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<p>Monotherapy with hypomethylating agents (HMA) remains the standard of care for patients with higher-risk myelodysplastic neoplasms (HR-MDS). Recently, the randomized phase III VERONA study evaluating azacitidine plus venetoclax (VEN) versus azacitidine plus placebo in newly diagnosed HR-MDS showed no difference in overall survival (OS) between the two arms. However, whether the addition of VEN to HMA improves outcomes among subsets of patients with HR-MDS remains debated. We analyzed 1907 patients with HR-MDS from 31 centers in 9 countries who were treated with HMA monotherapy or HMA/VEN in the frontline setting (HMA monotherapy: <i>n</i> = 1773; HMA/VEN: <i>n</i> = 134). Responses were assessed centrally by two investigators using the IWG 2023 response criteria. Addition of VEN improved composite complete remission (cCR) rates (48.8% vs. 27.7%; <i>p</i> &lt; 0.001) but not CR rates (17.1% vs. 11.7%; <i>p</i> = 0.16). In multivariable logistic regression analysis, cCR remained favorable for HMA/VEN vs. HMA monotherapy (Odds Ratio [OR]: 2.49; 95% CI: 1.56-3.96; <i>p</i> &lt; 0.001). However, we did not observe a statistically significant difference in OS for HMA/VEN vs. HMA monotherapy (Hazard Ratio [HR]: 0.83; 95% CI: 0.64-1.07; <i>p</i> = 0.15). In subgroup analyses, patients with <i>TP53</i> wild-type disease (HR: 0.47; 95% CI: 0.29-0.74; <i>p</i> = 0.002) had a significant improvement in OS and those with ≥10% bone marrow blasts (HR: 0.73; 95% CI: 0.53-1.01; <i>p</i> = 0.06) had a trend towards OS benefit with HMA/VEN.</p>

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Outcomes of patients with higher-risk myelodysplastic syndromes/neoplasms treated with hypomethylating agents + venetoclax—an analysis from the International Consortium for MDS (icMDS) VALIDATE database

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  • 发表日期：

    2026年6月29日

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  • Jan Philipp Bewersdorf,
  • Tariq Kewan,
  • Luca Lanino,
  • Wei Wei,
  • Tulika Rudra Gupta,
  • Jessica M. Stempel,
  • Najla H. Al Ali,
  • Amy E. DeZern,
  • Mikkael A. Sekeres,
  • Geoffrey L. Uy,
  • Samuel Urrutia,
  • Hetty E. Carraway,
  • Pinkal Desai,
  • Elizabeth A. Griffiths,
  • Eytan M. Stein,
  • Andrew M. Brunner,
  • Christine McMahon,
  • Rory M. Shallis,
  • Joshua F. Zeidner,
  • Michael R. Savona,
  • Hayley Hawkins,
  • Namrata Sonia Chandhok,
  • Constantine N. Logothetis,
  • Aram Bidikian,
  • Ted M. Getz,
  • Gail J. Roboz,
  • Benjamin Rolles,
  • Eunice S. Wang,
  • Amyah C. Harris,
  • Maria L. Amaya,
  • Somedeb Ball,
  • Justin Grenet,
  • Zhuoer Xie,
  • Yazan F. Madanat,
  • Yasmin Abaza,
  • Talha Badar,
  • Jaclynn Campos,
  • Torsten Haferlach,
  • Jaroslaw P. Maciejewski,
  • David Sallman,
  • Anoop Enjeti,
  • Kamal Al-Rabi,
  • Khalid Halahleh,
  • Devendra Hiwase,
  • Maria Diez-Campelo,
  • David Valcarcel,
  • Claudia Haferlach,
  • Lisa Pleyer,
  • Ioannis Kotsianidis,
  • Vassiliki Pappa,
  • Valeria Santini,
  • Angela Consagra,
  • Aref Al-Kali,
  • Seishi Ogawa,
  • Yasuhito Nannya,
  • Maximilian Stahl,
  • Matteo Giovanni Della Porta,
  • Rami S. Komrokji,
  • Amer M. Zeidan

摘要

Monotherapy with hypomethylating agents (HMA) remains the standard of care for patients with higher-risk myelodysplastic neoplasms (HR-MDS). Recently, the randomized phase III VERONA study evaluating azacitidine plus venetoclax (VEN) versus azacitidine plus placebo in newly diagnosed HR-MDS showed no difference in overall survival (OS) between the two arms. However, whether the addition of VEN to HMA improves outcomes among subsets of patients with HR-MDS remains debated. We analyzed 1907 patients with HR-MDS from 31 centers in 9 countries who were treated with HMA monotherapy or HMA/VEN in the frontline setting (HMA monotherapy: n = 1773; HMA/VEN: n = 134). Responses were assessed centrally by two investigators using the IWG 2023 response criteria. Addition of VEN improved composite complete remission (cCR) rates (48.8% vs. 27.7%; p < 0.001) but not CR rates (17.1% vs. 11.7%; p = 0.16). In multivariable logistic regression analysis, cCR remained favorable for HMA/VEN vs. HMA monotherapy (Odds Ratio [OR]: 2.49; 95% CI: 1.56-3.96; p < 0.001). However, we did not observe a statistically significant difference in OS for HMA/VEN vs. HMA monotherapy (Hazard Ratio [HR]: 0.83; 95% CI: 0.64-1.07; p = 0.15). In subgroup analyses, patients with TP53 wild-type disease (HR: 0.47; 95% CI: 0.29-0.74; p = 0.002) had a significant improvement in OS and those with ≥10% bone marrow blasts (HR: 0.73; 95% CI: 0.53-1.01; p = 0.06) had a trend towards OS benefit with HMA/VEN.