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
In brief
Complete responses tracked with longer survival in 1,268 higher-risk MDS patients
Across a retrospective database and a phase III trial cohort, patients with higher-risk myelodysplastic syndromes who achieved a complete response under the 2023 criteria had longer overall survival than those who did not. The finding supports the criteria's prognostic value, but response-associated survival does not show that a particular treatment improves outcomes.
- Journal
- Haematologica (Q1)
- Published
- 8 October 2026
- Study design
- Randomized controlled trial
- Evidence level
- Level 1, High (CEBM 1b)
- Authors
- Jan Philipp Bewersdorf, Tariq Kewan, Luca Lanino, Ondrej Blaha, Yiwen Liu, Jessica M Stempel, et al.
- PMID
- 42845057
- DOI
- 10.3324/haematol.2026.301314
Why clinicians should know about it
- Picked for Hematology (top studies of the week, 11 October 2026): Validation of IWG 2023 response criteria for high‑risk MDS
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.
Abstract as published, via PubMed.
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