2,462
Views
18
CrossRef citations to date
0
Altmetric
Review Article

The evolving treatment paradigm in myelofibrosis

Pages 242-251 | Received 05 Apr 2012, Accepted 07 Jul 2012, Published online: 14 Aug 2012

References

  • Barosi G, Mesa RA, Thiele J, . Proposed criteria for the diagnosis of post-polycythemia vera and post-essential thrombocythemia myelofibrosis: a consensus statement from the International Working Group for Myelofibrosis Research and Treatment. Leukemia 2008;22:437–438.
  • Hoffman R, Rondelli D. Biology and treatment of primary myelofibrosis. Hematology Am Soc Hematol Educ Program 2007:346–354.
  • Mesa RA, Niblack J, Wadleigh M, . The burden of fatigue and quality of life in myeloproliferative disorders (MPDs): an international internet-based survey of 1179 MPD patients. Cancer 2007;109:68–76.
  • Cervantes F, Dupriez B, Pereira A, . New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. Blood 2009;113:2895–2901.
  • Scherber R, Dueck AC, Johansson P, . The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF): international prospective validation and reliability trial in 402 patients. Blood 2011;118:401–408.
  • Mesa RA, Li CY, Ketterling RP, . Leukemic transformation in myelofibrosis with myeloid metaplasia: a single-institution experience with 91 cases. Blood 2005;105:973–977.
  • Wernig G, Kharas MG, Okabe R, . Efficacy of TG101348, a selective JAK2 inhibitor, in treatment of a murine model of JAK2V617f-induced polycythemia vera. Cancer Cell 2008;13:311–320.
  • Mesa RA. How I treat symptomatic splenomegaly in patients with myelofibrosis. Blood 2009;113:5394–5400.
  • Tefferi A. How I treat myelofibrosis. Blood 2011;117:3494–3504.
  • Gangat N, Caramazza D, Vaidya R, . 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;29:392–397.
  • Passamonti F, Cervantes F, Vannucchi AM, . 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;115:1703–1708.
  • Mesa RA, Schwager S, Radia D, . The Myelofibrosis Symptom Assessment Form (MFSAF): an evidence-based brief inventory to measure quality of life and symptomatic response to treatment in myelofibrosis. Leuk Res 2009;33:1199–1203.
  • Cherington C, Slack JL, Leis JF, . Induction therapy followed by allogeneic stem cell transplant for MPN blast phase: outcomes from Mayo Clinic in Arizona. Blood 2011;118(Suppl. 1): Abstract 1749.
  • Scott BL, Gooley TA, Sorror ML, . The Dynamic International Prognostic Scoring System for myelofibrosis predicts outcomes after hematopoietic cell transplantation. Blood 2012;119:2657–2664.
  • Ballen KK, Shrestha S, Sobocinski KA, . Outcome of transplantation for myelofibrosis. Biol Blood Marrow Transplant 2010;16:358–367.
  • Zang DY, Deeg HJ. Allogeneic hematopoietic cell transplantation for patients with myelofibrosis. Curr Opin Hematol 2009;16:140–146.
  • Kroger N, Holler E, Kobbe G, . 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;114:5264–5270.
  • Patriarca F, Bacigalupo A, Sperotto A, . Allogeneic hematopoietic stem cell transplantation in myelofibrosis: the 20-year experience of the Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Haematologica 2008;93:1514–1522.
  • Samuelson S, Sandmaier BM, Heslop HE, . Allogeneic haematopoietic cell transplantation for myelofibrosis in 30 patients 60–78 years of age. Br J Haematol 2011;153:76–82.
  • Tefferi A. Primary myelofibrosis: 2012 update on diagnosis, risk stratification, and management. Am J Hematol 2011;86:1017–1026.
  • Madaan K, Kaushik D, Verma T. Hydroxyurea: a key player in cancer chemotherapy. Expert Rev Anticancer Ther 2012;12:19–29.
  • Cervantes F, Pereira A. Advances in the understanding and management of primary myelofibrosis. Curr Opin Oncol 2011;23:665–671.
  • Martinez-Trillos A, Gaya A, Maffioli M, . Efficacy and tolerability of hydroxyurea in the treatment of the hyperproliferative manifestations of myelofibrosis: results in 40 patients. Ann Hematol 2010;89:1233–1237.
  • Mesa RA, Steensma DP, Pardanani A, . A phase 2 trial of combination low-dose thalidomide and prednisone for the treatment of myelofibrosis with myeloid metaplasia. Blood 2003;101:2534–2541.
  • Thapaliya P, Tefferi A, Pardanani A, . International working group for myelofibrosis research and treatment response assessment and long-term follow-up of 50 myelofibrosis patients treated with thalidomide-prednisone based regimens. Am J Hematol 2011;86:96–98.
  • Mesa RA, Yao X, Cripe LD, . Lenalidomide and prednisone for myelofibrosis: Eastern Cooperative Oncology Group (ECOG) phase 2 trial E4903. Blood 2010;116:4436–4438.
  • Quintas-Cardama A, Kantarjian HM, Manshouri T, . Lenalidomide plus prednisone results in durable clinical, histopathologic, and molecular responses in patients with myelofibrosis. J Clin Oncol 2009;27:4760–4766.
  • Jabbour E, Thomas D, Kantarjian H, . Comparison of thalidomide and lenalidomide as therapy for myelofibrosis. Blood 2011;118:899–902.
  • Kiladjian JJ, Mesa RA, Hoffman R. The renaissance of interferon therapy for the treatment of myeloid malignancies. Blood 2011;117:4706–4715.
  • Gowin K, Thapaliya P, Samuelsson J, . Experience with pegylated interferon alpha-2a in advanced myeloproliferative neoplasms in an international cohort of 118 patients. Haematologica 2012 Mar 14. [Epub ahead of print]
  • Ianotto JC, Kiladjian JJ, Demory JL, . PEG-IFN-α-2a therapy in patients with myelofibrosis. A study of the French Group d’Etudes des Myelofibroses (GEM) and France Intergroupe des syndromes Myeloproliferatifs (FIM). Br J Haematol 2009;146:223–225.
  • Silver RT, Vandris K, Goldman JJ. Recombinant interferon-α may retard progression of early primary myelofibrosis: a preliminary report. Blood 2011;117:6669–6672.
  • Quintas-Cardama A, Tong W, Kantarjian H, . A phase II study of 5-azacytidine for patients with primary and post-essential thrombocythemia/polycythemia vera myelofibrosis. Leukemia 2008; 22:965–970.
  • Nielsen I, Hasselbalch HC. Acute leukemia and myelodysplasia in patients with a Philadelphia chromosome negative chronic myeloproliferative disorder treated with hydroxyurea alone or with hydroxyurea after busulphan. Am J Hematol 2003;74:26–31.
  • Cervantes F, Alvarez-Larran A, Hernandez-Boluda JC, . Erythropoietin treatment of the anaemia of myelofibrosis with myeloid metaplasia: results in 20 patients and review of the literature. Br J Haematol 2004;127:399–403.
  • 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;83:154–155.
  • Mesa RA, Nagorney DS, Schwager S, . 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 2006;107:361–370.
  • Deeg HJ, Gooley TA, Flowers ME, . Allogeneic hematopoietic stem cell transplantation for myelofibrosis. Blood 2003;102:3912–3918.
  • Guardiola P, Anderson JE, Bandini G, . Allogeneic stem cell transplantation for agnogenic myeloid metaplasia: a European Group for Blood and Marrow Transplantation, Societe Francaise de Greffe de Moelle, Gruppo Italiano per il Trapianto del Midollo Osseo, and Fred Hutchinson Cancer Research Center Collaborative Study. Blood 1999;93:2831–2838.
  • Elliott MA, Chen MG, Silverstein MN, . Splenic irradiation for symptomatic splenomegaly associated with myelofibrosis with myeloid metaplasia. Br J Haematol 1998;103:505–511.
  • Baxter EJ, Scott LM, Campbell PJ, . Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders. Lancet 2005;365:1054–1061.
  • James C, Ugo V, Le Couedic JP, . A unique clonal JAK2 mutation leading to constitutive signalling causes polycythaemia vera. Nature 2005;434:1144–1148.
  • Kralovics R, Passamonti F, Buser AS, . A gain-of-function mutation of JAK2 in myeloproliferative disorders. N Engl J Med 2005;352:1779–1790.
  • Levine RL, Wadleigh M, Cools J, . Activating mutation in the tyrosine kinase JAK2 in polycythemia vera, essential thrombocythemia, and myeloid metaplasia with myelofibrosis. Cancer Cell 2005;7:387–397.
  • Quintas-Cardama A, Kantarjian H, Cortes J, . Janus kinase inhibitors for the treatment of myeloproliferative neoplasias and beyond. Nat Rev Drug Discov 2011;10:127–140.
  • Pikman Y, Lee BH, Mercher T, . MPLW515l is a novel somatic activating mutation in myelofibrosis with myeloid metaplasia. PLoS Med 2006;3:e270.
  • Oh ST, Simonds EF, Jones C, . Novel mutations in the inhibitory adaptor protein LNK drive JAK-STAT signaling in patients with myeloproliferative neoplasms. Blood 2010;116:988–992.
  • Delhommeau F, Dupont S, Della Valle V, . Mutation in TET2 in myeloid cancers. N Engl J Med 2009;360:2289–2301.
  • Carbuccia N, Murati A, Trouplin V, . Mutations of ASXl1 gene in myeloproliferative neoplasms. Leukemia 2009;23:2183–2186.
  • Barosi G, Bergamaschi G, Marchetti M, . JAK2 V617F mutational status predicts progression to large splenomegaly and leukemic transformation in primary myelofibrosis. Blood 2007;110:4030–4036.
  • Tefferi A, Lasho TL, Schwager SM, . The JAK2(V617F) tyrosine kinase mutation in myelofibrosis with myeloid metaplasia: lineage specificity and clinical correlates. Br J Haematol 2005;131:320–328.
  • Guglielmelli P, Barosi G, Specchia G, . Identification of patients with poorer survival in primary myelofibrosis based on the burden of JAK2V617F mutated allele. Blood 2009;114:1477–1483.
  • Tefferi A, Lasho TL, Huang J, . Low JAK2V617F allele burden in primary myelofibrosis, compared to either a higher allele burden or unmutated status, is associated with inferior overall and leukemia-free survival. Leukemia 2008;22:756–761.
  • Passamonti F, Rumi E. Clinical relevance of JAK2 (V617F) mutant allele burden. Haematologica 2009;94:7–10.
  • Passamonti F, Rumi E, Pietra D, . A prospective study of 338 patients with polycythemia vera: the impact of Jak2 (V617F) allele burden and leukocytosis on fibrotic or leukemic disease transformation and vascular complications. Leukemia 2010;24:1574–1579.
  • Quintas-Cardama A, Vaddi K, Liu P, . Preclinical characterization of the selective JAK1/2 inhibitor INCB018424: therapeutic implications for the treatment of myeloproliferative neoplasms. Blood 2010;115:3109–3117.
  • Verstovsek S, Kantarjian H, Mesa RA, . Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med 2010;363:1117–1127.
  • Verstovsek S, Mesa RA, Gotlib J, . A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med 2012;366:799–807.
  • Mesa RA, Gotlib J, Gupta V, . Associations between improvements in myelofibrosis (MF) symptoms and quality of life measures with splenomegaly reduction in COMFORT-1: A randomized, double-blind, phase III trial of the JAK1 and JAK2 inhibitor ruxolitinib versus placebo in patients with MF. Blood 2011;118(Suppl. 1): Abstract 3842.
  • Harrison C, Kiladjian JJ, Al-Ali HK, . JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med 2012;366:787–798.
  • Talpaz M, Hamburg SI, Jamieson K, . Preliminary safety and efficacy of ruxolitinib in patients (pts) with primary and secondary myelofibrosis (MF) with platelet counts (PC) of 50–100x109/L. J Clin Oncol 2012;30(Suppl.): Abstract 6630.
  • Mesa RA, Verstovsek S, Cervantes F, . Comparison of the efficacy of placebo and best available therapy for the treatment of myelofibrosis. Blood 2011;18(Suppl. 1): Abstract 1753.
  • Estey EH. Acute myeloid leukemia: 2012 update on diagnosis, risk stratification, and management. Am J Hematol 2012;87:89–99.
  • Li L, Bailey E, Greenblatt S, . Loss of the wild-type allele contributes to myeloid expansion and disease aggressiveness in FLT3/ITD knockin mice. Blood 2011;118:4935–4945.
  • Pardanani A, Gotlib JR, Jamieson C, . Safety and efficacy of TG101348, a selective JAK2 inhibitor, in myelofibrosis. J Clin Oncol 2011;29:789–796.
  • Pardanani A, Gotlib J, Jamieson C, . SAR302503: interim safety, efficacy and long-term impact of JAK2 V617F allele burden in a phase I/II study in patients with myelofibrosis. Blood 2011;118(Suppl. 1): Abstract 3838.
  • Tyner JW, Bumm TG, Deininger J, . CYT387, a novel JAK2 inhibitor, induces hematologic responses and normalizes inflammatory cytokines in murine myeloproliferative neoplasms. Blood 2010;115:5232–5240.
  • Pardanani AD, Caramazza D, George G, . Safety and efficacy of CYT387, a JAK-1/2 inhibitor, for the treatment of myelofibrosis. J Clin Oncol 2011;29(Suppl.): Abstract 6514.
  • Pardanani A, Gotlib J, Gupta V, . An expanded multicenter phase I/II study of CYT387, a JAK-1/2 inhibitor for the treatment of myelofibrosis. Blood 2011;118(Suppl. 1): Abstract 3849.
  • Hart S, Goh KC, Novotny-Diermayr V, . SB1518, a novel macrocyclic pyrimidine-based JAK2 inhibitor for the treatment of myeloid and lymphoid malignancies. Leukemia 2011;25:1751–1759.
  • Komrokji RS, Wadleigh M, Seymour JF, . Results of a phase 2 study of pacritinib (SB1518), a novel oral JAK2 inhibitor, in patients with primary, post-polycythemia vera, and post-essential thrombocythemia myelofibrosis. Blood 2011;118(Suppl. 1): Abstract 282.
  • Deeg HJ, Odenike O, Scott BL, . Phase II study of SB1518, an orally available novel JAK2 inhibitor, in patients with myelofibrosis. J Clin Oncol 2011;29(Suppl.): Abstract 6515.
  • Verstovsek S, Mesa RA, Rhoades SK, . Phase I study of the JAK2 V617F inhibitor, LY2784544, in patients with myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET). Blood 2011;118(Suppl.1): Abstract 2814.
  • Begna KH, Mesa RA, Pardanani A, . A phase-2 trial of low-dose pomalidomide in myelofibrosis. Leukemia 2011;25:301–304.
  • Mesa RA, Pardanani AD, Hussein K, . Phase1/-2 study of pomalidomide in myelofibrosis. Am J Hematol 2010;85:129–130.
  • Begna K, Pardanani A, Mesa RA, . Pomalidomide therapy for myelofibrosis: analysis of results from three consecutive clinical trials. Blood 2011;118(Suppl. 1): Abstract 1759.
  • Guglielmelli P, Barosi G, Rambaldi A, . Safety and efficacy of everolimus, a mTOR inhibitor, as single agent in a phase 1/2 study in patients with myelofibrosis. Blood 2011;118:2069–2076.
  • Quintas-Cardama A, Kantarjian HM, Borthakur G, . Therapy with histone deacetylase inhibitor SB939 for patients with myelofibrosis. Blood 2011;118(Suppl. 1): Abstract 3863.
  • Mascarenhas J, Mercado A, Rodriguez A, . Prolonged low dose therapy with pan-deacetylase inhibitor, panobinostat (LBH589), in patients with myelofibrosis. Blood 2011;118(Suppl. 1): Abstract 794.
  • Rambaldi A, Finazzi G, Vannucchi AM, . A phase II study of the HDAC inhibitor givinostat in combination with hydroxyurea in patients with polycythemia vera resistant to hydroxyurea monotherapy. Blood 2011;118(Suppl. 1): Abstract 1748.
  • Baffert F, Evrot E, Ebel N, . Improved efficacy upon combined JAK1/2 and pan-deacetylase inhibition using ruxolitinib (INC424) and panobinostat (LBH589) in preclinical mouse models of JAK2V617F-driven disease. Blood 2011;118(Suppl. 1): Abstract 798.