kotsianidis (ORCID: https://orcid.org/0000-0001-7668-0186)
- Anoop Kumar Enjeti (ORCID: https://orcid.org/0000-0001-8069-090X)
- Hetty E. Carraway (ORCID: https://orcid.org/0000-0001-5241-3614)
- Hayley Hawkins
- David Valcárcel (ORCID: https://orcid.org/0000-0002-8747-078X)
- Claudia Haferlach (ORCID: https://orcid.org/0000-0002-6333-5049)
- Rahul S. Vedula (ORCID: https://orcid.org/0000-0003-0486-3981)
- David Andrew Sallman (ORCID: https://orcid.org/0000-0003-0504-8233)
- Angela Consagra (ORCID: https://orcid.org/0009-0008-2007-4783)
- Harrison Kwei Tsai (ORCID: https://orcid.org/0000-0003-1134-9331)
- Amy Elizabeth Dezern (ORCID: https://orcid.org/0000-0001-5225-4609)
- Eunice S. Wang (ORCID: https://orcid.org/0000-0002-8644-0968)
- Tariq Zuheir Kewan (ORCID: https://orcid.org/0000-0003-4651-5125)
- Joshua F. Zeidner (ORCID: https://orcid.org/0000-0002-9014-1514)
- Somedeb Ball (ORCID: https://orcid.org/0000-0002-1754-1204)
- Maria L. Amaya (ORCID: https://orcid.org/0000-0001-9503-5019)
- Torsten Haferlach (ORCID: https://orcid.org/0000-0003-0196-2837)
- H. Moses Murdock (ORCID: https://orcid.org/0000-0003-4098-8968)
- Jessica M. Stempel (ORCID: https://orcid.org/0000-0001-9162-0579)
- Zhuoer Xie (ORCID: https://orcid.org/0000-0003-1266-9804)
- Ondřej Bláha (ORCID: https://orcid.org/0000-0003-3665-3276)
- Michael Robert Savona (ORCID: https://orcid.org/0000-0003-3763-5504)
- Evan Chris Chen (ORCID: https://orcid.org/0000-0002-9218-215X)
- Lisa Pleyer (ORCID: https://orcid.org/0000-0002-2790-5067)
- Mikkael Aaron Sekeres (ORCID: https://orcid.org/0000-0003-2009-6524)
- Devendra Hiwase (ORCID: https://orcid.org/0000-0002-6666-3056)
- Namrata S. Chandhok (ORCID: https://orcid.org/0000-0002-7464-4509)
- Elizabeth A. Griffiths (ORCID: https://orcid.org/0000-0002-0288-8248)
- Benjamin Rolles (ORCID: https://orcid.org/0000-0002-7393-861X)
- María Díez‐Campelo (ORCID: https://orcid.org/0000-0002-1467-6779)
- Khalid Halahleh (ORCID: https://orcid.org/0000-0002-1161-9138)
- Yasuhito Nannya (ORCID: https://orcid.org/0000-0002-6526-0992)
- Eytan M. Stein (ORCID: https://orcid.org/0000-0001-8992-9390)
- Amer Methqal Zeidan (ORCID: https://orcid.org/0000-0001-7017-8160)
- Christine M. McMahon (ORCID: https://orcid.org/0000-0001-9745-9595)
- Pinkal M. Desai (ORCID: https://orcid.org/0000-0002-9816-7284)
- Talha Badar (ORCID: https://orcid.org/0000-0003-1548-918X)
- Robert Coleman Lindsley (ORCID: https://orcid.org/0000-0001-9822-806X)
- Yiwen Liu
- Jan Philipp Bewersdorf (ORCID: https://orcid.org/0000-0003-3352-0902)
- Jaroslaw Pawel Maciejewski (ORCID: https://orcid.org/0000-0002-6837-4346)
- Justin E. Grenet (ORCID: https://orcid.org/0000-0001-8177-4531)
- Luca Lanino (ORCID: https://orcid.org/0000-0003-2404-8829)
- Matteo Giovanni Della Porta (ORCID: https://orcid.org/0000-0002-6915-5970)
- Yazan F. Madanat (ORCID: https://orcid.org/0000-0001-9573-2690)
- Rory Michael Shallis (ORCID: https://orcid.org/0000-0002-8542-2944)
- Valeria Santini (ORCID: https://orcid.org/0000-0002-5439-2172)
- Amyah C. Harris
- Najla H. Al Ali
- Ted M. Getz (ORCID: https://orcid.org/0000-0001-9886-5624)
- Maximilian Stahl
- Jaclynn Campos
- Geoffrey L Uy
- Constantine Logothetis
Institutions
- Northwestern University (US)
- University of North Carolina at Chapel Hill (US)
- Hebron University (PS)
- Roswell Park Comprehensive Cancer Center (US)
- Cleveland Clinic (US)
- Royal Adelaide Hospital (AU)
- Mayo Clinic (US)
- Memorial Sloan Kettering Cancer Center (US)
- Humanitas University (IT)
- Harvard University (US)
- Johns Hopkins University (US)
- University of Miami (US)
- Democritus University of Thrace (GR)
- Tokyo Medical University (JP)
- Universidad de Salamanca (ES)
- National and Kapodistrian University of Athens (GR)
- Washington University in St. Louis (US)
- Université Paris Cité (FR)
- Paracelsus Medical University (AT)
- King Hussein Cancer Center (JO)
- Moffitt Cancer Center (US)
- Yale University (US)
- Massachusetts General Hospital (US)
- Munich Leukemia Laboratory (Germany) (DE)
- Assistance Publique – Hôpitaux de Paris (FR)
- Azienda Ospedaliero-Universitaria Careggi (IT)
- Dana-Farber Cancer Institute (US)
- Yale New Haven Health System (US)
- Instituto de Investigación Biomédica de Salamanca (ES)
- University General Hospital Attikon (GR)
- Mayo Clinic in Florida (US)
- Calvary Mater Newcastle Hospital (AU)
- Sylvester Comprehensive Cancer Center (US)
- IRCCS Humanitas Research Hospital (IT)
- Sidney Kimmel Comprehensive Cancer Center (US)
- Weill Cornell Medicine (US)
- Vanderbilt-Ingram Cancer Center
- UNC Lineberger Comprehensive Cancer Center
- Harold C. Simmons Comprehensive Cancer Center
- University of Florence (IT)
- The University of Tokyo (JP)
- The University of Texas Southwestern Medical Center (US)
- Vanderbilt University Medical Center (US)
Publication Details
- Journal
- Haematologica
- Published
- 2026-10-08
- DOI
- https://doi.org/10.3324/haematol.2026.301314
- Primary Topic
- Acute Myeloid Leukemia Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00