Myelodysplastic syndrome

References

Key articles

Killick SB, Wiseman DH, Quek L, et al. British Society for Haematology guidelines for the diagnosis and evaluation of prognosis of adult myelodysplastic syndromes. Br J Haematol. 2021 Jul;194(2):282-93.Full text  Abstract

Killick SB, Ingram W, Culligan D, et al. British Society for Haematology guidelines for the management of adult myelodysplastic syndromes. Br J Haematol. 2021 Jul;194(2):267-81.Full text  Abstract

National Comprehensive Cancer Network. NCCN clinical practice guidelines in oncology: myelodysplastic syndromes [internet publication].Full text

Fenaux P, Haase D, Santini V, et al; ESMO Guidelines Committee. Myelodysplastic syndromes: ESMO clinical practice guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2021 Feb;32(2):142-56.Full text  Abstract

Reference articles

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12. Killick SB, Ingram W, Culligan D, et al. British Society for Haematology guidelines for the management of adult myelodysplastic syndromes. Br J Haematol. 2021 Jul;194(2):267-81.Full text  Abstract

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17. Fenaux P, Haase D, Santini V, et al; ESMO Guidelines Committee. Myelodysplastic syndromes: ESMO clinical practice guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2021 Feb;32(2):142-56.Full text  Abstract

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32. Sun L, Babushok DV. Secondary myelodysplastic syndrome and leukemia in acquired aplastic anemia and paroxysmal nocturnal hemoglobinuria. Blood. 2020 Jul 2;136(1):36-49.Full text  Abstract

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36. Morton LM, Dores GM, Schonfeld SJ, et al. Association of chemotherapy for solid tumors with development of therapy-related myelodysplastic syndrome or acute myeloid leukemia in the modern era. JAMA Oncol. 2019 Mar 1;5(3):318-25.Full text  Abstract

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43. Gañán-Gómez I, Wei Y, Starczynowski DT, et al. Deregulation of innate immune and inflammatory signaling in myelodysplastic syndromes. Leukemia. 2015 Jul;29(7):1458-69. Abstract

44. Zhan D, Park CY. Stem cells in the myelodysplastic syndromes. Front Aging. 2021;2:719010.Full text  Abstract

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48. Garcia-Manero G. Myelodysplastic syndromes: 2023 update on diagnosis, risk-stratification, and management. Am J Hematol. 2023 Aug;98(8):1307-25.Full text  Abstract

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51. van de Loosdrecht AA, Kern W, Porwit A, et al. Clinical application of flow cytometry in patients with unexplained cytopenia and suspected myelodysplastic syndrome: a report of the European LeukemiaNet International MDS-Flow Cytometry Working Group. Cytometry B Clin Cytom. 2023 Jan;104(1):77-86.Full text  Abstract

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54. Germing U, Hildebrandt B, Pfeilstöcker M, et al. Refinement of the international prognostic scoring system (IPSS) by including LDH as an additional prognostic variable to improve risk assessment in patients with primary myelodysplastic syndromes (MDS). Leukemia. 2005 Dec;19(12):2223-31. Abstract

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61. Bernard E, Tuechler H, Greenberg PL, et al. Molecular international prognostic scoring system for myelodysplastic syndromes. NEJM Evid. 2022;1(7).Full text

62. Greenberg PL, Tuechler H, Schanz J, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012 Sep 20;120(12):2454-65.Full text  Abstract

63. Malcovati L, Germing U, Kuendgen A, et al. Time-dependent prognostic scoring system for predicting survival and leukemic evolution in myelodysplastic syndromes. J Clin Oncol. 2007 Aug 10;25(23):3503-10.Full text  Abstract

64. Arber DA, Orazi A, Hasserjian R, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016 May 19;127(20):2391-405.Full text  Abstract

65. Greenberg P, Cox C, LeBeau MM, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997 Mar 15;89(6):2079-88. [Erratum in: Blood. 1998;91:1100.]Full text  Abstract

66. Valent P, Orazi A, Steensma DP, et al. Proposed minimal diagnostic criteria for myelodysplastic syndromes (MDS) and potential pre-MDS conditions. Oncotarget. 2017 Sep 26;8(43):73483-500.Full text  Abstract

67. Nordic MDS Group. Guidelines for the diagnosis and treatment of myelodysplastic syndromes and chronic myelomonocytic leukemia (10th update). Dec 2021 [internet publication].Full text

68. Cross NCP, Godfrey AL, Cargo C, et al. Good practice papers: the use of genetic tests to diagnose and manage patients with myeloproliferative and myeloproliferative/myelodysplastic neoplasms, and related disorders. Br J Haematol. 2021 Nov;195(3):338-51.Full text  Abstract

69. Pfeilstöcker M, Tuechler H, Sanz G, et al. Time-dependent changes in mortality and transformation risk in MDS. Blood. 2016 Aug;128(7):902-10.Full text  Abstract

70. Schanz J, Tüchler H, Solé F, et al. New comprehensive cytogenetic scoring system for primary myelodysplastic syndromes (MDS) and oligoblastic acute myeloid leukemia after MDS derived from an international database merge. J Clin Oncol. 2012 Mar;30(8):820-9.Full text  Abstract

71. Schiffer CA, Bohlke K, Delaney M, et al. Platelet transfusion for patients with cancer: American Society of Clinical Oncology clinical practice guideline update. J Clin Oncol. 2018 Jan;36(3):283-99.Full text  Abstract

72. Moyo V, Lefebvre P, Duh MS, et al. Erythropoiesis-stimulating agents in the treatment of anemia in myelodysplastic syndromes: a meta-analysis. Ann Hematol. 2008 Jul;87(7):527-36. Abstract

73. Platzbecker U, Symeonidis A, Oliva EN, et al. A phase 3 randomized placebo-controlled trial of darbepoetin alfa in patients with anemia and lower-risk myelodysplastic syndromes. Leukemia. 2017 Sep;31(9):1944-50.Full text  Abstract

74. Srinivasan S, Schiffer CA. Current treatment options and strategies for myelodysplastic syndromes. Expert Opin Pharmacother. 2008 Jul;9(10):1667-78. Abstract

75. Hutzschenreuter F, Monsef I, Kreuzer KA, et al. Granulocyte and granulocyte-macrophage colony stimulating factors for newly diagnosed patients with myelodysplastic syndromes. Cochrane Database Syst Rev. 2016 Feb;2(2):CD009310.Full text  Abstract

76. Jädersten M, Malcovati L, Dybedal I, et al. Erythropoietin and granulocyte-colony stimulating factor treatment associated with improved survival in myelodysplastic syndrome. J Clin Oncol. 2008 Jul;26(21):3607-13.Full text  Abstract

77. 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 Aug 6;136(6):674-83.Full text  Abstract

78. Jabbour E, Short NJ, Montalban-Bravo G, et al. Randomized phase 2 study of low-dose decitabine vs low-dose azacitidine in lower-risk MDS and MDS/MPN. Blood. 2017 Sep;130(13):1514-22.Full text  Abstract

79. Garcia-Manero G, Jabbour E, Borthakur G, et al. Randomized open-label phase II study of decitabine in patients with low- or intermediate-risk myelodysplastic syndromes. J Clin Oncol. 2013 Jul;31(20):2548-53.Full text  Abstract

80. Sasaki K, Jabbour E, Montalban-Bravo G, et al. Low-dose decitabine versus low-dose azacitidine in lower-risk MDS. NEJM Evid. 2022;1(10).Full text

81. Passweg JR, Giagounidis AA, Simcock M, et al. Immunosuppressive therapy for patients with myelodysplastic syndrome: a prospective randomized multicenter phase III trial comparing antithymocyte globulin plus cyclosporine with best supportive care--SAKK 33/99. J Clin Oncol. 2011 Jan;29(3):303-9.Full text  Abstract

82. Sloand EM, Wu CO, Greenberg P, et al. Factors affecting response and survival in patients with myelodysplasia treated with immunosuppressive therapy. J Clin Oncol. 2008 May;26(15):2505-11.Full text  Abstract

83. Saunthararajah Y, Nakamura R, Wesley R, et al. A simple method to predict response to immunosuppressive therapy in patients with myelodysplastic syndrome. Blood. 2003 Oct;102(8):3025-7.Full text  Abstract

84. Parikh AR, Olnes MJ, Barrett AJ. Immunomodulatory treatment of myelodysplastic syndromes: antithymocyte globulin, cyclosporine, and alemtuzumab. Semin Hematol. 2012 Oct;49(4):304-11.Full text  Abstract

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86. Stahl M, DeVeaux M, de Witte T, et al. The use of immunosuppressive therapy in MDS: clinical outcomes and their predictors in a large international patient cohort. Blood Adv. 2018 Jul;2(14):1765-72.Full text  Abstract

87. Fenaux P, Platzbecker U, Mufti GJ, et al. Luspatercept in patients with lower-risk myelodysplastic syndromes. N Engl J Med. 2020 Jan 9;382(2):140-51.Full text  Abstract

88. Sekeres MA. Lenalidomide in MDS: 4th time's a charm. Blood. 2011 Oct 6;118(14):3757-8. Abstract

89. Fenaux P, Giagounidis A, Selleslag D, et al. A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q. Blood. 2011 Oct 6;118(14):3765-76. Abstract

90. Leitch HA, Buckstein R, Shamy A, et al. The immunomodulatory agents lenalidomide and thalidomide for treatment of the myelodysplastic syndromes: a clinical practice guideline. Crit Rev Oncol Hematol. 2013 Feb;85(2):162-92. Abstract

91. List A, Dewald G, Bennett J, et al. Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion. N Engl J Med. 2006 Oct 5;355(14):1456-65.Full text  Abstract

92. Giagounidis A, Mufti GJ, Mittelman M, et al. Outcomes in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with isolated deletion 5q treated with lenalidomide: a subset analysis from the MDS-004 study. Eur J Haematol. 2014 Nov;93(5):429-38.Full text  Abstract

93. Cutler CS, Lee SJ, Greenberg P, et al. A decision analysis of allogeneic bone marrow transplantation for the myelodysplastic syndromes: delayed transplantation for low-risk myelodysplasia is associated with improved outcome. Blood. 2004 Jul 15;104(2):579-85. Abstract

94. Oliansky DM, Antin JH, Bennett JM, et al. The role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of myelodysplastic syndromes: an evidence-based review. Biol Blood Marrow Transplant. 2009 Feb;15(2):137-72. Abstract

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96. 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 Mar;10(3):223-32.Full text  Abstract

97. Zeidan AM, Davidoff AJ, Long JB, et al. Comparative clinical effectiveness of azacitidine versus decitabine in older patients with myelodysplastic syndromes. Br J Haematol. 2016 Dec;175(5):829-40.Full text  Abstract

98. Estey EH, Thall PF, Cortes JE, et al. Comparison of idarubicin + ara-C-, fludarabine + ara-C-, and topotecan + ara-C-based regimens in treatment of newly diagnosed acute myeloid leukemia, refractory anemia with excess blasts in transformation, or refractory anemia with excess blasts. Blood. 2001 Dec 15;98(13):3575-83.Full text  Abstract

99. Wei AH, Garcia JS, Borate U, et al. A phase 1b study evaluating the safety and efficacy of venetoclax in combination with azacitidine in treatment-naïve patients with higher-risk myelodysplastic syndrome. Blood. 2019;134(1 suppl):568.Full text

100. Zeidan AM, Borate U, Pollyea DA, et al. A phase 1b study of venetoclax and azacitidine combination in patients with relapsed or refractory myelodysplastic syndromes. Am J Hematol. 2023 Feb;98(2):272-81.Full text  Abstract

101. Kantarjian HM, Giles FJ, Greenberg PL, et al. Phase 2 study of romiplostim in patients with low- or intermediate-risk myelodysplastic syndrome receiving azacitidine therapy. Blood. 2010 Oct 28;116(17):3163-70.Full text  Abstract

102. Giagounidis A, Mufti GJ, Fenaux P, et al. Results of a randomized, double-blind study of romiplostim versus placebo in patients with low/intermediate-1-risk myelodysplastic syndrome and thrombocytopenia. Cancer. 2014 Jun 15;120(12):1838-46.Full text  Abstract

103. Oliva EN, Riva M, Niscola P, et al. Eltrombopag for low-risk myelodysplastic syndromes with thrombocytopenia: interim results of a phase-II, randomized, placebo-controlled clinical trial (EQOL-MDS). J Clin Oncol. 2023 Jun:JCO2202699.Full text  Abstract

104. 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 Mar;4(3):e127-36. Abstract

105. 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 Dec;105(12):2785-94.Full text  Abstract

106. Mittelman M, Platzbecker U, Afanasyev B, et al. Eltrombopag for advanced myelodysplastic syndromes or acute myeloid leukaemia and severe thrombocytopenia (ASPIRE): a randomised, placebo-controlled, phase 2 trial. Lancet Haematol. 2018 Jan;5(1):e34-43.Full text  Abstract

107. 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 Apr;7(4):e309-19. Abstract

108. Foran JM, DiNardo CD, Watts JM, et al. Ivosidenib (AG-120) in patients with IDH1-mutant relapsed/refractory myelodysplastic syndrome: updated enrollment of a phase 1 dose escalation and expansion study. Paper presented at: 61st American Society of Hematology Annual Meeting. 7-10 Dec 2019. Orlando, FL. Poster abstracts. 637. Myelodysplastic syndromes - clinical studies. Blood. 2019 Nov 13;134(Suppl 1):4254.Full text

109. Sebert M, Cluzeau T, Rauzy, OB, et al. Ivosidenib monotherapy is effective in patients with IDH1 mutated myelodysplastic syndrome (MDS): the idiome phase 2 study By the GFM group. Blood. 2021;138(1 suppl):62.Full text

110. 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 Jan;39(1):48-56.Full text  Abstract

111. Zeidan AM, Platzbecker U, Santini V, et al. IMerge: results from a phase 3, randomized, double-blind, placebo-controlled study of imetelstat in patients (pts) with heavily transfusion dependent (TD) non-del(5q) lower-risk myelodysplastic syndromes (LR-MDS) relapsed/refractory (R/R) to erythropoiesis stimulating agents (ESA).J Clin Oncol. 2023;41(16 suppl):7004.Full text

112. Lee S, Mohan S, Knupp J, et al. Oral eltanexor treatment of patients with higher-risk myelodysplastic syndrome refractory to hypomethylating agents. J Hematol Oncol. 2022 Aug;15(1):103.Full text  Abstract

113. ClinicalTrials.gov. Tamibarotene plus azacitidine in participants with newly diagnosed RARA-positive higher-risk myelodysplastic syndrome. NCT04797780. Aug 2022 [internet publication].Full text

114. Dezern AE, Marconi G, Deeren D, et al. A randomized, double-blind, placebo-controlled study of tamibarotene/azacitidine versus placebo/azacitidine in newly diagnosed adult patients selected for RARA+ HR-MDS (SELECT-MDS-1). Paper presented at: 2022 American Society of Clinical Oncology Annual Meeting. 3-7 Jun 2022. Chicago, IL/virtual. Hematologic malignancies - leukemia, myelodysplastic syndromes, and allotransplant. Meeting abstract TPS7075. J Clin Oncol. 2022;40(16 Suppl):TPS7075.Full text

115. ClinicalTrials.gov. Magrolimab + azacitidine versus azacitidine + placebo in untreated participants with myelodysplastic syndrome (MDS) (ENHANCE). NCT04313881. Aug 2022 [internet publication].Full text

116. Garcia-Manero G, Daver NG, Xu J, et al. Magrolimab + azacitidine versus azacitidine + placebo in untreated higher risk (HR) myelodysplastic syndrome (MDS): the phase 3, randomized, ENHANCE study. Paper presented at: 2021 American Society of Clinical Oncology Annual Meeting. 4-8 Jun 2021. Virtual. Hematologic malignancies - leukemia, myelodysplastic syndromes, and allotransplant. Meeting abstract TPS7055. J Clin Oncol. 2021;39(15 Suppl):TPS7055.Full text

117. Haider M, Duncavage EJ, Afaneh KF, et al. New insight into the biology, risk stratification, and targeted treatment of myelodysplastic syndromes. Am Soc Clin Oncol Educ Book. 2018 Oct 29;37:480-94.Full text  Abstract

118. Jensen PD, Heickendorff L, Pedersen B, et al. The effect of iron chelation on haemopoiesis in MDS patients with transfusional iron overload. Br J Haematol. 1996 Aug;94(2):288-99. Abstract

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