An agenda to advance research in myelodysplastic syndromes: a TOP 10 priority list from the first international workshop in MDS

Maximilian Stahl, Omar Abdel-Wahab, Andrew H. Wei, Michael R. Savona, Mina L. Xu, Zhuoer Xie, Justin Taylor, Daniel Starczynowski, Guillermo F. Sanz, David A. Sallman, Valeria Santini, Gail J. Roboz, Mrinal M. Patnaik, Eric Padron, Olatoyosi Odenike, Aziz Nazha, Stephen D. Nimer, Ravindra Majeti, Richard F. Little, Steven Gore, Alan F. List, Vijay Kutchroo, Rami S. Komrokji, Tae Kon Kim, Nina Kim, Christopher S. Hourigan, Robert P. Hasserjian, Stephanie Halene, Elizabeth A. Griffiths, Peter L. Greenberg, Maria Figueroa, Pierre Fenaux, Fabio Efficace, Amy E. DeZern, Matteo G. Della Porta, Naval G. Daver, Jane E. Churpek, Hetty E. Carraway, Andrew M. Brunner, Uma Borate, John M. Bennett, Rafael Bejar, Jacqueline Boultwood, Sanam Loghavi, Jan Philipp Bewersdorf, Uwe Platzbecker, David P. Steensma, Mikkael A. Sekeres, Rena J. Buckstein, and Amer M. Zeidan

[1]  A. Brunner,et al.  Evaluating complete remission with partial hematologic recovery (CRh) as a response criterion in myelodysplastic syndromes (MDS) , 2022, Blood Cancer Journal.

[2]  B. Ebert,et al.  Diagnosis and Management of AML in Adults: 2022 ELN Recommendations from an International Expert Panel. , 2022, Blood.

[3]  M. Loh,et al.  International Consensus Classification of Myeloid Neoplasms and Acute Leukemia: Integrating Morphological, Clinical, and Genomic Data. , 2022, Blood.

[4]  Y. Natkunam,et al.  The 5th edition of the World Health Organization Classification of Haematolymphoid Tumours: Myeloid and Histiocytic/Dendritic Neoplasms , 2022, Leukemia.

[5]  D. Faller,et al.  Pevonedistat plus azacitidine vs azacitidine alone in higher-risk MDS/chronic myelomonocytic leukemia or low-blast-percentage AML , 2022, Blood advances.

[6]  M. Jongen‐Lavrencic,et al.  Molecular characterization of mutant TP53 acute myeloid leukemia and high-risk myelodysplastic syndrome , 2022, Blood.

[7]  R. Epstein,et al.  Real-world use and outcomes of hypomethylating agent therapy in higher-risk myelodysplastic syndromes: why are we not achieving the promise of clinical trials? , 2021, Future oncology.

[8]  M. Cazzola,et al.  Relationship between clone metrics and clinical outcome in clonal cytopenia. , 2021, Blood.

[9]  E. Estey,et al.  Distinguishing AML from MDS: a fixed blast percentage may no longer be optimal , 2021, Blood.

[10]  D. Pollyea,et al.  The complete story of less than complete responses: The evolution and application of acute myeloid leukemia clinical responses. , 2021, Blood reviews.

[11]  J. Barnard,et al.  Validation of International Working Group response criteria in higher‐risk myelodysplastic syndromes: A report on behalf of the MDS Clinical Research Consortium , 2020, Cancer medicine.

[12]  J. Klco,et al.  Advances in germline predisposition to acute leukaemias and myeloid neoplasms , 2020, Nature Reviews Cancer.

[13]  Stuart M. Gardos,et al.  Cancer therapy shapes the fitness landscape of clonal hematopoiesis , 2020, Nature Genetics.

[14]  K. Pradhan,et al.  U2AF1 mutations induce oncogenic IRAK4 isoforms and activate innate immune pathways in myeloid malignancies , 2019, Nature Cell Biology.

[15]  Y. Kluger,et al.  A highly efficient and faithful MDS patient-derived xenotransplantation model for pre-clinical studies , 2018, Nature Communications.

[16]  D. Steensma,et al.  A call for action: Increasing enrollment of untreated patients with higher‐risk myelodysplastic syndromes in first‐line clinical trials , 2017, Cancer.

[17]  M. Cazzola,et al.  Clinical significance of somatic mutation in unexplained blood cytopenia. , 2017, Blood.

[18]  C. Bloomfield,et al.  Randomized Phase II Study of Azacitidine Alone or in Combination With Lenalidomide or With Vorinostat in Higher-Risk Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia: North American Intergroup Study SWOG S1117. , 2017, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[19]  M. Broder,et al.  The Incidence and Health Care Resource Burden of the Myelodysplastic Syndromes in Patients in Whom First-Line Hypomethylating Agents Fail. , 2017, The oncologist.

[20]  D. Neuberg,et al.  Prognostic Mutations in Myelodysplastic Syndrome after Stem‐Cell Transplantation , 2017, The New England journal of medicine.

[21]  Bob Löwenberg,et al.  Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel. , 2017, Blood.

[22]  J. Cleveland,et al.  The NLRP3 inflammasome functions as a driver of the myelodysplastic syndrome phenotype. , 2016, Blood.

[23]  S. Nahas,et al.  Impact of TP53 mutation variant allele frequency on phenotype and outcomes in myelodysplastic syndromes , 2016, Leukemia.

[24]  M. McCarthy,et al.  Age-related clonal hematopoiesis associated with adverse outcomes. , 2014, The New England journal of medicine.

[25]  M. Stratton,et al.  Clinical and biological implications of driver mutations in myelodysplastic syndromes. , 2013, Blood.

[26]  B. Esterni,et al.  Outcome of high-risk myelodysplastic syndrome after azacitidine treatment failure. , 2011, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[27]  J. Issa,et al.  Outcome of patients with myelodysplastic syndrome after failure of decitabine therapy , 2010, Cancer.

[28]  H. Dombret,et al.  Azacitidine prolongs overall survival compared with conventional care regimens in elderly patients with low bone marrow blast count acute myeloid leukemia. , 2010, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[29]  B. Cheson,et al.  Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia. , 2006, Blood.

[30]  B. Cheson,et al.  Report of an international working group to standardize response criteria for myelodysplastic syndromes. , 2000, Blood.