骨髓纤维化

Reilly JT, McMullin MF, Beer PA, et al; British Committee for Standards in Haematology. Guideline for the diagnosis and management of myelofibrosis. Br J Haematol. 2012 Aug;158(4):453-71.全文  摘要

National Comprehensive Cancer Network. NCCN clinical practice guidelines in oncology: myeloproliferative neoplasms [internet publication].全文

Reilly JT, McMullin MF, Beer PA, et al. Use of JAK inhibitors in the management of myelofibrosis: a revision of the British Committee for Standards in Haematology guidelines for investigation and management of myelofibrosis 2012. Br J Haematol. 2014 Nov;167(3):418-20. 摘要

Vannucchi AM, Barbui T, Cervantes F, et al. Philadelphia chromosome-negative chronic myeloproliferative neoplasms: ESMO clinical practice guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2015 Sep;26(5 suppl):v85-99.全文  摘要

Kröger NM, Deeg JH, Olavarria E, et al. Indication and management of allogeneic stem cell transplantation in primary myelofibrosis: a consensus process by an EBMT/ELN international working group. Leukemia. 2015 Nov;29(11):2126-33. 摘要

1. Mesa RA, Verstovsek S, Cervantes F, et al. Primary myelofibrosis (PMF), post polycythemia vera myelofibrosis (post-PV MF), post essential thrombocythemia myelofibrosis (post-ET MF), blast phase PMF (PMF-BP): consensus on terminology by the international working group for myelofibrosis research and treatment (IWG-MRT). Leuk Res. 2007 Jun;31(6):737-40. 摘要

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9. Vannucchi AM, Guglielmelli P. Molecular prognostication in Ph-negative MPNs in 2022. Hematology Am Soc Hematol Educ Program. 2022 Dec 9;2022(1):225-34.全文  摘要

10. Passamonti F, Cervantes F, Vannucchi AM, et al. A dynamic prognostic model to predict survival in primary myelofibrosis: a study by the IWG-MRT (International Working Group for Myeloproliferative Neoplasms Research and Treatment). Blood. 2010 Mar 4;115(9):1703-8.全文  摘要

11. Tefferi A, Nicolosi M, Mudireddy M, et al. Revised cytogenetic risk stratification in primary myelofibrosis: analysis based on 1002 informative patients. Leukemia. 2018 May;32(5):1189-99.全文  摘要

12. Tefferi A, Guglielmelli P, Lasho TL, et al. MIPSS70+ version 2.0: mutation and karyotype-enhanced international prognostic scoring system for primary myelofibrosis. J Clin Oncol. 2018 Jun 10;36(17):1769-70.全文  摘要

13. Pardanani A, Guglielmelli P, Lasho TL, et al. Primary myelofibrosis with or without mutant MPL: comparison of survival and clinical features involving 603 patients. Leukemia. 2011 Dec;25(12):1834-9. 摘要

14. Tefferi A, Lasho TL, Finke CM, et al. CALR vs JAK2 vs MPL-mutated or triple-negative myelofibrosis: clinical, cytogenetic and molecular comparisons. Leukemia. 2014 Jul;28(7):1472-7. 摘要

15. Rumi E, Passamonti F, Pietra D, et al. JAK2 (V617F) as an acquired somatic mutation and a secondary genetic event associated with disease progression in familial myeloproliferative disorders. Cancer. 2006 Nov 1;107(9):2206-11.全文  摘要

16. Rumi E, Cazzola M. Advances in understanding the pathogenesis of familial myeloproliferative neoplasms. Br J Haematol. 2017 Sep;178(5):689-98.全文  摘要

17. Venkatesan A, Satin LS, Raghavan M. Roles of calreticulin in protein folding, immunity, calcium signaling and cell transformation. Prog Mol Subcell Biol. 2021;59:145-62.全文  摘要

18. Elf S, Abdelfattah NS, Baral AJ, et al. Defining the requirements for the pathogenic interaction between mutant calreticulin and MPL in MPN. Blood. 2018 Feb 15;131(7):782-6.全文  摘要

19. Bilbao-Sieyro C, Florido Y, Gómez-Casares MT. CALR mutation characterization in myeloproliferative neoplasms. Oncotarget. 2016 Aug 16;7(33):52614-7.全文  摘要

20. Alshemmari SH, Rajan R, Emadi A. Molecular pathogenesis and clinical significance of driver mutations in primary myelofibrosis: a review. Med Princ Pract. 2016;25(6):501-9.全文  摘要

21. Milosevic Feenstra JD, Nivarthi H, Gisslinger H, et al. Whole-exome sequencing identifies novel MPL and JAK2 mutations in triple-negative myeloproliferative neoplasms. Blood. 2016 Jan 21;127(3):325-32.全文  摘要

22. Tefferi A, Barbui T. Polycythemia vera and essential thrombocythemia: 2017 update on diagnosis, risk-stratification, and management. Am J Hematol. 2017 Jan;92(1):94-108.全文  摘要

23. Brecqueville M, Rey J, Devillier R, et al. Array comparative genomic hybridization and sequencing of 23 genes in 80 patients with myelofibrosis at chronic or acute phase. Haematologica. 2014 Jan;99(1):37-45.全文  摘要

24. Tefferi A. Pathogenesis of myelofibrosis with myeloid metaplasia. J Clin Oncol. 2005 Nov 20;23(33):8520-30. 摘要

25. Agarwal A, Morrone K, Bartenstein M, et al. Bone marrow fibrosis in primary myelofibrosis: pathogenic mechanisms and the role of TGF-β. Stem Cell Investig. 2016 Feb 26;3:5.全文  摘要

26. Zahr AA, Salama ME, Carreau N, et al. Bone marrow fibrosis in myelofibrosis: pathogenesis, prognosis and targeted strategies. Haematologica. 2016 Jun;101(6):660-71.全文  摘要

27. Kuter DJ, Bain B, Mufti G, et al. Bone marrow fibrosis: pathophysiology and clinical significance of increased bone marrow stromal fibres. Br J Haematol. 2007 Nov;139(3):351-62. 摘要

28. Moliterno AR, Hankins WD, Spivak JL. Impaired expression of the thrombopoietin receptor by platelets from patients with polycythemia vera. N Engl J Med. 1998 Feb 26;338(9):572-80.全文  摘要

29. Wilkins BS, Erber WN, Bareford D, et al. Bone marrow pathology in essential thrombocythemia: interobserver reliability and utility for identifying disease subtypes. Blood. 2008 Jan 1;111(1):60-70.全文  摘要

30. Tefferi A. Primary myelofibrosis: 2021 update on diagnosis, risk-stratification and management. Am J Hematol. 2021 Jan;96(1):145-62.全文  摘要

31. Cervantes F, Pereira A, Esteve J, et al. Identification of 'short-lived' and 'long-lived' patients at presentation of idiopathic myelofibrosis. Br J Haematol. 1997 Jun;97(3):635-40. 摘要

32. Glew RH, Haese WH, Mclntyre PA. Myeloid metaplasia with myelofibrosis: the clinical spectrum of extramedullary hematopoiesis and tumor formation. Johns Hopkins Med J. 1973 May;132(5):253-70. 摘要

33. Mughal TI, Vaddi K, Sarlis NJ, et al. Myelofibrosis-associated complications: pathogenesis, clinical manifestations, and effects on outcomes. Int J Gen Med. 2014 Jan 29;7:89-101.全文  摘要

34. Reilly JT, McMullin MF, Beer PA, et al; British Committee for Standards in Haematology. Guideline for the diagnosis and management of myelofibrosis. Br J Haematol. 2012 Aug;158(4):453-71.全文  摘要

35. National Comprehensive Cancer Network. NCCN clinical practice guidelines in oncology: myeloproliferative neoplasms [internet publication].全文

36. Andriani A, Latagliata R, Anaclerico B, et al. Spleen enlargement is a risk factor for thrombosis in essential thrombocythemia: Evaluation on 1,297 patients. Am J Hematol. 2016 Mar;91(3):318-21.全文  摘要

37. Accurso V, Santoro M, Raso S, et al. Splenomegaly impacts prognosis in essential thrombocythemia and polycythemia vera: a single center study. Hematol Rep. 2019 Nov 29;11(4):8281.全文  摘要

38. Reilly JT, McMullin MF, Beer PA, et al. Use of JAK inhibitors in the management of myelofibrosis: a revision of the British Committee for Standards in Haematology guidelines for investigation and management of myelofibrosis 2012. Br J Haematol. 2014 Nov;167(3):418-20. 摘要

39. Emanuel RM, Dueck AC, Geyer HL, et al. Myeloproliferative neoplasm (MPN) symptom assessment form total symptom score: prospective international assessment of an abbreviated symptom burden scoring system among patients with MPNs. J Clin Oncol. 2012 Nov 20;30(33):4098-103.全文  摘要

40. Cervantes F, Dupriez B, Pereira A, et al. New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. Blood. 2009 Mar 26;113(13):2895-901.全文  摘要

41. Gangat N, Caramazza D, Vaidya R, et al. DIPSS plus: a refined Dynamic International Prognostic Scoring System for primary myelofibrosis that incorporates prognostic information from karyotype, platelet count, and transfusion status. J Clin Oncol. 2011 Feb 1;29(4):392-7.全文  摘要

42. Guglielmelli P, Lasho TL, Rotunno G, et al. MIPSS70: mutation-enhanced international prognostic score system for transplantation-age patients with primary myelofibrosis. J Clin Oncol. 2018 Feb 1;36(4):310-8.全文  摘要

43. Tefferi A, Guglielmelli P, Nicolosi M, et al. GIPSS: genetically inspired prognostic scoring system for primary myelofibrosis. Leukemia. 2018 Jul;32(7):1631-42.全文  摘要

44. Vannucchi AM, Barbui T, Cervantes F, et al. Philadelphia chromosome-negative chronic myeloproliferative neoplasms: ESMO clinical practice guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2015 Sep;26(5 suppl):v85-99.全文  摘要

45. Silver RT, Kiladjian JJ, Hasselbalch HC. Interferon and the treatment of polycythemia vera, essential thrombocythemia and myelofibrosis. Expert Rev Hematol. 2013 Feb;6(1):49-58. 摘要

46. Kröger NM, Deeg JH, Olavarria E, et al. Indication and management of allogeneic stem cell transplantation in primary myelofibrosis: a consensus process by an EBMT/ELN international working group. Leukemia. 2015 Nov;29(11):2126-33. 摘要

47. Rondelli D, Goldberg JD, Isola L, et al. MPD-RC 101 prospective study of reduced-intensity allogeneic hematopoietic stem cell transplantation in patients with myelofibrosis. Blood. 2014 Aug 14;124(7):1183-91.全文  摘要

48. Ballen KK, Shrestha S, Sobocinski KA, et al. Outcome of transplantation for myelofibrosis. Biol Blood Marrow Transplant. 2010 Mar;16(3):358-67.全文  摘要

49. Kröger N, Holler E, Kobbe G, et al. Allogeneic stem cell transplantation after reduced-intensity conditioning in patients with myelofibrosis: a prospective, multicenter study of the Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation. Blood. 2009 Dec 17;114(26):5264-70.全文  摘要

50. Verstovsek S, Mesa RA, Gotlib J, et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med. 2012 Mar 1;366(9):799-807.全文  摘要

51. Harrison C, Kiladjian JJ, Al-Ali HK, et al. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med. 2012 Mar 1;366(9):787-98.全文  摘要

52. Cervantes F, Vannucchi AM, Kiladjian JJ, et al.; COMFORT-II investigators. Three-year efficacy, safety, and survival findings from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for myelofibrosis. Blood. 2013 Dec 12;122(25):4047-53.全文  摘要

53. Harrison CN, Mesa RA, Kiladjian JJ, et al. Health-related quality of life and symptoms in patients with myelofibrosis treated with ruxolitinib versus best available therapy. Br J Haematol. 2013 Jul;162(2):229-39. 摘要

54. Verstovsek S, Mesa RA, Gotlib J, et al. Efficacy, safety and survival with ruxolitinib in patients with myelofibrosis: results of a median 2-year follow-up of COMFORT-I. Haematologica. 2013 Dec;98(12):1865-71.全文  摘要

55. Pardanani A, Tefferi A, Masszi T, et al. Updated results of the placebo-controlled, phase III JAKARTA trial of fedratinib in patients with intermediate-2 or high-risk myelofibrosis. Br J Haematol. 2021 Oct;195(2):244-8.全文  摘要

56. Harrison CN, Schaap N, Vannucchi AM, et al. Fedratinib in patients with myelofibrosis previously treated with ruxolitinib: an updated analysis of the JAKARTA2 study using stringent criteria for ruxolitinib failure. Am J Hematol. 2020 Jun;95(6):594-603.全文  摘要

57. Harrison CN, Mesa RA, Jamieson C, et al. Case series of potential Wernicke's encephalopathy in patients treated with fedratinib. Blood. 2017 Dec 8;130(1 suppl):4197.全文

58. Mesa RA, Vannucchi AM, Mead A, et al. Pacritinib versus best available therapy for the treatment of myelofibrosis irrespective of baseline cytopenias (PERSIST-1): an international, randomised, phase 3 trial. Lancet Haematol. 2017 May;4(5):e225-36.全文  摘要

59. Mascarenhas J, Hoffman R, Talpaz M, et al. Pacritinib vs best available therapy, including ruxolitinib, in patients with myelofibrosis: a randomized clinical trial. JAMA Oncol. 2018 May 1;4(5):652-9.全文  摘要

60. Mesa RA. How I treat symptomatic splenomegaly in patients with myelofibrosis. Blood. 2009 May 28;113(22):5394-400.全文  摘要

61. Thomas DA, Giles FJ, Albitar M, et al. Thalidomide therapy for myelofibrosis with myeloid metaplasia. Cancer. 2006 May 1;106(9):1974-84.全文  摘要

62. Mesa RA, Steensma DP, Pardanani A, et al. A phase 2 trial of combination low-dose thalidomide and prednisone for the treatment of myelofibrosis with myeloid metaplasia. Blood. 2003 Apr 1;101(7):2534-41.全文  摘要

63. Elliott MA, Chen MG, Silverstein MN, et al. Splenic irradiation for symptomatic splenomegaly associated with myelofibrosis with myeloid metaplasia. Br J Haematol. 1998 Nov;103(2):505-11.全文  摘要

64. Bouabdallah R, Coso D, Gonzague-Casabianca L, et al. Safety and efficacy of splenic irradiation in the treatment of patients with idiopathic myelofibrosis: a report on 15 patients. Leuk Res. 2000 Jun;24(6):491-5. 摘要

65. Elliott MA, Tefferi A. Splenic irradiation in myelofibrosis with myeloid metaplasia: a review. Blood Rev. 1999 Sep;13(3):163-70. 摘要

66. Silver RT, Vandris K, Goldman JJ. Recombinant interferon-alpha may retard progression of early primary myelofibrosis: a preliminary report. Blood. 2011 Jun 16;117(24):6669-72.全文  摘要

67. Hernández-Boluda JC, Correa JG, García-Delgado R, et al. Predictive factors for anemia response to erythropoiesis-stimulating agents in myelofibrosis. Eur J Haematol. 2017 Apr;98(4):407-14. 摘要

68. Steensma DP, Mesa RA, Li CY, et al. Etanercept, a soluble tumor necrosis factor receptor, palliates constitutional symptoms in patients with myelofibrosis with myeloid metaplasia: results of a pilot study. Blood. 2002 Mar 15;99(6):2252-4.全文  摘要

69. Tefferi A, Guglielmelli P, Larson DR, et al. Long-term survival and blast transformation in molecularly annotated essential thrombocythemia, polycythemia vera, and myelofibrosis. Blood. 2014 Oct 16;124(16):2507-13.全文  摘要

70. Guglielmelli P, Lasho TL, Rotunno G, et al. The number of prognostically detrimental mutations and prognosis in primary myelofibrosis: an international study of 797 patients. Leukemia. 2014 Sep;28(9):1804-10. 摘要

71. Mesa RA, Li CY, Ketterling RP, et al. Leukemic transformation in myelofibrosis with myeloid metaplasia: a single-institution experience with 91 cases. Blood. 2005 Feb 1;105(3):973-7.全文  摘要