NCCN Guidelines® Insights: Myelodysplastic Syndromes, Version 3.2022

Featured Updates to the NCCN Guidelines

Authors:
Peter L. Greenberg Stanford Cancer Institute;

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Richard M. Stone Dana-Farber/Brigham and Women’s Cancer Center;

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Aref Al-Kali Mayo Clinic Cancer Center;

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John M. Bennett University of Rochester;

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Uma Borate The Ohio State University Comprehensive Cancer Center – James Cancer Hospital and Solove Research Institute;

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Andrew M. Brunner Massachusetts General Hospital Cancer Center;

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Wanxing Chai-Ho UCLA Jonsson Comprehensive Cancer Center;

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Peter Curtin City of Hope National Medical Center;

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Carlos M. de Castro Duke Cancer Institute;

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H. Joachim Deeg Fred Hutchinson Cancer Research Center/Seattle Cancer Care Alliance;

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Amy E. DeZern The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins;

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Shira Dinner Robert H. Lurie Comprehensive Cancer Center of Northwestern University;

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Charles Foucar University of Michigan Rogel Cancer Center;

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Karin Gaensler UCSF Helen Diller Family Comprehensive Cancer Center;

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Guillermo Garcia-Manero The University of Texas MD Anderson Cancer Center;

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Elizabeth A. Griffiths Roswell Park Comprehensive Cancer Center;

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David Head Vanderbilt-Ingram Cancer Center;

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Brian A. Jonas UC Davis Comprehensive Cancer Center;

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Sioban Keel Fred Hutchinson Cancer Research Center/Seattle Cancer Care Alliance;

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Yazan Madanat UT Southwestern Simmons Comprehensive Cancer Center;

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Lori J. Maness Fred & Pamela Buffett Cancer Center;

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James Mangan UC San Diego Moores Cancer Center;

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Shannon McCurdy Abramson Cancer Center at the University of Pennsylvania;

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Christine McMahon University of Colorado Cancer Center;

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Bhumika Patel Case Comprehensive Cancer Center/University Hospitals Seidman Cancer Center and Cleveland Clinic Taussig Cancer Institute;

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Vishnu V. Reddy O'Neal Comprehensive Cancer Center at UAB;

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David A. Sallman Moffitt Cancer Center;

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Rory Shallis Yale Cancer Center/Smilow Cancer Hospital;

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Paul J. Shami Huntsman Cancer Institute at the University of Utah;

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Swapna Thota St. Jude Children's Research Hospital/The University of Tennessee Health Science Center;

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Asya Nina Varshavsky-Yanovsky Fox Chase Cancer Center;

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Peter Westervelt Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine; and

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Elizabeth Hollinger National Comprehensive Cancer Network.

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Dorothy A. Shead National Comprehensive Cancer Network.

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Cindy Hochstetler National Comprehensive Cancer Network.

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The NCCN Guidelines for Myelodysplastic Syndromes (MDS) provide recommendations for the evaluation, diagnosis, and management of patients with MDS based on a review of clinical evidence that has led to important advances in treatment or has yielded new information on biologic factors that may have prognostic significance in MDS. The multidisciplinary panel of MDS experts meets on an annual basis to update the recommendations. These NCCN Guidelines Insights focus on some of the updates for the 2022 version of the NCCN Guidelines, which include treatment recommendations both for lower-risk and higher-risk MDS, emerging therapies, supportive care recommendations, and genetic familial high-risk assessment for hereditary myeloid malignancy predisposition syndromes.

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  • 1.

    Beck DB, Ferrada MA, Sikora KA, et al. Somatic mutations in UBA1 and severe adult-onset autoinflammatory disease. N Engl J Med 2020;383:26282638.

  • 2.

    National Cancer Institute. SEER cancer statistics review 1975–2016: myelodysplastic syndromes (MDS), chronic myeloproliferative disorders (CMD), and chronic myelomonocytic leukemia (CMML). Accessed January 8, 2020. Available at: https://seer.cancer.gov/csr/1975_2016/browse_csr.php?sectionSEL=30&pageSEL=sect_30_intro.01

    • PubMed
    • Search Google Scholar
    • Export Citation
  • 3.

    Zeidan AM, Shallis RM, Wang R, et al. Epidemiology of myelodysplastic syndromes: why characterizing the beast is a prerequisite to taming it. Blood Rev 2019;34:115.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 4.

    Greenberg PL. The myelodysplastic syndromes. In: Hoffman R, Benz E, Shattil S, et al., eds. Hematology: Basic Principles and Practice. 3rd ed. New York, NY: Churchill Livingstone; 2000:11061129.

    • PubMed
    • Search Google Scholar
    • Export Citation
  • 5.

    Pfeilstöcker M, Tuechler H, Sanz G, et al. Time-dependent changes in mortality and transformation risk in MDS. Blood 2016;128: 902910.

  • 6.

    Garcia-Manero G, Griffiths EA, Steensma DP, et al. Oral cedazuridine/decitabine for MDS and CMML: a phase 2 pharmacokinetic/pharmacodynamic randomized crossover study. Blood 2020;136:674683.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 7.

    Townsley DM, Scheinberg P, Winkler T, et al. Eltrombopag added to standard immunosuppression for aplastic anemia. N Engl J Med 2017;376:15401550.

  • 8.

    Assi R, Garcia-Manero G, Ravandi F, et al. Addition of eltrombopag to immunosuppressive therapy in patients with newly diagnosed aplastic anemia. Cancer 2018;124:41924201.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 9.

    Hashimoto S, Toba K, Fuse I, et al. Thrombopoietin activates the growth of megakaryoblasts in patients with chronic myeloproliferative disorders and myelodysplastic syndrome. Eur J Haematol 2000;64:225230.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 10.

    Luo SS, Ogata K, Yokose N, et al. Effect of thrombopoietin on proliferation of blasts from patients with myelodysplastic syndromes. Stem Cells 2000;18:112119.

  • 11.

    Oliva EN, Alati C, Santini V, et al. Eltrombopag versus placebo for low-risk myelodysplastic syndromes with thrombocytopenia (EQoL-MDS): phase 1 results of a single-blind, randomised, controlled, phase 2 superiority trial. Lancet Haematol 2017;4:e127136.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 12.

    Vicente A, Patel BA, Gutierrez-Rodrigues F, et al. Eltrombopag monotherapy can improve hematopoiesis in patients with low to intermediate risk-1 myelodysplastic syndrome. Haematologica 2020;105:27852794.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 13.

    Fan X, Desmond R, Winkler T, et al. Eltrombopag for patients with moderate aplastic anemia or uni-lineage cytopenias. Blood Adv 2020;4:17001710.

  • 14.

    Platzbecker U, Kiewe P, Germing U, et al. MDS 191: long-term efficacy and safety of luspatercept in lower-risk myelodysplastic syndromes (MDS): phase 2 PACE-MDS study [abstract]. Clin Lymphoma Myeloma Leuk 2020;20(Suppl):Abstract MDS-191.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 15.

    Steensma DP, Fenaux P, Van Eygen K, et al. Imetelstat achieves meaningful and durable transfusion independence in high transfusion-burden patients with lower-risk myelodysplastic syndromes in a phase II study. J Clin Oncol 2021;39:4856.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 16.

    Fong CY, Wei AH, Frattini MG, et al. Phase 1b study of venetoclax in combination with azacitidine in patients with treatment-naïve higher-risk myelodysplastic syndromes [abstract]. J Clin Oncol 2018;36(Suppl):Abstract TPS7082.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 17.

    Garcia JS, Wei AH, Borate U, et al. Safety, efficacy, and patient-reported outcomes of venetoclax in combination with azacitidine for the treatment of patients with higher-risk myelodysplastic syndrome: a phase 1b study [abstract]. Blood 2020;136(Suppl):5557.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 18.

    Zeidan AM, Pollyea DA, Garcia JS, et al. A phase 1b study evaluating the safety and efficacy of venetoclax as monotherapy or in combination with azacitidine for the treatment of relapsed/refractory myelodysplastic syndrome [abstract]. Blood 2019;134(Suppl):Abstract 565.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 19.

    DiNardo CD, Stein EM, de Botton S, et al. Durable remissions with ivosidenib in IDH1-mutated relapsed or refractory AML. N Engl J Med 2018;378:23862398.

  • 20.

    Thol F, Weissinger EM, Krauter J, et al. IDH1 mutations in patients with myelodysplastic syndromes are associated with an unfavorable prognosis. Haematologica 2010;95:16681674.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 21.

    Kosmider O, Gelsi-Boyer V, Slama L, et al. Mutations of IDH1 and IDH2 genes in early and accelerated phases of myelodysplastic syndromes and MDS/myeloproliferative neoplasms. Leukemia 2010;24:10941096.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 22.

    Patnaik MM, Hanson CA, Hodnefield JM, et al. Differential prognostic effect of IDH1 versus IDH2 mutations in myelodysplastic syndromes: a Mayo Clinic study of 277 patients. Leukemia 2012;26:101105.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 23.

    Stein EM, Fathi AT, DiNardo CD, et al. Enasidenib in patients with mutant IDH2 myelodysplastic syndromes: a phase 1 subgroup analysis of the multicentre, AG221-C-001 trial. Lancet Haematol 2020; 7:e309319.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 24.

    Venugopal S, Dinardo CD, Takahashi K, et al. Phase II study of the IDH2-inhibitor enasidenib in patients with high-risk IDH2-mutated myelodysplastic syndromes (MDS) [abstract]. J Clin Oncol 2021;39(Suppl):Abstract 7010.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 25.

    Fenaux P, Mufti GJ, Hellstrom-Lindberg E, et al. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol 2009;10:223232.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 26.

    Nakamura R, Saber W, Martens MJ, et al. A multi-center biologic assignment trial comparing reduced intensity allogeneic hematopoietic cell transplantation to hypomethylating therapy or best supportive care in patients aged 50-75 with advanced myelodysplastic syndrome: Blood and Marrow Transplant Clinical Trials Network study 1102 [abstract]. Blood 2020;136(Suppl):1921.

    • PubMed
    • Search Google Scholar
    • Export Citation
  • 27.

    Nakamura R, Saber W, Martens MJ, et al. Biologic assignment trial of reduced-intensity hematopoietic cell transplantation based on donor availability in patients 50-75 years of age with advanced myelodysplastic syndrome. J Clin Oncol 2021;39:33283339.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 28.

    Schroeder T, Wegener N, Lauseker M, et al. Comparison between upfront transplantation and different pretransplant cytoreductive treatment approaches in patients with high-risk myelodysplastic syndrome and secondary acute myelogenous leukemia. Biol Blood Marrow Transplant 2019;25:15501559.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 29.

    Festuccia M, Deeg HJ, Gooley TA, et al. Minimal identifiable disease and the role of conditioning intensity in hematopoietic cell transplantation for myelodysplastic syndrome and acute myelogenous leukemia evolving from myelodysplastic syndrome. Biol Blood Marrow Transplant 2016;22:12271233.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 30.

    Majhail NS, Farnia SH, Carpenter PA, et al. Indications for autologous and allogeneic hematopoietic cell transplantation: guidelines from the American Society for Blood and Marrow Transplantation. Biol Blood Marrow Transplant 2015;21:18631869.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 31.

    Sarasin A, Quentin S, Droin N, et al. Familial predisposition to TP53/complex karyotype MDS and leukemia in DNA repair-deficient xeroderma pigmentosum. Blood 2019;133:27182724.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 32.

    Oetjen KA, Levoska MA, Tamura D, et al. Predisposition to hematologic malignancies in patients with xeroderma pigmentosum. Haematologica 2020;105:e144146.

  • 33.

    Tummala H, Kirwan M, Walne AJ, et al. ERCC6L2 mutations link a distinct bone-marrow-failure syndrome to DNA repair and mitochondrial function. Am J Hum Genet 2014;94:246256.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 34.

    Tummala H, Dokal AD, Walne A, et al. Genome instability is a consequence of transcription deficiency in patients with bone marrow failure harboring biallelic ERCC6L2 variants. Proc Natl Acad Sci USA 2018;115:77777782.

    • Crossref
    • PubMed
    • Search Google Scholar
    • Export Citation
  • 35.

    Douglas SPM, Siipola P, Kovanen PE, et al. ERCC6L2 defines a novel entity within inherited acute myeloid leukemia. Blood 2019;133:27242728.

  • 36.

    Chistiakov DA, Voronova NV, Chistiakov AP. Ligase IV syndrome. Eur J Med Genet 2009;52:373378.

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