Mielofibrose

Tefferi A. Primary myelofibrosis: 2023 update on diagnosis, risk-stratification, and management. Am J Hematol. 2023 May;98(5):801-21.Texto completo  Resumo

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

McLornan DP, Godfrey AL, Green A, et al. Diagnosis and evaluation of prognosis of myelofibrosis: a British Society for Haematology Guideline. Br J Haematol. 2024 Jan;204(1):127-35.Texto completo  Resumo

Kröger N, Bacigalupo A, Barbui T, et al. Indication and management of allogeneic haematopoietic stem-cell transplantation in myelofibrosis: updated recommendations by the EBMT/ELN International Working Group. Lancet Haematol. 2024 Jan;11(1):e62-74. Resumo

McLornan DP, Psaila B, Ewing J, et al. The management of myelofibrosis: a British Society for Haematology Guideline. Br J Haematol. 2024 Jan;204(1):136-50.Texto completo  Resumo

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. Resumo

2. 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.Texto completo  Resumo

3. 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):1200-8.Texto completo  Resumo

4. Verstovsek S, Yu J, Scherber RM, et al. Changes in the incidence and overall survival of patients with myeloproliferative neoplasms between 2002 and 2016 in the United States. Leuk Lymphoma. 2022 Mar;63(3):694-702.Texto completo  Resumo

5. HMRN. ​Factsheets [internet publication].Texto completo

6. Moulard O, Mehta J, Fryzek J, et al. Epidemiology of myelofibrosis, essential thrombocythemia, and polycythemia vera in the European Union. Eur J Haematol. 2014 Apr;92(4):289-97. Resumo

7. Visfeldt J, Andersson M. Pathoanatomical aspects of malignant haematological disorders among Danish patients exposed to thorium dioxide. APMIS. 1995 Jan;103(1):29-36. Resumo

8. Anderson RE, Hoshino T, Yamamoto T. Myelofibrosis with myeloid metaplasia in survivors of the atomic bomb in Hiroshima. Ann Intern Med. 1964 Jan;60:1-18. Resumo

9. Hu H. Benzene-associated myelofibrosis. Ann Intern Med. 1987 Jan;106(1):171-2. Resumo

10. 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.Texto completo  Resumo

11. 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.Texto completo  Resumo

12. 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.Texto completo  Resumo

13. 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.Texto completo  Resumo

14. 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. Resumo

15. 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. Resumo

16. 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.Texto completo  Resumo

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

18. 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.Texto completo  Resumo

19. Rumi E, Pietra D, Pascutto C, et al. Clinical effect of driver mutations of JAK2, CALR, or MPL in primary myelofibrosis. Blood. 2014 Aug 14;124(7):1062-9.Texto completo  Resumo

20. Tefferi A, Nicolosi M, Mudireddy M, et al. Driver mutations and prognosis in primary myelofibrosis: Mayo-Careggi MPN alliance study of 1,095 patients. Am J Hematol. 2018 Mar;93(3):348-55.Texto completo  Resumo

21. Tefferi A, Lasho TL, Finke C, et al. Type 1 vs type 2 calreticulin mutations in primary myelofibrosis: differences in phenotype and prognostic impact. Leukemia. 2014 Jul;28(7):1568-70.Texto completo  Resumo

22. 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.Texto completo  Resumo

23. 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.Texto completo  Resumo

24. Tefferi A. Primary myelofibrosis: 2023 update on diagnosis, risk-stratification, and management. Am J Hematol. 2023 May;98(5):801-21.Texto completo  Resumo

25. 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.Texto completo  Resumo

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

27. Rumi E, Trotti C, Vanni D, et al. The genetic basis of primary myelofibrosis and its clinical relevance. Int J Mol Sci. 2020 Nov 24;21(23):8885.Texto completo  Resumo

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

29. 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.Texto completo  Resumo

30. 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.Texto completo  Resumo

31. 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. Resumo

32. 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.Texto completo  Resumo

33. 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.Texto completo  Resumo

34. Passamonti F, Giorgino T, Mora B, et al. A clinical-molecular prognostic model to predict survival in patients with post polycythemia vera and post essential thrombocythemia myelofibrosis. Leukemia. 2017 Dec;31(12):2726-31. Resumo

35. 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. Resumo

36. 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. Resumo

37. 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.Texto completo  Resumo

38. 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  Resumo

39. McLornan DP, Godfrey AL, Green A, et al. Diagnosis and evaluation of prognosis of myelofibrosis: a British Society for Haematology Guideline. Br J Haematol. 2024 Jan;204(1):127-35.Texto completo  Resumo

40. 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.Texto completo  Resumo

41. 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.Texto completo  Resumo

42. 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.Texto completo  Resumo

43. 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.Texto completo  Resumo

44. 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.Texto completo  Resumo

45. 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.Texto completo  Resumo

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

47. Gagelmann N, Ditschkowski M, Bogdanov R, et al. Comprehensive clinical-molecular transplant scoring system for myelofibrosis undergoing stem cell transplantation. Blood. 2019 May 16;133(20):2233-42.Texto completo  Resumo

48. Kröger N, Bacigalupo A, Barbui T, et al. Indication and management of allogeneic haematopoietic stem-cell transplantation in myelofibrosis: updated recommendations by the EBMT/ELN International Working Group. Lancet Haematol. 2024 Jan;11(1):e62-74. Resumo

49. Hernández-Boluda JC, Pereira A, Correa JG, et al. Performance of the myelofibrosis secondary to PV and ET-prognostic model (MYSEC-PM) in a series of 262 patients from the Spanish registry of myelofibrosis. Leukemia. 2018 Feb;32(2):553-5.Texto completo  Resumo

50. McLornan DP, Psaila B, Ewing J, et al. The management of myelofibrosis: a British Society for Haematology Guideline. Br J Haematol. 2024 Jan;204(1):136-50.Texto completo  Resumo

51. 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.Texto completo  Resumo

52. 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. Resumo

53. 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.Texto completo  Resumo

54. 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.Texto completo  Resumo

55. Mesa RA, Kiladjian JJ, Catalano JV, et al. SIMPLIFY-1: a phase III randomized trial of momelotinib versus ruxolitinib in Janus kinase inhibitor-naïve patients with myelofibrosis. J Clin Oncol. 2017 Dec 1;35(34):3844-50.Texto completo  Resumo

56. Harrison CN, Vannucchi AM, Platzbecker U, et al. Momelotinib versus best available therapy in patients with myelofibrosis previously treated with ruxolitinib (SIMPLIFY 2): a randomised, open-label, phase 3 trial. Lancet Haematol. 2018 Feb;5(2):e73-81.Texto completo  Resumo

57. Mesa R, Harrison C, Oh ST, et al. Overall survival in the SIMPLIFY-1 and SIMPLIFY-2 phase 3 trials of momelotinib in patients with myelofibrosis. Leukemia. 2022 Sep;36(9):2261-8.Texto completo  Resumo

58. Harrison CN, Schaap N, Vannucchi AM, et al. Janus kinase-2 inhibitor fedratinib in patients with myelofibrosis previously treated with ruxolitinib (JAKARTA-2): a single-arm, open-label, non-randomised, phase 2, multicentre study. Lancet Haematol. 2017 Jul;4(7):e317-24. Resumo

59. 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.Texto completo  Resumo

60. 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.Texto completo  Resumo

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

62. 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.Texto completo  Resumo

63. Daghia G, Zabelina T, Zeck G, et al. Allogeneic stem cell transplantation for myelofibrosis patients aged ≥65 years. Eur J Haematol. 2019 Oct;103(4):370-8.Texto completo  Resumo

64. Hernández-Boluda JC, Pereira A, Kröger N, et al. Allogeneic hematopoietic cell transplantation in older myelofibrosis patients: a study of the chronic malignancies working party of EBMT and the Spanish Myelofibrosis Registry. Am J Hematol. 2021 Oct 1;96(10):1186-94.Texto completo  Resumo

65. Gagelmann N, Salit RB, Schroeder T, et al. High molecular and cytogenetic risk in myelofibrosis does not benefit from higher intensity conditioning before hematopoietic cell transplantation: an international collaborative analysis. Hemasphere. 2022 Oct;6(10):e784.Texto completo  Resumo

66. 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.Texto completo  Resumo

67. 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.Texto completo  Resumo

68. 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.Texto completo  Resumo

69. 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. Resumo

70. 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.Texto completo  Resumo

71. Vannucchi AM, Kantarjian HM, Kiladjian JJ, et al. A pooled analysis of overall survival in COMFORT-I and COMFORT-II, 2 randomized phase III trials of ruxolitinib for the treatment of myelofibrosis. Haematologica. 2015 Sep;100(9):1139-45.Texto completo  Resumo

72. 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.Texto completo  Resumo

73. 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.Texto completo

74. Polverelli N, Mauff K, Kröger N, et al. Impact of spleen size and splenectomy on outcomes of allogeneic hematopoietic cell transplantation for myelofibrosis: a retrospective analysis by the chronic malignancies working party on behalf of European society for blood and marrow transplantation (EBMT). Am J Hematol. 2021 Jan;96(1):69-79.Texto completo  Resumo

75. Tefferi A, Mesa RA, Nagorney DM, et al. Splenectomy in myelofibrosis with myeloid metaplasia: a single-institution experience with 223 patients. Blood. 2000 Apr 1;95(7):2226-33.Texto completo  Resumo

76. 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.Texto completo  Resumo

77. 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. Resumo

78. 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. Resumo

79. Crisà E, Cilloni D, Elli EM, et al. The use of erythropoiesis-stimulating agents is safe and effective in the management of anaemia in myelofibrosis patients treated with ruxolitinib. Br J Haematol. 2018 Sep;182(5):701-4.Texto completo  Resumo

80. Huang J, Tefferi A. Erythropoiesis stimulating agents have limited therapeutic activity in transfusion-dependent patients with primary myelofibrosis regardless of serum erythropoietin level. Eur J Haematol. 2009 Aug;83(2):154-5.Texto completo  Resumo

81. Cervantes F, Isola IM, Alvarez-Larrán A, et al. Danazol therapy for the anemia of myelofibrosis: assessment of efficacy with current criteria of response and long-term results. Ann Hematol. 2015 Nov;94(11):1791-6.Texto completo  Resumo

82. 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. Resumo

83. Gerds AT, Harrison C, Kiladjian J-J, et al. Safety and efficacy of luspatercept for the treatment of anemia in patients with myelofibrosis: Results from the ACE-536-MF-001 study. J Clin Oncol. 2023;41:7016.Texto completo

84. Kröger N, Sbianchi G, Sirait T, et al. Impact of prior JAK-inhibitor therapy with ruxolitinib on outcome after allogeneic hematopoietic stem cell transplantation for myelofibrosis: a study of the CMWP of EBMT. Leukemia. 2021 Dec;35(12):3551-60.Texto completo  Resumo

85. Shanavas M, Popat U, Michaelis LC, et al. Outcomes of allogeneic hematopoietic cell transplantation in patients with myelofibrosis with prior exposure to Janus kinase 1/2 inhibitors. Biol Blood Marrow Transplant. 2016 Mar;22(3):432-40.Texto completo  Resumo

86. Smith CJ, Thomas JW, Ruan G, et al. A population-based study of outcomes in polycythemia vera, essential thrombocythemia, and primary myelofibrosis in the United States from 2001 to 2015: comparison with data from a Mayo Clinic single institutional series. Am J Hematol. 2021 Dec 1;96(12):E464-8.Texto completo  Resumo

87. Szuber N, Vallapureddy RR, Penna D, et al. Myeloproliferative neoplasms in the young: Mayo Clinic experience with 361 patients age 40 years or younger. Am J Hematol. 2018 Dec;93(12):1474-84.Texto completo  Resumo

88. 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.Texto completo  Resumo