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Journal Articles

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

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

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

Blood (2025) 146 (Supplement 1): 3165.

https://doi.org/10.1182/blood-2025-3165

Published: 2025

Journal Articles

Real life study of ivosidenib in either monotherapy or combined with azacytidine for first line mutant IDH1 AML: A study from the french AML intergroup ALFA/filo

Blood (2025) 146 (Supplement 1): 1658.

https://doi.org/10.1182/blood-2025-1658

Published: 2025

Journal Articles

PyramIDH: A phase 3 study of ivosidenib monotherapy or azacitidine monotherapy in patients with mutant isocitrate dehydrogenase 1 myelodysplastic syndromes who have not received prior hypomethylating agent therapy

Marie Sebert, Valeria Santini, Uwe Platzbecker, David Valcarcel, Uma Borate, Weishi Yuan, Stephanie Kapsalis, Lorene Simonot, Marissa Panzica, Prapti Patel, Guillermo Garcia-Manero

Blood (2025) 146 (Supplement 1): 8254.

https://doi.org/10.1182/blood-2025-8254

Published: 2025

Journal Articles

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

Blood (2025) 146 (Supplement 1): 5294.

https://doi.org/10.1182/blood-2025-5294

Published: 2025

Journal Articles

Treatments and outcomes of adult patients with TP53-mutated acute myeloid leukemia (AML) in the real-life –Report of the prospective french observational ALFA-PPP study.

Blood (2025) 146 (Supplement 1): 6386.

https://doi.org/10.1182/blood-2025-6386

Published: 2025

Journal Articles

Ivosidenib in refractory or relapsed IDH1-mutated Acute Myeloid Leukemia patients in real life settings: The ivoobs observational study from the french AML intergroup ALFA/filo

Blood (2025) 146 (Supplement 1): 3455.

https://doi.org/10.1182/blood-2025-3455

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

Blood (2025) 146 (14): 1693–1706.

https://doi.org/10.1182/blood.2024027540

Published: 2025

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

Blood (2024) 144 (Supplement 1): 1839.

https://doi.org/10.1182/blood-2024-201317

Published: 2024

Journal Articles

Prognostic Significance of Monocytic-like Phenotype in AML Patients Treated with Venetoclax and Azacytidine

Lin Pierre Zhao, Typhaine Dumas-Rivero, Lauren Barette, Lorea Aguinaga, Arij Cheffai, Pierre Lemaire, Stéphanie Mathis, Clémentine Chauvel, Reinaldo Dal Bello, Emmanuelle Clappier, Matthieu Duchmann, Pierre Fenaux, Marie Sebert, Lionel Ades, Raphael Itzykson

Blood (2024) 144 (Supplement 1): 4316.

https://doi.org/10.1182/blood-2024-203353

Published: 2024

Journal Articles

Phase 3 Study of Either Ivosidenib (IVO) Monotherapy or Azacitidine (AZA) Monotherapy in Patients with IDH1 Mutant Myelodysplastic Syndromes (MDS) Who Are Hypomethylating Agent (HMA) Naive (PyramIDH)

Marie Sebert, Uwe Platzbecker, David Valcarcel Ferreiras, Valeria Santini, Uma Borate, Stephanie M. Kapsalis, Lorene Simonot, Weishi Yuan, Prapti A. Patel, Guillermo Garcia-Manero

Blood (2024) 144 (Supplement 1): 1845.1.

https://doi.org/10.1182/blood-2024-198172

Published: 2024

Journal Articles

Combining ESA and Luspatercept in Non-RS MDS Patients Having Failed ESA - Results of the Phase 1-2 Part a of the GFM Combola Study

Lionel Ades, Thomas Cluzeau, Thibault Comont, Lorea Aguinaga, Aspasia Stamatoullas, Mathieu Meunier, Emmanuel Gyan, Alice Garnier, Maud D'Aveni, Sylvain Thépot, Marie Sebert, Marius Moldovan, Anouk Walter Petrich, Karine Lemarie, Fatiha Chermat, Michaela Fontenay, Sylvie Chevret, Pierre Fenaux

Blood (2024) 144 (Supplement 1): 351.

https://doi.org/10.1182/blood-2024-204791

Published: 2024

Journal Articles

Efficacy and Safety of Luspatercept +/- Erythropoiesis-Stimulating Agent (ESA) in Patients with Myelodysplastic Syndromes with Ring Sideroblasts (MDS-RS): A French Multicenter Prospective Real-Life Registry

Blood (2023) 142 (Supplement 1): 6471.

https://doi.org/10.1182/blood-2023-177787

Published: 2023

Journal Articles

Phase I/II Clinical Trial Evaluating Azacitidine + Venetoclax + Donor Lymphocyte Infusion in Post-Transplant Relapse Myelodysplastic Syndromes and Acute Myeloid Leukemia: Preliminary Results of Ventograft, a GFM Study

Thomas Cluzeau, Marie Sebert, Michael Loschi, Lionel Ades, Sylvain Thepot, Martin Carre, Nicolas Duployez, Rosa Sapena, Fatiha Chermat, Claude Preudhomme, Pierre Fenaux, Marie Robin

Blood (2023) 142 (Supplement 1): 3246.

https://doi.org/10.1182/blood-2023-178021

Published: 2023

Journal Articles

Lymphopenia Confers Poorer Prognosis in Myelodysplastic Neoplasms (MDS) with Very Low and Low IPSS-M

David Fandrei, Tony Huynh, Marie Sebert, Lorea Aguinaga, Valeria Bisio, Marion Espéli, Karl Balabanian, Hélène Moins-Teisserenc, Antoine Toubert, Nicolas Dulphy, Pierre Fenaux, Lionel Ades, Lin-Pierre Zhao

Blood (2023) 142 (Supplement 1): 4597.

https://doi.org/10.1182/blood-2023-187707

Published: 2023

Journal Articles

The Splicing-Associated Network PAK1-Clk-SRRM1 Is a Critical Vulnerability to Overcome Chemoresistance in Acute Myeloid Leukemia

Blood (2023) 142 (Supplement 1): 409.

https://doi.org/10.1182/blood-2023-186264

Published: 2023

Journal Articles

IPSS-M in myelodysplastic neoplasms arising from aplastic anemia and paroxysmal nocturnal hemoglobinuria

Blood (2023) 141 (25): 3122–3125.

https://doi.org/10.1182/blood.2023020108

Published: 2023

Includes: Supplemental data

Journal Articles

Molecular International Prognostic Scoring System (IPSS-M) in Myelodysplastic Syndromes Arising from Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Blood (2022) 140 (Supplement 1): 5788–5789.

https://doi.org/10.1182/blood-2022-160458

Published: 2022

Journal Articles

Prognostic impact of DDX41 germline mutations in intensively treated acute myeloid leukemia patients: an ALFA-FILO study

Blood (2022) 140 (7): 756–768.

https://doi.org/10.1182/blood.2021015328

Published: 2022

Includes: Supplemental data

Journal Articles

Clinical, pathological, and molecular features of myelodysplasia cutis

Jérémie Delaleu, Rathana Kim, Lin-Pierre Zhao, Adèle de Masson, Marie-Dominique Vignon-Pennamen, Charles Cassius, Caroline Ram-Wolff, Martine Bagot, Emmanuelle Clappier, Lionel Adès, Marie Sebert, Jean-David Bouaziz, Pierre Fenaux, Maxime Battistella

Blood (2022) 139 (8): 1251–1253.

https://doi.org/10.1182/blood.2021013967

Published: 2022

Includes: Supplemental data

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

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

Real life study of ivosidenib in either monotherapy or combined with azacytidine for first line mutant IDH1 AML: A study from the french AML intergroup ALFA/filo

PyramIDH: A phase 3 study of ivosidenib monotherapy or azacitidine monotherapy in patients with mutant isocitrate dehydrogenase 1 myelodysplastic syndromes who have not received prior hypomethylating agent therapy

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

Treatments and outcomes of adult patients with TP53-mutated acute myeloid leukemia (AML) in the real-life –Report of the prospective french observational ALFA-PPP study.

Ivosidenib in refractory or relapsed IDH1-mutated Acute Myeloid Leukemia patients in real life settings: The ivoobs observational study from the french AML intergroup ALFA/filo

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

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

Prognostic Significance of Monocytic-like Phenotype in AML Patients Treated with Venetoclax and Azacytidine

Phase 3 Study of Either Ivosidenib (IVO) Monotherapy or Azacitidine (AZA) Monotherapy in Patients with IDH1 Mutant Myelodysplastic Syndromes (MDS) Who Are Hypomethylating Agent (HMA) Naive (PyramIDH)

Combining ESA and Luspatercept in Non-RS MDS Patients Having Failed ESA - Results of the Phase 1-2 Part a of the GFM Combola Study

Efficacy and Safety of Luspatercept +/- Erythropoiesis-Stimulating Agent (ESA) in Patients with Myelodysplastic Syndromes with Ring Sideroblasts (MDS-RS): A French Multicenter Prospective Real-Life Registry

Phase I/II Clinical Trial Evaluating Azacitidine + Venetoclax + Donor Lymphocyte Infusion in Post-Transplant Relapse Myelodysplastic Syndromes and Acute Myeloid Leukemia: Preliminary Results of Ventograft, a GFM Study

Lymphopenia Confers Poorer Prognosis in Myelodysplastic Neoplasms (MDS) with Very Low and Low IPSS-M

The Splicing-Associated Network PAK1-Clk-SRRM1 Is a Critical Vulnerability to Overcome Chemoresistance in Acute Myeloid Leukemia

IPSS-M in myelodysplastic neoplasms arising from aplastic anemia and paroxysmal nocturnal hemoglobinuria

Molecular International Prognostic Scoring System (IPSS-M) in Myelodysplastic Syndromes Arising from Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Prognostic impact of DDX41 germline mutations in intensively treated acute myeloid leukemia patients: an ALFA-FILO study

Clinical, pathological, and molecular features of myelodysplasia cutis

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