Myelodysplastic syndrome

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

1. Khoury JD, Solary E, Abla O, et al. The 5th edition of the World Health Organization classification of haematolymphoid tumours: myeloid and histiocytic/dendritic neoplasms. Leukemia. 2022 Jul;36(7):1703-19.Full text  Abstract

2. Arber DA, Orazi A, Hasserjian RP, et al. International Consensus Classification of myeloid neoplasms and acute leukemias: integrating morphologic, clinical, and genomic data. Blood. 2022 Sep 15;140(11):1200-28.Full text  Abstract

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

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. Abstract

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

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

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

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. Abstract

9. Sekeres MA, Taylor J. Diagnosis and treatment of myelodysplastic syndromes: a review. JAMA. 2022 Sep 6;328(9):872-80. Abstract

10. 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.Full text  Abstract

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.Full text  Abstract

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

13. National Institue of Health; National Cancer Institute. All cancer sites combined: recent trends in SEER age-adjusted incidence rates, 2000-2022. Jul 2025 [internet publication].Full text

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

15. 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

16. 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.Full text  Abstract

17. 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. Abstract

18. 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. Abstract

19. 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.Full text  Abstract

20. 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.Full text  Abstract

21. Saygin C, Godley LA. Genetics of Myelodysplastic Syndromes. Cancers (Basel). 2021 Jul;13(14):3380.Full text  Abstract

22. 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.Full text  Abstract

23. 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.Full text  Abstract

24. 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.Full text  Abstract

25. Oetjen KA, Levoska MA, Tamura D, et al. Predisposition to hematologic malignancies in patients with xeroderma pigmentosum. Haematologica. 2020 Apr;105(4):e144-6.Full text  Abstract

26. 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. Abstract

27. 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.Full text  Abstract

28. 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.Full text  Abstract

29. 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.Full text  Abstract

30. 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

31. 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.Full text  Abstract

32. 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.Full text  Abstract

33. 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.Full text  Abstract

34. 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.Full text  Abstract

35. 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.Full text  Abstract

36. 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. Abstract

37. Calip GS, Malmgren JA, Lee WJ, et al. Myelodysplastic syndrome and acute myeloid leukemia following adjuvant chemotherapy with and without granulocyte colony-stimulating factors for breast cancer. Breast Cancer Res Treat. 2015 Nov;154(1):133-43.Full text  Abstract

38. 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.Full text  Abstract

39. Leone G, Pagano L, Ben-Yehuda D, et al. Therapy-related leukemia and myelodysplasia: susceptibility and incidence. Haematologica. 2007 Oct;92(10):1389-98.Full text  Abstract

40. 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. Abstract

41. 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.Full text  Abstract

42. 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

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

44. 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.Full text  Abstract

45. 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.Full text  Abstract

46. 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. Abstract

47. 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

48. Sallman DA, Padron E. Myelodysplasia in younger adults: outlier or unique molecular entity? Haematologica. 2017 Jun;102(6):967-8.Full text  Abstract

49. Hirsch CM, Przychodzen BP, Radivoyevitch T, et al. Molecular features of early onset adult myelodysplastic syndrome. Haematologica. 2017 Jun;102(6):1028-34.Full text  Abstract

50. 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.Full text  Abstract

51. 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.Full text  Abstract

52. 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

53. 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.Full text  Abstract

54. 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.Full text  Abstract

55. 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

56. 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.Full text  Abstract

57. Toma A, Fenaux P, Dreyfus F, et al. Infections in myelodysplastic syndromes. Haematologica. 2012 Oct;97(10):1459-70.Full text  Abstract

58. 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.Full text  Abstract

59. Carraway HE, Saygin C. Therapy for lower-risk MDS. Hematology Am Soc Hematol Educ Program. 2020 Dec;2020(1):426-33.Full text  Abstract

60. 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. Abstract

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

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

63. 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

64. 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.Full text  Abstract

65. Tentori CA, Gregorio C, Robin M, et al. Clinical and genomic-based decision support system to define the optimal timing of allogeneic hematopoietic stem-cell transplantation in patients with myelodysplastic syndromes. J Clin Oncol. 2024 Aug 20;42(24):2873-86.Full text  Abstract

66. Baer C, Huber S, Hutter S, et al. Risk prediction in MDS: independent validation of the IPSS-M-ready for routine? Leukemia. 2023 Apr;37(4):938-41.Full text

67. Wu H, Li S, Yang J, et al. IPSS-M outperforms IPSS-R in prognostic stratification and guides effective interventions for very high-risk myelodysplastic syndrome patients undergoing allogeneic hematopoietic stem cell transplantation. Discov Oncol. 2025 Jul 11;16(1):1315.Full text  Abstract

68. Lee WH, Tsai MT, Tsai CH, et al. Validation of the molecular international prognostic scoring system in patients with myelodysplastic syndromes defined by international consensus classification. Blood Cancer J. 2023 Aug 9;13(1):120.Full text  Abstract

69. Mo A, McQuilten ZK, Wood EM, et al. Red cell transfusion thresholds in myelodysplastic syndromes: a clinician survey to inform future clinical trials. Intern Med J. 2017 Jun;47(6):695-8. Abstract

70. DeFilipp Z, Ciurea SO, Cutler C, et al. Hematopoietic cell transplantation in the management of myelodysplastic syndrome: an evidence-based review from the American Society for Transplantation and Cellular Therapy Committee on Practice Guidelines. Transplant Cell Ther. 2023 Feb;29(2):71-81.Full text  Abstract

71. Bohlius J, Bohlke K, Castelli R, et al. Management of cancer-associated anemia with erythropoiesis-stimulating agents: ASCO/ASH clinical practice guideline update. J Clin Oncol. 2019 May 20;37(15):1336-51. Abstract

72. 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

73. Hellström-Lindberg E, Negrin R, Stein R, et al. Erythroid response to treatment with G-CSF plus erythropoietin for the anaemia of patients with myelodysplastic syndromes: proposal for a predictive model. Br J Haematol. 1997 Nov;99(2):344-51. Abstract

74. Hellström-Lindberg E, Gulbrandsen N, Lindberg G, et al. A validated decision model for treating the anaemia of myelodysplastic syndromes with erythropoietin + granulocyte colony-stimulating factor: significant effects on quality of life. Br J Haematol. 2003 Mar;120(6):1037-46. Abstract

75. Platzbecker U, Della Porta MG, Santini V, et al. Efficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): interim analysis of a phase 3, open-label, randomised controlled trial. Lancet. 2023 Jul 29;402(10399):373-85. Abstract

76. Toma A, Kosmider O, Chevret S, et al. Lenalidomide with or without erythropoietin in transfusion-dependent erythropoiesis-stimulating agent-refractory lower-risk MDS without 5q deletion. Leukemia. 2016 Apr;30(4):897-905.Full text  Abstract

77. 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

78. 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

79. 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

80. 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

81. 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.Full text  Abstract

82. 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

83. Fenaux P, Muus P, Kantarjian H, et al. Romiplostim monotherapy in thrombocytopenic patients with myelodysplastic syndromes: long-term safety and efficacy. Br J Haematol. 2017 Sep;178(6):906-13.Full text  Abstract

84. 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

85. 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

86. 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

87. 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

88. 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

89. Garcia‑Manero G, Platzbecker U, Fenaux P, et al. MDS‑1497: primary analysis of the randomized, Phase 3 VERONA study of venetoclax with azacitidine vs placebo with azacitidine in patients with treatment‑naïve, intermediate and higher‑risk myelodysplastic syndromes. Clin Lymphoma Myeloma Leuk. 2025 Sep;25(Suppl 1):S644–5.

90. Cortes JE, Yang J, Roboz GJ, et al. Olutasidenib alone or combined with azacitidine in patients with mutant IDH1 myelodysplastic syndrome. Blood Adv. 2025 Jul 16:bloodadvances.2025016718.Full text  Abstract

91. Sebert M, Chevret S, Dimicoli‑Salazar S, et al. Enasidenib (ENA) monotherapy in patients with IDH2‑mutated myelodysplastic syndrome (MDS), the Ideal Phase 2 Study by the GFM and Emsco Groups. Blood. 2024 Nov 5;144(Suppl 1):1839.​Full text

92. 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

93. Moreno Berggren D, Garelius H, Willner Hjelm P, et al. Therapy-related MDS dissected based on primary disease and treatment-a nationwide perspective. Leukemia. 2023 May;37(5):1103-12.Full text  Abstract

94. 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

95. Bejar R, Stevenson K, Abdel-Wahab O, et al. Clinical effect of point mutations in myelodysplastic syndromes. N Engl J Med. 2011 Jun 30;364(26):2496-506.Full text  Abstract

96. 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

97. 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

98. Angelucci E, Li J, Greenberg P, et al. Iron chelation in transfusion-dependent patients with low- to intermediate-1-risk myelodysplastic syndromes: a randomized trial. Ann Intern Med. 2020 Apr;172(8):513-22.Full text  Abstract

99. 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

100. Radsak M, Platzbecker U, Schmidt CS, et al. Infectious complications in patients with myelodysplastic syndromes: a review of the literature with emphasis on patients treated with 5-azacitidine. Eur J Haematol. 2017 Aug;99(2):112-8.Full text  Abstract

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