Síndrome mielodisplásico

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.Texto completo  Resumen

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.Texto completo  Resumen

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

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.Texto completo  Resumen

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3. Steensma DP, Bennett JM. The myelodysplastic syndromes: diagnosis and treatment. Mayo Clin Proc. 2006 Jan;81(1):104-30. Resumen

4. Meyers CA, Albitar M, Estey E. Cognitive impairment, fatigue, and cytokine levels in patients with acute myelogenous leukemia or myelodysplastic syndrome. Cancer. 2005 Aug 15;104(4):788-93. Resumen

5. Pomeroy C, Oken MM, Rydell RE, et al. Infection in the myelodysplastic syndromes. Am J Med. 1991 Mar;90(3):338-44. Resumen

6. Wolach O, Stone R. Autoimmunity and inflammation in myelodysplastic syndromes. Acta Haematol. 2016;136(2):108-17.Texto completo  Resumen

7. Komrokji RS, Kulasekararaj A, Al Ali NH, et al. Autoimmune diseases and myelodysplastic syndromes. Am J Hematol. 2016 May;91(5):E280-3.Texto completo  Resumen

8. Enright H, Jacobs HS, Vercellotti G, et al. Paraneoplastic autoimmune phenomena in patients with myelodysplastic syndromes: response to immunosuppressive therapy. Br J Haematol. 1995 Oct;91(2):403-8. Resumen

9. Sallman DA, Padron E. Myelodysplasia in younger adults: outlier or unique molecular entity? Haematologica. 2017 Jun;102(6):967-8.Texto completo  Resumen

10. Hirsch CM, Przychodzen BP, Radivoyevitch T, et al. Molecular features of early onset adult myelodysplastic syndrome. Haematologica. 2017 Jun;102(6):1028-34.Texto completo  Resumen

11. 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.Texto completo  Resumen

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.Texto completo  Resumen

13. Roman E, Smith A, Appleton S, et al. Myeloid malignancies in the real-world: occurrence, progression and survival in the UK's population-based Haematological Malignancy Research Network 2004-15. Cancer Epidemiol. 2016 Jun;42:186-98.Texto completo  Resumen

14. National Cancer Institute; Surveillance, Epidemiology, and End Results program (SEER). SEER*Explorer: an interactive website for SEER cancer statistics. Apr 2023 [internet publication].Texto completo

15. Ma X. Epidemiology of myelodysplastic syndromes. Am J Med. 2012 Jul;125(7 suppl):S2-5.Texto completo  Resumen

16. National Comprehensive Cancer Network. NCCN clinical practice guidelines in oncology: myelodysplastic syndromes [internet publication].Texto completo

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.Texto completo  Resumen

18. Xavier AC, Kutny M, Costa LJ. Incidence and outcomes of paediatric myelodysplastic syndrome in the United States. Br J Haematol. 2018 Mar;180(6):898-901.Texto completo  Resumen

19. Sperling AS, Gibson CJ, Ebert BL. The genetics of myelodysplastic syndrome: from clonal haematopoiesis to secondary leukaemia. Nat Rev Cancer. 2017 Jan;17(1):5-19. Resumen

20. Chen B, Zhao WL, Jin J, et al. Clinical and cytogenetic features of 508 Chinese patients with myelodysplastic syndrome and comparison with those in Western countries. Leukemia. 2005 May;19(5):767-75. Resumen

21. Haase D, Germing U, Schanz J, et al. New insights into the prognostic impact of the karyotype in MDS and correlation with subtypes: evidence from a core dataset of 2124 patients. Blood. 2007 Dec 15;110(13):4385-95.Texto completo  Resumen

22. Smith SM, Le Beau MM, Huo D, et al. Clinical-cytogenetic associations in 306 patients with therapy-related myelodysplasia and myeloid leukemia: the University of Chicago series. Blood. 2003 Jul 1;102(1):43-52.Texto completo  Resumen

23. Saygin C, Godley LA. Genetics of Myelodysplastic Syndromes. Cancers (Basel). 2021 Jul;13(14):3380.Texto completo  Resumen

24. Papaemmanuil E, Gerstung M, Malcovati L, et al. Clinical and biological implications of driver mutations in myelodysplastic syndromes. Blood. 2013 Nov;122(22):3616-27; quiz 3699.Texto completo  Resumen

25. Haferlach T, Nagata Y, Grossmann V, et al. Landscape of genetic lesions in 944 patients with myelodysplastic syndromes. Leukemia. 2014 Feb;28(2):241-7.Texto completo  Resumen

26. Wang SA, Ok CY, Kim AS, et al. Myelodysplastic syndromes with no somatic mutations detected by next-generation sequencing display similar features to myelodysplastic syndromes with detectable mutations. Am J Hematol. 2021 Nov;96(11):E420-3.Texto completo  Resumen

27. Oetjen KA, Levoska MA, Tamura D, et al. Predisposition to hematologic malignancies in patients with xeroderma pigmentosum. Haematologica. 2020 Apr;105(4):e144-6.Texto completo  Resumen

28. Aktas D, Koc A, Boduroglu K, et al. Myelodysplastic syndrome associated with monosomy 7 in a child with Bloom syndrome. Cancer Genet Cytogenet. 2000 Jan;116(1):44-6. Resumen

29. Sutton JF, Stacey M, Kearns WG, et al. Increased risk for aplastic anemia and myelodysplastic syndrome in individuals lacking glutathione S-transferase genes. Pediatr Blood Cancer. 2004 Feb;42(2):122-6.Texto completo  Resumen

30. Chen H, Sandler DP, Taylor JA, et al. Increased risk for myelodysplastic syndromes in individuals with glutathione transferase theta 1 (GSTT1) gene defect. Lancet. 1996 Feb 3;347(8997):295-7.Texto completo  Resumen

31. Dutzmann CM, Spix C, Popp I, et al. Cancer in children with Fanconi anemia and ataxia-telangiectasia - a nationwide register-based cohort study in Germany. J Clin Oncol. 2022 Jan;40(1):32-9.Texto completo  Resumen

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.Texto completo  Resumen

33. Tong H, Hu C, Yin X, et al. A meta-analysis of the relationship between cigarette smoking and incidence of myelodysplastic syndromes. PLoS One. 2013;8(6):e67537.Texto completo  Resumen

34. Schnatter AR, Glass DC, Tang G, et al. Myelodysplastic syndrome and benzene exposure among petroleum workers: an international pooled analysis. J Natl Cancer Inst. 2012 Nov;104(22):1724-37.Texto completo  Resumen

35. Mundt KA, Dell LD, Boffetta P, et al. The importance of evaluating specific myeloid malignancies in epidemiological studies of environmental carcinogens. BMC Cancer. 2021 Mar;21(1):227.Texto completo  Resumen

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;5(3):318-25.Texto completo  Resumen

37. Sill H, Olipitz W, Zebisch A, et al. Therapy-related myeloid neoplasms: pathobiology and clinical characteristics. Br J Pharmacol. 2011 Feb;162(4):792-805.Texto completo  Resumen

38. Kaplan H, Malmgren J, De Roos AJ. Risk of myelodysplastic syndrome and acute myeloid leukemia post radiation treatment for breast cancer: a population-based study. Breast Cancer Res Treat. 2013 Feb;137(3):863-7. Resumen

39. Kaplan HG, Malmgren JA, Atwood MK. Increased incidence of myelodysplastic syndrome and acute myeloid leukemia following breast cancer treatment with radiation alone or combined with chemotherapy: a registry cohort analysis 1990-2005. BMC Cancer. 2011 Jun 21;11:260.Texto completo  Resumen

40. Leone G, Pagano L, Ben-Yehuda D, et al. Therapy-related leukemia and myelodysplasia: susceptibility and incidence. Haematologica. 2007 Oct;92(10):1389-98.Texto completo  Resumen

41. Stone RM, Neuberg D, Soiffer R, et al. Myelodysplastic syndrome as a late complication following autologous bone marrow transplantation for non-Hodgkin's lymphoma. J Clin Oncol. 1994 Dec;12(12):2535-42. Resumen

42. Janssen JW, Buschle M, Layton M, et al. Clonal analysis of myelodysplastic syndromes: evidence of multipotent stem cell origin. Blood. 1989 Jan;73(1):248-54.Texto completo  Resumen

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. Resumen

44. Zhan D, Park CY. Stem cells in the myelodysplastic syndromes. Front Aging. 2021;2:719010.Texto completo  Resumen

45. Linet MS, Gilbert ES, Vermeulen R, et al; Chinese Center for Disease Control and Prevention - US National Cancer Institute Benzene Study Group. Benzene exposure response and risk of myeloid neoplasms in Chinese workers: a multicenter case-cohort study. J Natl Cancer Inst. 2019 May 1;111(5):465-74.Texto completo  Resumen

46. Yarosh R, Roesler MA, Murray T, et al. Risk factors for de novo and therapy-related myelodysplastic syndromes (MDS). Cancer Causes Control. 2021 Mar;32(3):241-50.Texto completo  Resumen

47. West RR, Stafford DA, Farrow A, et al. Occupational and environmental exposures and myelodysplasia: a case-control study. Leuk Res. 1995 Feb;19(2):127-39. Resumen

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

49. Juneja SK, Imbert M, Jouault H, et al. Haematological features of primary myelodysplastic syndromes (PMDS) at initial presentation: a study of 118 cases. J Clin Pathol. 1983 Oct;36(10):1129-35.Texto completo  Resumen

50. Katsarou O, Terpos E, Patsouris E, et al. Myelodysplastic features in patients with long-term HIV infection and haemophilia. Haemophilia. 2001 Jan;7(1):47-52.Texto completo  Resumen

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.Texto completo  Resumen

52. de Witte T, Bowen D, Robin M, et al. Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel. Blood. 2017 Mar 30;129(13):1753-62.Texto completo  Resumen

53. Wimazal F, Sperr WR, Kundi M, et al. Prognostic value of lactate dehydrogenase activity in myelodysplastic syndromes. Leuk Res. 2001 Apr;25(4):287-94.Texto completo  Resumen

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. Resumen

55. Sanz GF, Sanz MA, Vallespí T, et al. Two regression models and a scoring system for predicting survival and planning treatment in myelodysplastic syndromes: a multivariate analysis of prognostic factors in 370 patients. Blood. 1989 Jul;74(1):395-408.Texto completo  Resumen

56. Toma A, Fenaux P, Dreyfus F, et al. Infections in myelodysplastic syndromes. Haematologica. 2012 Oct;97(10):1459-70.Texto completo  Resumen

57. Malcovati L, Porta MG, Pascutto C, et al. Prognostic factors and life expectancy in myelodysplastic syndromes classified according to WHO criteria: a basis for clinical decision making. J Clin Oncol. 2005 Oct 20;23(30):7594-603.Texto completo  Resumen

58. Carraway HE, Saygin C. Therapy for lower-risk MDS. Hematology Am Soc Hematol Educ Program. 2020 Dec;2020(1):426-33.Texto completo  Resumen

59. Garcia-Manero G, Shan J, Faderl S, et al. A prognostic score for patients with lower risk myelodysplastic syndrome. Leukemia. 2008 Mar;22(3):538-43. Resumen

60. Heaney ML, Golde DW. Myelodysplasia. N Engl J Med. 1999 May 27;340(21):1649-60. Resumen

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

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.Texto completo  Resumen

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.Texto completo  Resumen

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.Texto completo  Resumen

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.]Texto completo  Resumen

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.Texto completo  Resumen

67. 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.Texto completo  Resumen

68. 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.Texto completo  Resumen

69. 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.Texto completo  Resumen

70. 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. Resumen

71. 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.Texto completo  Resumen

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

73. 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.Texto completo  Resumen

74. 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.Texto completo  Resumen

75. 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.Texto completo  Resumen

76. 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.Texto completo  Resumen

77. 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.Texto completo  Resumen

78. 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).Texto completo

79. 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.Texto completo  Resumen

80. 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.Texto completo  Resumen

81. 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.Texto completo  Resumen

82. Parikh AR, Olnes MJ, Barrett AJ. Immunomodulatory treatment of myelodysplastic syndromes: antithymocyte globulin, cyclosporine, and alemtuzumab. Semin Hematol. 2012 Oct;49(4):304-11.Texto completo  Resumen

83. Saunthararajah Y, Nakamura R, Nam JM, et al. HLA-DR15 (DR2) is overrepresented in myelodysplastic syndrome and aplastic anemia and predicts a response to immunosuppression in myelodysplastic syndrome. Blood. 2002 Sep 1;100(5):1570-4.Texto completo  Resumen

84. 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.Texto completo  Resumen

85. 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.Texto completo  Resumen

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

87. 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. Resumen

88. 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. Resumen

89. 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.Texto completo  Resumen

90. 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.Texto completo  Resumen

91. 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. Resumen

92. 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. Resumen

93. National Comprehensive Cancer Network. NCCN clinical practice guidelines in oncology: hematopoietic cell transplantation [internet publication].Texto completo

94. 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.Texto completo  Resumen

95. 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.Texto completo  Resumen

96. 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.Texto completo  Resumen

97. 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​.Texto completo

98. 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.Texto completo  Resumen

99. 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.Texto completo  Resumen

100. 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.Texto completo  Resumen

101. 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 9;:JCO2202699.Texto completo  Resumen

102. 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. Resumen

103. 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.Texto completo  Resumen

104. 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.Texto completo  Resumen

105. 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. Resumen

106. 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.Texto completo

107. 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.Texto completo

108. 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.Texto completo  Resumen

109. 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.Texto completo

110. 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.Texto completo  Resumen

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

112. 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.Texto completo

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