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Journal Articles

Validation of the international working group (IWG) 2023 criteria for clinical benefit in higher risk Myelodysplastic Syndromes (HR-MDS) using a large, international, randomized Phase 3 clinical trial dataset

Free
Maximilian Stahl, Jan Philipp Bewersdorf, Rahul Vedula, Yiwen Liu, H. Moses Murdock, Harrison Tsai, Evan Chen, Benjamin Rolles, Nikolai Podoltsev, Lourdes Mendez, Luca Lanino, Alain Mina, Luis E. Aguirre, Mikkael Sekeres, Lionel Ades, R. Coleman Lindsley, Amer Zeidan
Journal: Blood
Blood (2025) 146 (Supplement 1): 5651.
https://doi.org/10.1182/blood-2025-5651
Published: 2025
Journal Articles

Remark: A phase II, open-label, multicenter study of orally administered romaciclib (RVU120) for the treatment of anemia in patients with lower-risk myelodysplastic neoplasms (LR-MDS)

Free
Uwe Platzbecker, Elena Crisa, Sophie Salazar, Thomas Cluzeau, Antonella Poloni, Raquel De Paz Arias, Thibault Comont, Sebastian Grosicki, Aristoteles Giagounidis, Matteo Della Porta, Pellegrino Musto, Mar Tormo, Dominic Brauer, Ana Alfonso-Pierola, Stefania Paolini, Sophie Park, Annelies Weyn, Lionel Ades, Valeria Santini
Journal: Blood
Blood (2025) 146 (Supplement 1): 5649.
https://doi.org/10.1182/blood-2025-5649
Published: 2025
Journal Articles

A phase II study evaluating the efficacy and safety of imetelstat in patients with advanced myelodysplastic neoplasms or AML failing HMA-based therapy – interim analysis results of the IMpress study

Free
Lionel Ades, Steven Lane, Alice Garnier, Thomas Cluzeau, Odile Rauzy, Katharina S. Götze, Aristoteles Giagounidis, Deepak Singhal, Dominic Brauer, Carolyn Grove, Katharina Mohr, Fatiha Chermat, Joanna Nkyekyer, Uwe Platzbecker
Journal: Blood
Blood (2025) 146 (Supplement 1): 5155.
https://doi.org/10.1182/blood-2025-5155
Published: 2025
Journal Articles

Wide variability and inconsistency in reporting of results of Myelodysplastic Syndromes/neoplasms (MDS) clinical trials: An icMDS systematic review of published manuscripts

Free
Ted Getz, Kelly Pugh, Andrew Brunner, Jan Philipp Bewersdorf, Amy DeZern, Tariq Kewan, Uma Borate, Uwe Platzbecker, Mikkael Sekeres, Andrew Wei, Shahram Kordasti, Rena Buckstein, Gail Roboz, Michael Savona, Sanam Loghavi, Robert Hasserjian, Pierre Fenaux, David Sallman, Christopher Hourigan, Matteo Della Porta, Stephen Nimer, Richard Little, Valeria Santini, Fabio Efficace, Justin Taylor, Olatoyosi Odenike, Tae Kon Kim, Stephanie Halene, Rami Komrokji, Elizabeth Griffiths, Peter Greenberg, Mina Xu, Zhuoer Xie, Rafael Bejar, Guillermo Sanz, Mrinal Patnaik, Maria E. Figueroa, Hetty Carraway, Omar Abdel-Wahab, Daniel Starczynowski, Eric Padron, Jacqueline Boultwood, Steven Gore, Naval Daver, Jane Churpek, Ravi Majeti, John Bennett, Alan List, Adrian Mosquera Orgueira, Klaus Metzeler, Francesc Sole, Lisa Pleyer, Arjan Van de Loosdrecht, Thomas Cluzeau, David Steensma, Ghulam Mufti, Lionel Ades, Luca Lanino, Yazan Madanat, Anne Sophie Kubasch, Marie Sebert, R. Coleman Lindsley, Katharina S. Götze, Carmelo Gurnari, Yasushi Miyazaki, Aref Al-Kali, Abdulraheem Yacoub, Bing Li, Ioannis Kotsianidis, Maximilian Stahl, Amer Zeidan
Journal: Blood
Blood (2025) 146 (Supplement 1): 3852.
https://doi.org/10.1182/blood-2025-3852
Published: 2025
Journal Articles

Characteristics and prognostic significance of myelodysplastic syndrome (MDS)-related features in vexas syndrome

Free
Lin-Pierre Zhao, Olivier Kosmider, Mael Heiblig, Thomas Moulinet, Thibault Comont, Hervé Lobbes, Jean-Denis Karam, Louis Terriou, Samuel Ardois, Guillaume Le Guenno, Vincent Jachiet, Louis Wolff, Jerome Hadjadj, Jean-David Bouaziz, Olivier Fain, Benjamin Terrier, Sophie Georgin-Lavialle, Lise Larcher, Emmanuelle Clappier, Marie Sebert, Lionel Ades, Arsene Mekinian, Pierre Fenaux
Journal: Blood
Blood (2025) 146 (Supplement 1): 3165.
https://doi.org/10.1182/blood-2025-3165
Published: 2025
Journal Articles

Clinical and biological correlates of blast-transformed chronic myelomonocytic leukemia (CMML): A multicenter study of 493 paired cases at chronic phase and at blast transformation

Free
Clifford Csizmar, Guillermo Montalban-Bravo, Leila Cysique-Foinlan, Anuya Natu, Terra Lasho, Christy Finke, Aref Al-Kali, Hassan Alkhateeb, Ali Alsugair, Kebede Begna, Saubia Fathima, Naseema Gangat, Abhishek Mangaonkar, Antoine Saliba, Ayalew Tefferi, Margot Morabito, Nathalie Droin, Cyril Quivoron, Benjamin Podvin, Nicolas Duployez, Olivier Kosmider, Christophe Marzac, Sylvain Thépot, Mathieu Meunier, Céline Berthon, Thorsten Braun, Christophe Willekens, Stephane De Botton, Lionel Ades, Alexandre Bazinet, Kelly Chien, Danielle Hammond, Courtney DiNardo, Tapan Kadia, Rashmi Kanagal-Shamanna, Sanam Loghavi, Naveen Pemmaraju, Farhad Ravandi, Koji Sasaki, Hagop Kantarjian, Pierre Fenaux, Eric Solary, Guillermo Garcia-Manero, Aline Renneville, Mrinal Patnaik
Journal: Blood
Blood (2025) 146 (Supplement 1): 2081.
https://doi.org/10.1182/blood-2025-2081
Published: 2025
Journal Articles

Luspatercept initiated at the maximum-approved dose in transfusion-dependent lower-risk myelodysplastic syndromes: Interim analysis from maxilus

Free
Amer Zeidan, Maria Diez-Campelo, Valeria Santini, Rena Buckstein, Lionel Adès, Lea Sahagun, Gitanjali Das, Tatiana Zelinsky, Yinzhi Lai, Dimana Miteva, Ali McBride, Jose Alberto Nadal, Krzysztof Madry, David Valcarcel, Jose Miguel Torregrosa Diaz, Dries Deeren, Sebastian Grosicki, Sangeetha Venugopal, Uwe Platzbecker, Matteo Della Porta
Journal: Blood
Blood (2025) 146 (Supplement 1): 789.
https://doi.org/10.1182/blood-2025-789
Published: 2025
Journal Articles

Does stringent cytoreduction improve survival in myeloproliferative chronic myelomonocytic leukemia? a correlative study on the dacota trial.

Free
Dorothée Selimoglu-Buet, Sylvie Chevret, Valeria Santini, Sylvain Thépot, Margot Morabito, Lionel Ades, Lucie Laplane, Aristoteles Giagounidis, Nathalie Droin, Michael Lübbert, Rosa Sapena, Stanislas Nimubona, Jose Miguel Torregrosa Diaz, Ulrich Germing, Anna Maria Pelizzari, Sophie Park, Nadja Jaekel, Georgia Metzgeroth, Francesco Onida, Franciane Paul, Andrea Patriarca, Aspasia Stamatoulas Bastard, Katharina S. Götze, Fatiha Chermat, Uwe Platzbecker, Pierre Fenaux, Eric Solary, Raphael Itzykson
Journal: Blood
Blood (2025) 146 (Supplement 1): 791.
https://doi.org/10.1182/blood-2025-791
Published: 2025
Journal Articles

Real-life CD34+CD38- leukemic stem cell (LSC) flow quantification in adult AML at diagnosis independently predicts survival of intensively but not AZA-VEN treated patients: A study of prospective observational ALFA-PPP acute leukemia french association

Free
Adriana Plesa, Alexandra COELHO, Stéphanie Mathis, Veronique Saada, Isabelle Arnoux, Valerie Bardet, Florent Dumezy, Clémentine Chauvel, Jaja Zhu, Vanessa Nivaggioni, Delphine Manzoni, Iole Oster, Dominique Penther, Nicolas Duployez, Karine Celli-Lebras, Nicole Raus, Emmanuel Raffoux, Lionel Ades, Christophe Willekens, Jean-Baptiste Micol, Stephane De Botton, Laure Farnault, Sylvain Chantepie, Thomas Cluzeau, Julia HIEULLE, Céline Berthon, Ahmad Aljijakli, Claire Bories, Kevin-James Wattebled, Renaud Buffet, Raphael Itzykson, Hervé Dombret, Claude Preudhomme, Christophe Roumier
Journal: Blood
Blood (2025) 146 (Supplement 1): 3483.
https://doi.org/10.1182/blood-2025-3483
Published: 2025
Journal Articles

Real-world patterns of treatment intensity and prognostic model performance in older AML: Insights from the french ALFA-PPP cohort

Free
Lin-Pierre Zhao, Céline Berthon, Lauren Fenwarth, Stephane De Botton, Thomas Cluzeau, Laure Farnault, Sylvain Chantepie, Pierre Arnautou, Thorsten Braun, Mathieu Leclerc, Delphine Lebon, Ahmad Aljijakli, Isabelle Plantier, Ollivier Legrand, Juliette Lambert, Christophe Willekens, Emmanuel Raffoux, Claire Bories, Emilie Lemasle, Kevin-James Wattebled, Ambroise Marçais, Celia Salanoubat, Pascal Turlure, Jamilé Frayfer, Benjamin Carpentier, Madalina Uzunov, Reda Garidi, Ioana Vaida, Renaud Buffet, Alexandra COELHO, Karine Celli-Lebras, Raphael Itzykson, Claude Preudhomme, Nicolas Duployez, Hervé Dombret, Lionel Ades
Journal: Blood
Blood (2025) 146 (Supplement 1): 454.
https://doi.org/10.1182/blood-2025-454
Published: 2025
Journal Articles

Real life treatments and outcomes of older patients aged more than 60 years with FLT3-mutated acute myeloid leukemia: Report from the multicentric french observational ALFA-PPP study

Free
Ramy Rahmé, Emmanuel Raffoux, Lionel Ades, Stephane De Botton, Nicolas Duployez, Alexandra COELHO, Jean-Baptiste Micol, Sylvain Chantepie, Thomas Cluzeau, Delphine Lebon, Pierre Arnautou, Mathieu Leclerc, Laure Farnault, Ollivier Legrand, Ahmad Aljijakli, Céline Berthon, Juliette Lambert, Claire Bories, Ambroise Marçais, Julia HIEULLE, Celia Salanoubat, Pascal Turlure, Kevin-James Wattebled, Benjamin Carpentier, Jamile Frayfer, Ioana Vaida, Madalina Uzunov, Reda Garidi, Luca Inchiappa, Karine Celli-Lebras, Dominique Penther, Raynier Devillier, Raphael Itzykson, Claude Preudhomme, Renaud Buffet, Herve Dombret, Thorsten Braun
Journal: Blood
Blood (2025) 146 (Supplement 1): 449.
https://doi.org/10.1182/blood-2025-449
Published: 2025
Journal Articles

Outcomes of NPM1-mutated AMLs in first hematological relapse and prognostic value of MRD

Free
Sofiane Fodil, Matthieu Duchmann, Edoardo Tamellini, Anne Couprie, Rathana KIM, Pierre-Edouard Debureaux, Marie Passet, Nicolas Duployez, Etienne Lengline, Mattoe Dragani, Laureen Chat, Reinaldo Dal Bello, Matthieu Jestin, Lin-Pierre Zhao, Regis Peffault De Latour, Marie Sebert, Delphine Lebon, Nicolas Boissel, Lionel Ades, Emmanuelle Clappier, Emmanuel Raffoux, Raphael Itzykson
Journal: Blood
Blood (2025) 146 (Supplement 1): 5294.
https://doi.org/10.1182/blood-2025-5294
Published: 2025
Journal Articles

IPSS-M risk and specific sex-associated somatic mutations predict response to ESA therapy in LR-MDS: building a new score

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Clinical Trials & Observations
Marco G. Raddi, Sophie Park, Michaela Fontenay, Olivier Kosmider, Maria Diez Campelo, Juan C. Berrocal, Marie Sebert, Lionel Adès, Uwe Platzbecker, Matteo G. Della Porta, Giorgio Mattiuz, Sven De Pourcq, Lorenzo Tofani, Giulia Maggioni, Elena Tofacchi, Angela Consagra, Luca Rigodanza, Gloria Andreossi, Cristina Amato, Mathieu Meunier, Celia Orlando, Emmanuelle Clappier, Nicolas Chapuis, Pierre Walczak, Katharina Zoldan, Anne Sophie Kubasch, Valeria Santini
Journal: Blood
Blood (2025) 146 (14): 1693–1706.
https://doi.org/10.1182/blood.2024027540
Published: 2025
Includes: Supplemental data
Journal Articles

Clinical-genomic profiling of MDS to inform allo-HCT: recommendations from an international panel on behalf of the EBMT

Free
Carmelo Gurnari, Marie Robin, Lionel Adès, Mahmoud Aljurf, Antonio Almeida, Fernando Barroso Duarte, Elsa Bernard, Corey Cutler, Matteo Giovanni Della Porta, Theo De Witte, Amy DeZern, Joanna Drozd-Sokolowska, Eric Duncavage, Pierre Fenaux, Nico Gagelmann, Guillermo Garcia-Manero, Claudia Haferlach, Torsten Haferlach, Robert Hasserjian, Eva Hellström-Lindberg, Meagan Jacoby, Austin Kulasekararaj, R. Coleman Lindsley, Jaroslaw P. Maciejewski, Hideki Makishima, Luca Malcovati, Moshe Mittelman, Anders E. Myhre, Seishi Ogawa, Francesco Onida, Elli Papaemmanuil, Jakob Passweg, Uwe Platzbecker, Lisa Pleyer, Kavita Raj, Valeria Santini, Anna Sureda, Magnus Tobiasson, Maria Teresa Voso, Ibrahim Yakoub-Agha, Amer Zeidan, Matthew Walter, Nicolaus Kröger, Donal P. McLornan, Mario Cazzola
Journal: Blood
Blood (2025) 145 (18): 1987–2001.
https://doi.org/10.1182/blood.2024025131
Published: 2025
Includes: Supplemental data
Journal Articles

Jumping barriers in clinical trials: release the brake

Free
Clinical Trials & Observations
Lionel Adès
Journal: Blood
Blood (2025) 145 (13): 1335–1336.
https://doi.org/10.1182/blood.2024027711
Published: 2025
Journal Articles

Risk prediction for clonal cytopenia: multicenter real-world evidence

Free
Clinical Trials & Observations
Zhuoer Xie, Rami Komrokji, Najla Al Ali, Alexandra Regelson, Susan Geyer, Anand Patel, Caner Saygin, Amer M. Zeidan, Jan Philipp Bewersdorf, Lourdes Mendez, Ashwin Kishtagari, Joshua F. Zeidner, Catherine C. Coombs, Yazan F. Madanat, Stephen Chung, Talha Badar, James Foran, Pinkal Desai, Charlton Tsai, Elizabeth A. Griffiths, Monzr M. Al Malki, Idoroenyi Amanam, Catherine Lai, H. Joachim Deeg, Lionel Ades, Cecilia Arana Yi, Afaf E. G. Osman, Shira Dinner, Yasmin Abaza, Justin Taylor, Namrata Chandhok, Deborah Soong, Andrew M. Brunner, Hetty E. Carraway, Abhay Singh, Chiara Elena, Jacqueline Ferrari, Anna Gallì, Sara Pozzi, Eric Padron, Mrinal M. Patnaik, Luca Malcovati, Michael R. Savona, Aref Al-Kali
Journal: Blood
Blood (2024) 144 (19): 2033–2044.
https://doi.org/10.1182/blood.2024024756
Published: 2024
Includes: Supplemental data
Journal Articles

Enasidenib (ENA) Monotherapy in Patients with IDH2 mutated Myelodysplastic Syndrome (MDS), the Ideal Phase 2 Study By the GFM and Emsco Groups

Free
Marie Sebert, Sylvie Chevret, Sophie Dimicoli-Salazar, Thomas Cluzeau, Odile Rauzy, Aspasia Stamatoulas Bastard, Kamel Laribi, Gaelle Fossard, Sylvain Thépot, Silke Gloaguen, Kristell Desseaux, Norbert Vey, Pierre Peterlin, Anne-Laure Taksin, Caroline Lejeune, Sophie Park, Anne Banos, Aristoteles Giagounidis, Emmanuel Gyan, Mario Ojeda-Uribe, Stefanie Groepper, Fatiha Chermat, Emmanuelle Clappier, Uwe Platzbecker, Pierre Fenaux, Lionel Ades
Journal: Blood
Blood (2024) 144 (Supplement 1): 1839.
https://doi.org/10.1182/blood-2024-201317
Published: 2024
Journal Articles

IL-1RAP Is a Targetable Surface Antigen in Relapsed/Refractoy AML

Free
Mohamad Sabbah, Morgane Fontaine, Kim Pacchiardi, Alexis Delabrière, Stephane De Botton, Catherine Lonchamp, Renaud Buffet, Laure Farnault, Jean-Baptiste Micol, Typhaine Dumas-Rivero, Emmanuel Raffoux, Lionel Ades, Nicolas Boissel, Juliette Lambert, Kevin-James Wattebled, Matthieu Duchmann, Karine Celli-Lebras, Alexandra Coelho, Hervé Dombret, Edouard De Dreuzy, Marina Deschamps, Christophe Ferrand, Jens Hasskarl, Richard Sainson, Raphael Itzykson
Journal: Blood
Blood (2024) 144 (Supplement 1): 4311.
https://doi.org/10.1182/blood-2024-199673
Published: 2024
Journal Articles

JAK Inhibition Results in Aggressive Cutaneous Squamous Cell Carcinoma through MAPK Pathway Activation and Shapes the Tumor Immune Microenvironment to Create Novel Therapeutic Opportunities

Free
Helene Pasquer, Valeria Bisio, Antoine Forget, Maxime Battistella, Barouyr Baroudjian, Raphael Marie, Emmanuelle Latour, Cecile Culeux, Lea Pelissier, Blandine Roux, Khansa Saadallah, Matthieu Duchmann, Thierry Tchenio, Abdellah Tebani, Amandine Gouverneur, Fanelie Jouenne, Jerome Lambert, Isabelle Madelaine, Lionel Ades, Emmanuel Raffoux, Pernelle Noize, Bruno Cassinat, Soumeya Bekri, Juliette Soret-Dulphy, Samia Mourah, Camille Lobry, Alexandre Puissant, Jean-Jacques Kiladjian, Nicolas Dulphy, Celeste Lebbe, Lina Benajiba
Journal: Blood
Blood (2024) 144 (Supplement 1): 4513.
https://doi.org/10.1182/blood-2024-205839
Published: 2024
Journal Articles

A Phase II Study Evaluating the Efficacy and Safety of Imetelstat in Patients with Advanced Myelodysplastic Neoplasms or AML Failing HMA-Based Therapy - Interim Analysis Results of the Impress Study

Free
Uwe Platzbecker, Steven W Lane, Alice Garnier, Thomas Cluzeau, Odile Rauzy, Katharina S. S. Götze, Aristoteles Giagounidis, Deepak Singhal, Aicha Kopecky, Fatiha Chermat, Lionel Ades
Journal: Blood
Blood (2024) 144 (Supplement 1): 3222.
https://doi.org/10.1182/blood-2024-194185
Published: 2024