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

Maximilian Stahl¹, Omar Abdel-Wahab², Andrew H Wei³, Michael R Savona⁴, Mina L Xu⁵, Zhuoer Xie⁶, Justin Taylor⁷, Daniel Starczynowski⁸, Guillermo F Sanz^{9

10

11}, David A Sallman⁶, Valeria Santini¹², Gail J Roboz¹³, Mrinal M Patnaik¹⁴, Eric Padron⁶, Olatoyosi Odenike¹⁵, Aziz Nazha¹⁶, Stephen D Nimer⁷, Ravindra Majeti¹⁷, Richard F Little¹⁸, Steven Gore¹⁸, Alan F List¹⁹, Vijay Kutchroo²⁰, Rami S Komrokji⁶, Tae Kon Kim⁴, Nina Kim¹⁸, Christopher S Hourigan²¹, Robert P Hasserjian²², Stephanie Halene²³, Elizabeth A Griffiths²⁴, Peter L Greenberg¹⁷, Maria Figueroa⁷, Pierre Fenaux²⁵, Fabio Efficace²⁶, Amy E DeZern²⁷, Matteo G Della Porta²⁸, Naval G Daver²⁹, Jane E Churpek³⁰, Hetty E Carraway³¹, Andrew M Brunner³², Uma Borate³³, John M Bennett³⁴, Rafael Bejar³⁵, Jacqueline Boultwood³⁶, Sanam Loghavi³⁷, Jan Philipp Bewersdorf², Uwe Platzbecker³⁸, David P Steensma³⁹, Mikkael A Sekeres⁷, Rena J Buckstein⁴⁰, Amer M Zeidan²³

Affiliations

¹ Department of Medical Oncology, Dana-Farber Cancer Institute and Harvard Medical School, Boston, MA.
² Department of Medicine, Leukemia Service, Memorial Sloan Kettering Cancer Center, New York, NY.
³ Peter MacCallum Cancer Centre, Royal Melbourne Hospital, University of Melbourne and Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia.
⁴ Division of Hematology/Oncology, Department of Medicine, Vanderbilt University Medical Center, Nashville, TN.
⁵ Departments of Pathology & Laboratory Medicine, Yale University School of Medicine and Yale Cancer Center, New Haven, CT.
⁶ Department of Malignant Hematology, H. Lee Moffitt Cancer Center, Tampa, FL.
⁷ Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami, FL.
⁸ Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
⁹ Hematology Department, Hospital Universitario y Politécnico La Fe, Valencia, Spain.
¹⁰ Health Research Institute La Fe, Valencia, Spain.
¹¹ CIBERONC, Instituto de Salud Carlos III, Madrid, Spain.
¹² MDS Unit, AOUC, University of Florence, Florence, Italy.
¹³ Weill Cornell Medicine and The New York Presbyterian Hospital, New York, NY.
¹⁴ Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN.
¹⁵ The University of Chicago, Chicago, IL.
¹⁶ Department of Medical Oncology, Sidney Kimmel Cancer Center, Thomas Jefferson University, Philadelphia, PA.
¹⁷ Division of Hematology, Department of Medicine, Cancer Institute, Stanford University School of Medicine, Stanford, CA.
¹⁸ National Cancer Institute, National Institutes of Health, Bethesda, MD.
¹⁹ Precision BioSciences, Inc, Durham, NC.
²⁰ Evergrande Center for Immunologic Diseases, Harvard Medical School, Brigham and Women's Hospital, Boston, MA.
²¹ Laboratory of Myeloid Malignancies, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD.
²² Department of Pathology, Massachusetts General Hospital, Boston, MA.
²³ Section of Hematology, Department of Internal Medicine, Yale University School of Medicine and Yale Cancer Center, New Haven, CT.
²⁴ Roswell Park Comprehensive Cancer Center, Buffalo, NY.
²⁵ Hôpital Saint Louis, Paris, France.
²⁶ Italian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy.
²⁷ The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, MD.
²⁸ Humanitas Clinical and Research Center & Humanitas University, Department of Biomedical Sciences, Milan, Italy.
²⁹ Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX.
³⁰ Department of Hematology, Oncology, and Palliative Care, Carbone Cancer Center, The University of Wisconsin-Madison, Madison, WI.
³¹ Leukemia Program, Hematology and Medical Oncology, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH.
³² Center for Leukemia, Massachusetts General Hospital, Boston, MA.
³³ Division of Hematology, Department of Internal Medicine, James Cancer Center, Ohio State University, Columbus, OH.
³⁴ Hematopathology Division, Departments of Pathology and Medicine, University of Rochester Medical Center, Rochester, NY.
³⁵ Division of Hematology and Oncology, Moores Cancer Center, UC San Diego, La Jolla, CA.
³⁶ Blood Cancer UK Molecular Haematology Unit, Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK.
³⁷ Department of Hematopathology, The University of Texas MD Anderson Cancer Center, Houston, TX.
³⁸ Department of Hematology and Cellular Therapy, Medical Clinic and Policlinic I, Leipzig University Hospital, Leipzig, Germany.
³⁹ Novartis Institutes of BioMedical Research.
⁴⁰ Odette Cancer Centre, Sunnybrook Health Sciences Centre, Toronto, Canada.

PMID: 36260702
PMCID: PMC10333740
DOI: 10.1182/bloodadvances.2022008747

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

Maximilian Stahl et al. Blood Adv. 2023.

. 2023 Jun 27;7(12):2709-2714.

doi: 10.1182/bloodadvances.2022008747.

Authors

Affiliations

¹ Department of Medical Oncology, Dana-Farber Cancer Institute and Harvard Medical School, Boston, MA.
² Department of Medicine, Leukemia Service, Memorial Sloan Kettering Cancer Center, New York, NY.
³ Peter MacCallum Cancer Centre, Royal Melbourne Hospital, University of Melbourne and Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia.
⁴ Division of Hematology/Oncology, Department of Medicine, Vanderbilt University Medical Center, Nashville, TN.
⁵ Departments of Pathology & Laboratory Medicine, Yale University School of Medicine and Yale Cancer Center, New Haven, CT.
⁶ Department of Malignant Hematology, H. Lee Moffitt Cancer Center, Tampa, FL.
⁷ Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami, FL.
⁸ Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
⁹ Hematology Department, Hospital Universitario y Politécnico La Fe, Valencia, Spain.
¹⁰ Health Research Institute La Fe, Valencia, Spain.
¹¹ CIBERONC, Instituto de Salud Carlos III, Madrid, Spain.
¹² MDS Unit, AOUC, University of Florence, Florence, Italy.
¹³ Weill Cornell Medicine and The New York Presbyterian Hospital, New York, NY.
¹⁴ Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN.
¹⁵ The University of Chicago, Chicago, IL.
¹⁶ Department of Medical Oncology, Sidney Kimmel Cancer Center, Thomas Jefferson University, Philadelphia, PA.
¹⁷ Division of Hematology, Department of Medicine, Cancer Institute, Stanford University School of Medicine, Stanford, CA.
¹⁸ National Cancer Institute, National Institutes of Health, Bethesda, MD.
¹⁹ Precision BioSciences, Inc, Durham, NC.
²⁰ Evergrande Center for Immunologic Diseases, Harvard Medical School, Brigham and Women's Hospital, Boston, MA.
²¹ Laboratory of Myeloid Malignancies, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD.
²² Department of Pathology, Massachusetts General Hospital, Boston, MA.
²³ Section of Hematology, Department of Internal Medicine, Yale University School of Medicine and Yale Cancer Center, New Haven, CT.
²⁴ Roswell Park Comprehensive Cancer Center, Buffalo, NY.
²⁵ Hôpital Saint Louis, Paris, France.
²⁶ Italian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy.
²⁷ The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, MD.
²⁸ Humanitas Clinical and Research Center & Humanitas University, Department of Biomedical Sciences, Milan, Italy.
²⁹ Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX.
³⁰ Department of Hematology, Oncology, and Palliative Care, Carbone Cancer Center, The University of Wisconsin-Madison, Madison, WI.
³¹ Leukemia Program, Hematology and Medical Oncology, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH.
³² Center for Leukemia, Massachusetts General Hospital, Boston, MA.
³³ Division of Hematology, Department of Internal Medicine, James Cancer Center, Ohio State University, Columbus, OH.
³⁴ Hematopathology Division, Departments of Pathology and Medicine, University of Rochester Medical Center, Rochester, NY.
³⁵ Division of Hematology and Oncology, Moores Cancer Center, UC San Diego, La Jolla, CA.
³⁶ Blood Cancer UK Molecular Haematology Unit, Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK.
³⁷ Department of Hematopathology, The University of Texas MD Anderson Cancer Center, Houston, TX.
³⁸ Department of Hematology and Cellular Therapy, Medical Clinic and Policlinic I, Leipzig University Hospital, Leipzig, Germany.
³⁹ Novartis Institutes of BioMedical Research.
⁴⁰ Odette Cancer Centre, Sunnybrook Health Sciences Centre, Toronto, Canada.

PMID: 36260702
PMCID: PMC10333740
DOI: 10.1182/bloodadvances.2022008747

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Conflict of interest statement

Conflict-of-interest disclosure: The first international workshop on MDS was planned by an independent scientific steering committee. The meeting was supported by unrestricted educational grants from Gilead, Novartis, Bristol Myers Squibb, Syros, Geron, and Karyopharm to Magdalen Medical/VJHemOnc. The participants met in Miami, FL, in June 2022, and deliberated on the content of this manuscript, which was subsequently finalized after the meeting. The meeting sponsors were not involved in the development of this manuscript. A.N., D.P.S., R.B., A.F.L., and N.K. authored this manuscript in their personal capacity, and their views do not necessarily represent/reflect that of their employers. The content is the sole responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. M.M.P., E.P., U.P., R.B., and S.L. were authors of the WHO classification. R.P.H. was a cochair of the Pathology ICC and co-leader of the ICC Premalignant Clonal Cytopenias and MDS working group. O.O., M.R.S., and A.H.W. were the authors of the ICC classification. A.M.Z. received research funding (institutional) from Celgene/Bristol Myers Squibb, AbbVie, Astex, Pfizer, Medimmune/AstraZeneca, Boehringer-Ingelheim, Cardiff Oncology, Incyte, Takeda, Novartis, Aprea, and ADC Therapeutics. A.M.Z. participated in advisory boards, and/or had a consultancy with and received honoraria from AbbVie, Otsuka, Pfizer, Celgene/Bristol Myers Squibb, Jazz, Incyte, Agios, Boehringer-Ingelheim, Novartis, Acceleron, Astellas, Daiichi Sankyo, Cardinal Health, Taiho, Seattle Genetics, BeyondSpring, Cardiff Oncology, Takeda, Ionis, Amgen, Janssen, Epizyme, Syndax, Gilead, Kura, Chiesi, ALX Oncology, BioCryst, Notable, Orum, and Tyme, and served on the clinical trial committees for Novartis, AbbVie, Gilead, BioCryst, ALX Oncology, Geron, and Celgene/Bristol Myers Squibb. D.P.S. is an employee of Novartis. U.P. received honoraria from Bristol Myers Squibb, Jazz Pharmaceuticals, AbbVie, and Geron. The National Heart, Lung, and Blood Institute received research funding for the laboratory of C.S.K. from Sellas and the Foundation of the NIH AML M.R.D. Biomarkers Consortium. F.E. had a consultancy or advisory role for Amgen, AbbVie, Janssen, and Novartis, and received research funding (institutional) from AbbVie, Amgen, and Novartis, outside the submitted work. O.O. has served on the advisory board of Bristol Myers Squibb, Celgene, Novartis, Taiho, and Kymera Therapeutics; serves on a DSMB for Threadwell Therapeutics; and received research funding (institutional) from AbbVie, Agios, Aprea, Astex, AstraZeneca, Bristol Myers Squibb, Celgene, CTI, Daiichi, Incyte, Janssen, Kartos, Novartis, NS-Pharma, and Oncotherapy Sciences. P.L.G. received research funding (institutional) from Celgene/Bristol Myers Squibb and Gilead. P.F. received research funding from Bristol Myers Squibb, AbbVie, Jazz Pharmaceuticals, Novartis, and Janssen, and had consultancy with and received honoraria from Bristol Myers Squibb, AbbVie, Jazz Pharmaceuticals, and Novartis. M.S. consulted for Curis Oncology and Boston Consulting; served on the advisory boards for Novartis and Kymera; and participated in the GME activity for Novartis, Curis Oncology, Haymarket Media, and Clinical Care Options (CCO). G.F.S. received grants/research support (institutional) from Bristol Myers Squibb, Novartis, and Gamida Cell. R.M. is on the Board of Directors of CircBio; on the advisory boards of Kodikaz Therapeutic Solutions, Syros Pharmaceuticals, and TenSixteen Bio; is an inventor on a number of patents related to CD47 cancer immunotherapy licensed to Gilead Sciences; received research support from the Gilead Sciences and CircBio; and is the cofounder and equity holder of CircBio, Pheast Therapeutics, MyeloGene, and RNAC Therapeutics. G.F.S. received honoraria, advisory board membership, or consultation fees from AbbVie, Bristol Myers Squibb, Novartis, Roche, and Takeda, and participated in the sponsored speaker’s bureau for Bristol Myers Squibb, Novartis, and Takeda. A.N. owns stock at Amazon and is an employee of Incyte Pharmaceuticals. D.T.S. is a consultant and received research funding from Kymera Therapeutics, Kurome Therapeutics, Captor Therapeutics, and Tolero Therapeutics. D.T.S. has equity in Kurome Therapeutics. D.A.S. served on the advisory boards of Aprea, AvenCell, Bluebird Bio, Bristol Myers Squibb, Intellia, Kite, Novartis, Shattuck Labs, Servier, and Syndax; served as a consultant for AbbVie, Magenta, Molecular Partners AG, and Takeda; served on the speakers’ bureau for Bristol Myers Squibb, Incyte, and Servier; and received research funding from Aprea and Jazz Pharmaceuticals. M.M.P. received research funding from Kura Oncology and StemLine Pharmaceuticals. A.M.B. received consulting or advisory board honoraria from Novartis, Acceleron, Agios, AbbVie, Takeda, Celgene/Bristol Myers Squibb, Keros Therapeutics, Taiho, and Gilead, and has research support from NIH SPORE in Myeloid Malignancies and the Edward P. Evans Foundation. E.P. receives research funding from Incyte and Bristol Myers Squibb, and honoraria from Stemline, Taiho, Blueprint, and Bristol Myers Squibb. T.K.K. received research funding from Nextcure and is a consultant for the Agenus. R.S.K. served on the speaker bureau of Jazz Pharmaceuticals, Servio, CTI, and PharmaEssentia, and served on advisory boards and received honoraria from Bristol Myers Squibb, Novartis, AbbVie, Jazz Pharmaceuticals, Servio, PharmaEssentia, Taiho, Geron, and CTI. M.S.A. has served on advisory boards for Bristol Myers Squibb, Novartis, Kurome, and Gilead. R.B. is employed by and has equity in Aptose Biosciences, and serves on independent drug monitoring committees for Gilead and Epizyme. R.P.H. has a consultancy agreement with Bluebird Bio. G.J.R. has a consultancy agreement and/or serves on the advisory board or data and safety monitoring committee for AbbVie, Amgen, Astellas, AstraZeneca, Bristol Myers Squibb, Blueprint Medicines, Bluebird Bio, Celgene, Glaxo SmithKline, Janssen, Jasper Therapeutics, Jazz Pharmaceuticals, Mesoblast, Novartis, Pfizer, Syndax, and Takeda, and received research support from Janssen. S.H. received honoraria from Forma Therapeutics. A.H.W. has served on advisory boards for Novartis, AstraZeneca, Astellas, Janssen, Amgen, Roche, Pfizer, AbbVie, Servier, Gilead, Bristol Myers Squibb, Shoreline, Macrogenics, and Agios; receives research funding (to institution) from Novartis, AbbVie, Servier, Janssen, Bristol Myers Squibb, Syndax, Astex, AstraZeneca, and Amgen; serves on speakers' bureaus for AbbVie, Novartis, Bristol Myers Squibb, Servier, and Astellas; is an employee of the Walter and Eliza Hall Institute (WEHI); and is eligible for financial benefits associated with payments that WEHI receives in relation to venetoclax. A.F.L. is employed by and has equity in Precision BioSciences, and has served as a consultant for Halia Therapeutics, CTI Biopharma, and Aileron. M.X. participated in advisory boards and/or had a consultancy agreement with and received honoraria from Seattle Genetics, Pure Marrow and Blueprint Medicines. N.G.D. has received research funding from Daiichi-Sankyo, Bristol Myers Squibb, Pfizer, Gilead, Sevier, Genentech, Astellas, Daiichi-Sankyo, AbbVie, Hanmi, Trovagene, Fate Therapeutics, Amgen, Novimmune, Glycomimetics, Trillium, and ImmunoGen, and has served in a consulting or advisory role for Daiichi-Sankyo, Bristol Myers Squibb, Arog, Pfizer, Novartis, Jazz, Celgene, AbbVie, Astellas, Genentech, ImmunoGen, Servier, Syndax, Trillium, Gilead, Amgen, Shattuck Labs, and Agios. S.L. received research support from Astellas and Amgen; owns stock from AbbVie; and has received consultancy fees/honoraria from AbbVie, Gerson Lehrman Group, QualWorld, and Guidepoint. E.A.G. received honoraria from AbbVie, Alexion Pharmaceuticals, Genentech, Novartis, CTI Biopharma, Apellis, Celgene/Bristol Myers Squibb, Takeda Oncology, Taiho Oncology, Physician Educational Resource, MediCom Worldwide, American Society of Hematology, Picnic Health, and AAMDSIF, and research support (institutional) from Astex Pharmaceuticals, Genentech, Blueprint Medicine, Alexion Pharmaceuticals, Apellis, Bristol Myers Squibb/Celgene, and Celldex Therapeutics. M.R.S. serves on a board or advisory committee for AbbVie, Bristol Myers Squibb, CTI, Forma, Geron, Karyopharm, Novartis, Ryvu, Sierra Oncology, Taiho, Takeda, and TG Therapeutics; receives research funding from ALX Oncology, Astex, Incyte, Takeda, and TG Therapeutics; and has equity ownership in Karyopharm and Ryvu. O.A.W. has served as a consultant for H3B Biomedicine, Foundation Medicine Inc., Merck, Prelude Therapeutics, and Janssen; is on the scientific advisory board of Envisagenics Inc., AIChemy, Harmonic Discovery Inc., and Pfizer Boulder; and has received prior research funding from H3B Biomedicine and LOXO Oncology, unrelated to the manuscript. The remaining authors declare no competing financial interests.

References

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

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

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