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    Chronic Myeloproliferative Neoplasms Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Primary Myelofibrosis


    Hydroxyurea is useful in patients with splenomegaly but may have a potential leukemogenic effect.[4] In patients with thrombocytosis and hepatomegaly after splenectomy, cladribine has shown responses as an alternative to hydroxyurea.[43] The use of interferon-alpha can result in hematologic responses, including reduction in spleen size in 30% to 50% of patients, though many patients do not tolerate this medication.[44,45] Favorable responses to thalidomide and lenalidomide have been reported in about 20% to 60% of patients.[19,20,21,46,47,48][Level of evidence: 3iiiDiv]

    A response defined as 50% reduction of splenomegaly or development of transfusion independence was attained by one-third of 34 symptomatic patients using tipifarnib.[49][Level of evidence: 3iiiDiv] A more aggressive approach involves allogeneic peripheral stem cell or bone marrow transplantation when a suitable donor is available.[50,51,52,53,54,55] Allogeneic stem cell transplantation is the only potentially curative treatment available, but the associated morbidity and mortality limit its use to younger, high-risk patients.[53,56] Detection of the JAK2 mutation after transplantation is associated with a worse prognosis.[57]

    Treatment options:

    1. Ruxolitinib.[30,31,36,37]
    2. Clinical trials involving other JAK2 inhibitors.
    3. Hydroxyurea.[3,4]
    4. Allogeneic peripheral stem cell or bone marrow transplantation.[51,52,53,54,55]
    5. Thalidomide.[19,24,46,47,48,50]
    6. Lenalidomide.[21,25,26,27,48]
    7. Pomalidomide.[28]
    8. Splenectomy.[39,58]
    9. Splenic radiation therapy or radiation to sites of symptomatic extramedullary hematopoiesis (e.g., large lymph nodes, cord compression).[4]
    10. Cladribine.[43]
    11. Interferon-alpha.[44,45]

    Current Clinical Trials

    Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with primary myelofibrosis. The list of clinical trials can be further narrowed by location, drug, intervention, and other criteria.

    General information about clinical trials is also available from the NCI Web site.


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    3. Barosi G: Myelofibrosis with myeloid metaplasia: diagnostic definition and prognostic classification for clinical studies and treatment guidelines. J Clin Oncol 17 (9): 2954-70, 1999.
    4. Tefferi A: Myelofibrosis with myeloid metaplasia. N Engl J Med 342 (17): 1255-65, 2000.
    5. Tefferi A, Thiele J, Vardiman JW: The 2008 World Health Organization classification system for myeloproliferative neoplasms: order out of chaos. Cancer 115 (17): 3842-7, 2009.
    6. Klampfl T, Gisslinger H, Harutyunyan AS, et al.: Somatic mutations of calreticulin in myeloproliferative neoplasms. N Engl J Med 369 (25): 2379-90, 2013.
    7. Nangalia J, Massie CE, Baxter EJ, et al.: Somatic CALR mutations in myeloproliferative neoplasms with nonmutated JAK2. N Engl J Med 369 (25): 2391-405, 2013.
    8. Chim CS, Kwong YL, Lie AK, et al.: Long-term outcome of 231 patients with essential thrombocythemia: prognostic factors for thrombosis, bleeding, myelofibrosis, and leukemia. Arch Intern Med 165 (22): 2651-8, 2005 Dec 12-26.
    9. Hussein K, Pardanani AD, Van Dyke DL, et al.: International Prognostic Scoring System-independent cytogenetic risk categorization in primary myelofibrosis. Blood 115 (3): 496-9, 2010.
    10. Cervantes F, Barosi G, Demory JL, et al.: Myelofibrosis with myeloid metaplasia in young individuals: disease characteristics, prognostic factors and identification of risk groups. Br J Haematol 102 (3): 684-90, 1998.
    11. Strasser-Weippl K, Steurer M, Kees M, et al.: Age and hemoglobin level emerge as most important clinical prognostic parameters in patients with osteomyelofibrosis: introduction of a simplified prognostic score. Leuk Lymphoma 47 (3): 441-50, 2006.
    12. Tefferi A: Survivorship and prognosis in myelofibrosis with myeloid metaplasia. Leuk Lymphoma 47 (3): 379-80, 2006.
    13. Tam CS, Kantarjian H, Cortes J, et al.: Dynamic model for predicting death within 12 months in patients with primary or post-polycythemia vera/essential thrombocythemia myelofibrosis. J Clin Oncol 27 (33): 5587-93, 2009.
    14. Morel P, Duhamel A, Hivert B, et al.: Identification during the follow-up of time-dependent prognostic factors for the competing risks of death and blast phase in primary myelofibrosis: a study of 172 patients. Blood 115 (22): 4350-5, 2010.
    15. 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 113 (13): 2895-901, 2009.
    16. Tefferi A, Lasho TL, Jimma T, et al.: One thousand patients with primary myelofibrosis: the mayo clinic experience. Mayo Clin Proc 87 (1): 25-33, 2012.
    17. 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 29 (4): 392-7, 2011.
    18. Caramazza D, Begna KH, Gangat N, et al.: Refined cytogenetic-risk categorization for overall and leukemia-free survival in primary myelofibrosis: a single center study of 433 patients. Leukemia 25 (1): 82-8, 2011.
    19. Giovanni B, Michelle E, Letizia C, et al.: Thalidomide in myelofibrosis with myeloid metaplasia: a pooled-analysis of individual patient data from five studies. Leuk Lymphoma 43 (12): 2301-7, 2002.
    20. Marchetti M, Barosi G, Balestri F, et al.: Low-dose thalidomide ameliorates cytopenias and splenomegaly in myelofibrosis with myeloid metaplasia: a phase II trial. J Clin Oncol 22 (3): 424-31, 2004.
    21. Tefferi A, Cortes J, Verstovsek S, et al.: Lenalidomide therapy in myelofibrosis with myeloid metaplasia. Blood 108 (4): 1158-64, 2006.
    22. Cervantes F, Alvarez-Larrán A, Hernández-Boluda JC, et al.: Erythropoietin treatment of the anaemia of myelofibrosis with myeloid metaplasia: results in 20 patients and review of the literature. Br J Haematol 127 (4): 399-403, 2004.
    23. 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 83 (2): 154-5, 2009.
    24. Thomas DA, Giles FJ, Albitar M, et al.: Thalidomide therapy for myelofibrosis with myeloid metaplasia. Cancer 106 (9): 1974-84, 2006.
    25. Tefferi A, Lasho TL, Mesa RA, et al.: Lenalidomide therapy in del(5)(q31)-associated myelofibrosis: cytogenetic and JAK2V617F molecular remissions. Leukemia 21 (8): 1827-8, 2007.
    26. Mesa RA, Yao X, Cripe LD, et al.: Lenalidomide and prednisone for myelofibrosis: Eastern Cooperative Oncology Group (ECOG) phase 2 trial E4903. Blood 116 (22): 4436-8, 2010.
    27. Quintás-Cardama A, Kantarjian HM, Manshouri T, et al.: Lenalidomide plus prednisone results in durable clinical, histopathologic, and molecular responses in patients with myelofibrosis. J Clin Oncol 27 (28): 4760-6, 2009.
    28. Begna KH, Mesa RA, Pardanani A, et al.: A phase-2 trial of low-dose pomalidomide in myelofibrosis. Leukemia 25 (2): 301-4, 2011.
    29. Verstovsek S, Kantarjian H, Mesa RA, et al.: Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med 363 (12): 1117-27, 2010.
    30. Harrison C, Kiladjian JJ, Al-Ali HK, et al.: JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med 366 (9): 787-98, 2012.
    31. Verstovsek S, Mesa RA, Gotlib J, et al.: A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med 366 (9): 799-807, 2012.
    32. 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 98 (12): 1865-71, 2013.
    33. Cervantes F, Vannucchi AM, Kiladjian JJ, et al.: Three-year efficacy, safety, and survival findings from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for myelofibrosis. Blood 122 (25): 4047-53, 2013.
    34. Mascarenhas J, Hoffman R: A comprehensive review and analysis of the effect of ruxolitinib therapy on the survival of patients with myelofibrosis. Blood 121 (24): 4832-7, 2013.
    35. Verstovsek S, Mesa RA, Gotlib J, et al.: The clinical benefit of ruxolitinib across patient subgroups: analysis of a placebo-controlled, Phase III study in patients with myelofibrosis. Br J Haematol 161 (4): 508-16, 2013.
    36. Tefferi A, Litzow MR, Pardanani A: Long-term outcome of treatment with ruxolitinib in myelofibrosis. N Engl J Med 365 (15): 1455-7, 2011.
    37. Verstovsek S: Janus-activated kinase 2 inhibitors: a new era of targeted therapies providing significant clinical benefit for Philadelphia chromosome-negative myeloproliferative neoplasms. J Clin Oncol 29 (7): 781-3, 2011.
    38. Tefferi A: JAK inhibitors for myeloproliferative neoplasms: clarifying facts from myths. Blood 119 (12): 2721-30, 2012.
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    40. Lavrenkov K, Krepel-Volsky S, Levi I, et al.: Low dose palliative radiotherapy for splenomegaly in hematologic disorders. Leuk Lymphoma 53 (3): 430-4, 2012.
    41. Mesa RA, Nagorney DS, Schwager S, et al.: Palliative goals, patient selection, and perioperative platelet management: outcomes and lessons from 3 decades of splenectomy for myelofibrosis with myeloid metaplasia at the Mayo Clinic. Cancer 107 (2): 361-70, 2006.
    42. Ruggeri M, Rodeghiero F, Tosetto A, et al.: Postsurgery outcomes in patients with polycythemia vera and essential thrombocythemia: a retrospective survey. Blood 111 (2): 666-71, 2008.
    43. Tefferi A, Mesa RA, Nagorney DM, et al.: Splenectomy in myelofibrosis with myeloid metaplasia: a single-institution experience with 223 patients. Blood 95 (7): 2226-33, 2000.
    44. Sacchi S: The role of alpha-interferon in essential thrombocythaemia, polycythaemia vera and myelofibrosis with myeloid metaplasia (MMM): a concise update. Leuk Lymphoma 19 (1-2): 13-20, 1995.
    45. Gilbert HS: Long term treatment of myeloproliferative disease with interferon-alpha-2b: feasibility and efficacy. Cancer 83 (6): 1205-13, 1998.
    46. Strupp C, Germing U, Scherer A, et al.: Thalidomide for the treatment of idiopathic myelofibrosis. Eur J Haematol 72 (1): 52-7, 2004.
    47. Mesa RA, Elliott MA, Schroeder G, et al.: Durable responses to thalidomide-based drug therapy for myelofibrosis with myeloid metaplasia. Mayo Clin Proc 79 (7): 883-9, 2004.
    48. Jabbour E, Thomas D, Kantarjian H, et al.: Comparison of thalidomide and lenalidomide as therapy for myelofibrosis. Blood 118 (4): 899-902, 2011.
    49. Mesa RA, Camoriano JK, Geyer SM, et al.: A phase II trial of tipifarnib in myelofibrosis: primary, post-polycythemia vera and post-essential thrombocythemia. Leukemia 21 (9): 1964-70, 2007.
    50. Guardiola P, Anderson JE, Bandini G, et al.: Allogeneic stem cell transplantation for agnogenic myeloid metaplasia: a European Group for Blood and Marrow Transplantation, Société Française de Greffe de Moelle, Gruppo Italiano per il Trapianto del Midollo Osseo, and Fred Hutchinson Cancer Research Center Collaborative Study. Blood 93 (9): 2831-8, 1999.
    51. Deeg HJ, Gooley TA, Flowers ME, et al.: Allogeneic hematopoietic stem cell transplantation for myelofibrosis. Blood 102 (12): 3912-8, 2003.
    52. Daly A, Song K, Nevill T, et al.: Stem cell transplantation for myelofibrosis: a report from two Canadian centers. Bone Marrow Transplant 32 (1): 35-40, 2003.
    53. Gupta V, Hari P, Hoffman R: Allogeneic hematopoietic cell transplantation for myelofibrosis in the era of JAK inhibitors. Blood 120 (7): 1367-79, 2012.
    54. 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 114 (26): 5264-70, 2009.
    55. Abelsson J, Merup M, Birgegård G, et al.: The outcome of allo-HSCT for 92 patients with myelofibrosis in the Nordic countries. Bone Marrow Transplant 47 (3): 380-6, 2012.
    56. Alchalby H, Yunus DR, Zabelina T, et al.: Risk models predicting survival after reduced-intensity transplantation for myelofibrosis. Br J Haematol 157 (1): 75-85, 2012.
    57. Alchalby H, Badbaran A, Zabelina T, et al.: Impact of JAK2V617F mutation status, allele burden, and clearance after allogeneic stem cell transplantation for myelofibrosis. Blood 116 (18): 3572-81, 2010.
    58. Tefferi A, Silverstein MN, Li CY: 2-Chlorodeoxyadenosine treatment after splenectomy in patients who have myelofibrosis with myeloid metaplasia. Br J Haematol 99 (2): 352-7, 1997.

    This information is produced and provided by the National Cancer Institute (NCI). The information in this topic may have changed since it was written. For the most current information, contact the National Cancer Institute via the Internet web site at http:// cancer .gov or call 1-800-4-CANCER.

    WebMD Public Information from the National Cancer Institute

    Last Updated: May 28, 2015
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