Медицинская библиотека · Haematologica · 2026

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.

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

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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.

Для цитирования: Bewersdorf Jan Philipp, Kewan Tariq, Lanino Luca, Blaha Ondrej, Liu Yiwen, Stempel Jessica M, Al Ali Najla H, DeZern Amy E, Sekeres Mikkael A, Uy Geoffrey L, Urrutia Samuel, Carraway Hetty E, Desai Pinkal, Griffiths Elizabeth A, Stein Eytan M, Brunner Andrew M, McMahon Christine, Shallis Rory M, Zeidner Joshua F, Savona Michael R, Hawkins Hayley, Chandhok Namrata Sonia, Logothetis Constantine, Bidikian Aram, Getz Ted M, Roboz Gail J, Rolles Benjamin, Wang Eunice S, Harris Amyah C, Amaya Maria L, Ball Somedeb, Grenet Justin, Xie Zhuoer, Madanat Yazan F, Abaza Yasmin, Badar Talha, Campos Jaclynn, Haferlach Torsten, Maciejewski Jaroslaw P, Sallman David, Vedula Rahul S, Murdock H Moses, Tsai Harrison K, Chen Evan C, Ades Lionel, Lindsley R Coleman, Enjeti Anoop, Al-Rabi Kamal, Halahleh Khalid, Hiwase Devendra, Diez-Campelo Maria, Valcarcel David, Haferlach Claudia, Pleyer Lisa, Kotsianidis Ioannis, Pappa Vassiliki, Santini Valeria, Consagra Angela, Al-Kali Aref, Ogawa Seishi, Nannya Yasuhito, Porta Matteo Giovanni Della, Komrokji Rami S, Stahl Maximilian, Zeidan Amer M. 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.. Haematologica. 2026. DOI: 10.3324/haematol.2026.301314.

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