Recent developments in myelofibrosis

References

• Vardiman JW, Thiele J, Arber DA, et al. The 2008 revision of the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia: rationale and important changes. Blood. 2009;114(5):937–951.
   PubMed Web of Science ®Google Scholar
• Dameshek W. Some speculations on the myeloproliferative syndromes. Blood. 1951;6(4):372–375.
   PubMed Web of Science ®Google Scholar
• Correa PN, Eskinazi D, Axelrad AA. Circulating erythroid progenitors in polycythemia vera are hypersensitive to insulin-like growth factor-1 in vitro: studies in an improved serum-free medium. Blood. 1994;83(1):99–112.
   PubMed Web of Science ®Google Scholar
• Dai CH, Krantz SB, Green WE, Gilbert HS. Polycythaemia vera. III. Burst-forming units-erythroid (BEU-E) response to stem cell factor and c-kit receptor expression. Br J Haematol 1994;86(1):12–21.
   Web of Science ®Google Scholar
• Dai CH, Krantz SB, Means RT Jr, Horn ST, Gilbert HS. Polycythemia vera blood burst-forming units-erythroid are hypersensitive to interleukin-3. J Clin Invest. 1991;87(2):391–396.
   PubMed Web of Science ®Google Scholar
• Kobayashi S, Teramura M, Hoshino S, Motoji T, Oshimi K, Mizoguchi H. Circulating megakaryocyte progenitors in myeloproliferative disorders are hypersensitive to interleukin-3. Br J Haematol. 1993;83(4):539–544.
   Web of Science ®Google Scholar
• Baxter EJ, Scott LM, Campbell PJ, et al. Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders. Lancet. 2005;365(9464):1054–1061.
   PubMed Web of Science ®Google Scholar
• James C, Ugo V, Le Couedic JP, et al. A unique clonal JAK2 mutation leading to constitutive signalling causes polycythaemia vera. Nature. 2005;434(7037):1144–1148.
   PubMed Web of Science ®Google Scholar
• Kralovics R, Passamonti F, Buser AS, et al. A gain-of-function mutation of JAK2 in myeloproliferative disorders. N Engl J Med. 2005;352(17):1779–1790.
   PubMed Web of Science ®Google Scholar
• Levine RL, Wadleigh M, Cools J, et al. Activating mutation in the tyrosine kinase JAK2 in polycythemia vera, essential thrombocythemia, and myeloid metaplasia with myelofibrosis. Cancer Cell. 2005;7(4):387–397.
   PubMed Web of Science ®Google Scholar
• Klingmuller U, Wu H, Hsiao JG, et al. Identification of a novel pathway important for proliferation and differentiation of primary erythroid progenitors. Proc Natl Acad Sci U S A. 1997;94(7):3016–3021.
   Web of Science ®Google Scholar
• Walz C, Crowley BJ, Hudon HE, et al. Activated Jak2 with the V617F point mutation promotes G1/S phase transition. J Biol Chem. 2006;281(26):18177–18183.
   PubMed Web of Science ®Google Scholar
• Abdel-Wahab O, Pardanani A, Patel J, et al. Concomitant analysis of EZH2 and ASXL1 mutations in myelofibrosis, chronic myelomonocytic leukemia and blast-phase myeloproliferative neoplasms. Leukemia. 2011;25(7):1200–1202.
   PubMed Web of Science ®Google Scholar
• Delhommeau F, Dupont S, Della Valle V et al. Mutation in TET2 in myeloid cancers. N Engl J Med. 2009;360(22):2289–2301.
   PubMed Web of Science ®Google Scholar
• Pikman Y, Lee BH, Mercher T, et al. MPLW515L is a novel somatic activating mutation in myelofibrosis with myeloid metaplasia. PLoS Med. 2006;3(7):e270.
   PubMed Web of Science ®Google Scholar
• Stegelmann F, Bullinger L, Schlenk RF, et al. DNMT3A mutations in myeloproliferative neoplasms. Leukemia. 2011;25(7):1217–1219.
   PubMed Web of Science ®Google Scholar
• Walkley CR, Olsen GH, Dworkin S, et al. A microenvironment-induced myeloproliferative syndrome caused by retinoic acid receptor gamma deficiency. Cell. 2007;129(6):1097–1110.
   PubMed Web of Science ®Google Scholar
• Walkley CR, Shea JM, Sims NA, Purton LE, Orkin SH. Rb regulates interactions between hematopoietic stem cells and their bone marrow microenvironment. Cell. 2007;129(6):1081–1095.
   PubMed Web of Science ®Google Scholar
• Scherber R, Dueck AC, Johansson P, et al. The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF): international prospective validation and reliability trial in 402 patients. Blood. 2011;118(2):401–408.
   PubMed Web of Science ®Google Scholar
• Tibes R, Mesa RA. Blood consult: resistant and progressive essential thrombocythemia. Blood. 2011;118(2):240–242.
   PubMed Web of Science ®Google Scholar
• Kundranda MN, Tibes R, Mesa RA. Transformation of a chronic myeloproliferative neoplasm to acute myelogenous leukemia: does anything work? CurrHematolMaligRep. 2012;7(1):78–86.
   Google Scholar
• Noor SJ, Tan W, Wilding GE, et al. Myeloid blastic transformation of myeloproliferative neoplasms – a review of 112 cases. Leuk Res. 2011;35(5):608–613.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Silverstein MN, Jacobsen SJ, Wollan PC, Tefferi A. Population-based incidence and survival figures in essential throm- bocythemia and agnogenic myeloid metaplasia: an Olmsted County Study, 1976–1995. Am J Hematol. 1999;61(1):10–15.
   PubMed Web of Science ®Google Scholar
• Visani G, Finelli C, Castelli U, et al. Myelofibrosis with myeloid metaplasia: clinical and haematological parameters predicting survival in a series of 133 patients. Br J Haematol. 1990;75(1):4–9.
   PubMed Web of Science ®Google Scholar
• Bonduel M, Sciuccati G, Torres AF, Pierini A, Gallo G. Familial idiopathic myelofibrosis and multiple hemangiomas. Am J Hematol. 1998;59(2):175–177.
   Web of Science ®Google Scholar
• Saharinen P, Silvennoinen O. The pseudokinase domain is required for suppression of basal activity of Jak2 and Jak3 tyrosine kinases and for cytokine-inducible activation of signal transduction. J Biol Chem. 2002;277(49):47954–47963.
   PubMed Web of Science ®Google Scholar
• Saharinen P, Takaluoma K, Silvennoinen O. Regulation of the Jak2 tyrosine kinase by its pseudokinase domain. Mol Cell Biol. 2000 ;20(10):3387–3395.
   PubMed Web of Science ®Google Scholar
• Ash RC, Detrick RA, Zanjani ED. In vitro studies of human pluripotential hematopoietic progenitors in polycythemia vera. Direct evidence of stem cell involvement. J Clin Invest. 1982;69(5):1112–1118.
   PubMed Web of Science ®Google Scholar
• Tibes R, Mesa RA. JAK2 inhibitors in the treatment of myeloproliferative neoplasms: rationale and clinical data. Clin Investig (Lond). 2011;1(12):1681–1693.
   Google Scholar
• Oh ST, Simonds EF, Jones C, et al. Novel mutations in the inhibitory adaptor protein LNK drive JAK-STAT signaling in patients with myeloproliferative neoplasms. Blood. 2010;116(6):988–992.
   PubMed Web of Science ®Google Scholar
• Pardanani AD, Levine RL, Lasho T, et al. MPL515 mutations in myeloproliferative and other myeloid disorders: a study of 1182 patients. Blood. 2006;108(10):3472–3476.
   PubMed Web of Science ®Google Scholar
• Scott LM, Tong W, Levine RL, et al. JAK2 exon 12 mutations in polycythemia vera and idiopathic erythrocytosis. N Engl J Med. 2007;356(5):459–468.
   PubMed Web of Science ®Google Scholar
• Beer PA, Delhommeau F, Le Couedic JP, et al. Two routes to leukemic transformation after a JAK2 mutation-positive myeloproliferative neoplasm. Blood. 2010;115(14):2891–2900.
   PubMed Web of Science ®Google Scholar
• Tefferi A. Novel mutations and their functional and clinical relevance in myeloproliferative neoplasms: JAK2, MPL, TET2, ASXL1, CBL, IDH and IKZF1. Leukemia. 2010;24(6):1128–1138.
   PubMed Web of Science ®Google Scholar
• Silver RT, Vandris K, Wang YL, et al. JAK2(V617F) allele burden in polycythemia vera correlates with grade of myelofibrosis, but is not substantially affected by therapy. Leuk Res. 2011;35(2):177–182.
   Web of Science ®Google Scholar
• Barosi G, Bergamaschi G, Marchetti M, et al. JAK2 V617F mutational status predicts progression to large splenomegaly and leukemic transformation in primary myelofibrosis. Blood. 2007;110(12):4030–4036.
   PubMed Web of Science ®Google Scholar
• Passamonti F, Rumi E, Pietra D, et al. 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(9):1574–1579.
   PubMed Web of Science ®Google Scholar
• Larsen TS, Pallisgaard N, Moller MB, Hasselbalch HC. The JAK2 V617F allele burden in essential thrombocythemia, polycythemia vera and primary myelofibrosis – impact on disease phenotype. Eur J Haematol. 2007;79(6):508–515.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Lasho TL, Huang J, et al. 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(4):756–761.
   PubMed Web of Science ®Google Scholar
• Guglielmelli P, Barosi G, Specchia G, et al. Identification of patients with poorer survival in primary myelofibrosis based on the burden of JAK2V617F mutated allele. Blood. 2009;114(8):1477–1483.
   PubMed Web of Science ®Google Scholar
• Guglielmelli P, Pancrazzi A, Bergamaschi G, et al. Anaemia characterises patients with myelofibrosis harbouring Mpl mutation. Br J Haematol. 2007;137(3):244–247.
   PubMed Web of Science ®Google Scholar
• 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;115(9):1703–1708.
   PubMed Web of Science ®Google Scholar
• 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;29(4):392–397.
   PubMed Web of Science ®Google Scholar
• Dawson MA, Bannister AJ, Gottgens B, et al. JAK2 phosphorylates histone H3Y41 and excludes HP1alpha from chromatin. Nature. 2009;461(7265):819–822.
   PubMed Web of Science ®Google Scholar
• Griffiths DS, Li J, Dawson MA, et al. LIF-independent JAK signalling to chromatin in embryonic stem cells uncovered from an adult stem cell disease. Nat Cell Biol. 2011;13(1):13–21.
   Web of Science ®Google Scholar
• Rameshwar P, Denny TN, Stein D, Gascon P. Monocyte adhesion in patients with bone marrow fibrosis is required for the production of fibrogenic cytokines. Potential role for interleukin-1 and TGF-beta. J Immunol. 1994;153(6):2819–2830.
   Web of Science ®Google Scholar
• Bock O, Neuse J, Hussein K, et al. Aberrant collagenase expression in chronic idiopathic myelofibrosis is related to the stage of disease but not to the JAK2 mutation status. Am J Pathol. 2006;169(2):471–481.
   Web of Science ®Google Scholar
• Martyre MC, Romquin N, Le Bousse-Kerdiles MC, et al. Transforming growth factor-beta and megakaryocytes in the pathogenesis of idiopathic myelofibrosis. Br J Haematol. 1994;88(1):9–16.
   Web of Science ®Google Scholar
• Kerbauy DM, Gooley TA, Sale GE, et al. Hematopoietic cell transplantation as curative therapy for idiopathic myelofibrosis, advanced polycythemia vera, and essential thrombocythemia. Biol Blood Marrow Transplant. 2007;13(3):355–365.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Hanson CA, Rajkumar SV, Schroeder G, Tefferi A. Evaluation and clinical correlations of bone marrow angiogenesis in myelofibrosis with myeloid metaplasia. Blood. 2000;96(10):3374–3380.
   PubMed Web of Science ®Google Scholar
• Castro-Malaspina H, Jhanwar SC. Properties of myelofibrosis-derived fibroblasts. Prog Clin Biol Res. 1984;154:307–322.
   Google Scholar
• Mesa RA, Kantarjian H, Tefferi A, et al. Evaluating the serial use of the Myelofibrosis Symptom Assessment Form for measuring symptomatic improvement: performance in 87 myelofibrosis patients on a JAK1 and JAK2 inhibitor (INCB018424) clinical trial. Cancer. 2011;117(21):4869–4877.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Niblack J, Wadleigh M, et al. The burden of fatigue and quality of life in myeloproliferative disorders (MPDs): an international Internet-based survey of 1179 MPD patients. Cancer. 2007;109(1):68–76.
   PubMed Web of Science ®Google Scholar
• Liu TT, Chen JB, Chen WJ, Kuo CY, Lee CT. Idiopathic myelofibrosis associated with renal extramedullary hematopoiesis and nephrotic syndrome: case report. Chang Gung Med J. 2000;23(3):169–174.
   Google Scholar
• Merry GM, Aronowitz PB. Myelofibrosis with massive hepatosplenom- egaly and osteolytic bone lesions. J Hosp Med. 2010;5(3):E27–E28.
   Google Scholar
• Koch CA, Li CY, Mesa RA, Tefferi A. Nonhepatosplenic extramedullary hematopoiesis: associated diseases, pathology, clinical course, and treatment. Mayo Clin Proc. 2003;78(10):1223–1233.
   Web of Science ®Google Scholar
• Elliott MA, Pardanani A, Lasho TL, Schwager SM, Tefferi A. Thrombosis in myelofibrosis: prior thrombosis is the only predictive factor and most venous events are provoked. Haematologica. 2010;95(10):1788–1791.
   PubMed Web of Science ®Google Scholar
• Randi ML, Fabris F, Tison T, Barbone E, Rossi C, Girolami A. Prevalence of specific thrombotic accidents in patients with thrombocytosis. Haematologia (Budap). 1993;25(3):149–152.
   Google Scholar
• Wanless IR, Peterson P, Das A, Boitnott JK, Moore GW, Bernier V Hepatic vascular disease and portal hypertension in polycythemia vera and agnogenic myeloid metaplasia: a clinicopathological study of 145 patients examined at autopsy. Hepatology. 1990;12(5):1166–1174.
   Web of Science ®Google Scholar
• Adir Y, Humbert M. Pulmonary hypertension in patients with chronic myeloproliferative disorders. Eur Respir J. 2010;35(6):1396–1406.
   Web of Science ®Google Scholar
• Gupta R, Perumandla S, Patsiornik Y, Niranjan S, Ohri A. Incidence of pulmonary hypertension in patients with chronic myeloproliferative disorders. J Natl Med Assoc. 2006;98(11):1779–1182.
   PubMed Web of Science ®Google Scholar
• Garypidou V, Vakalopoulou S, Dimitriadis D, Tziomalos K, Sfikas G, Perifanis V. Incidence of pulmonary hypertension in patients with chronic myeloproliferative disorders. Haematologica. 2004;89(2):245–246.
   PubMed Web of Science ®Google Scholar
• Thiele J, Kvasnicka HM, Fischer R. Histochemistry and morphometry on bone marrow biopsies in chronic myeloproliferative disorders – aids to diagnosis and classification. Ann Hematol. 1999;78(11):495–506.
   Web of Science ®Google Scholar
• Johansson P, Mesa R, Scherber R, et al. Association between quality of life and clinical parameters in patients with myeloproliferative neoplasms. Leuk Lymphoma. 2012;53(3):441–444.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Li CY, Ketterling RP, Schroeder GS, Knudson RA, Tefferi A. Leukemic transformation in myelofibrosis with myeloid metaplasia: a single-institution experience with 91 cases. Blood. 2005;105(3):973–977.
   PubMed Web of Science ®Google Scholar
• Dupriez B, Morel P, Demory JL, et al. Prognostic factors in agnogenic myeloid metaplasia: a report on 195 cases with a new scoring system. Blood. 1996;88(3):1013–1018.
   PubMed Web of Science ®Google Scholar
• Tefferi A. Primary myelofibrosis: 2012 update on diagnosis, risk stratification, and management. Am J Hematol. 2011;86(12):1017–1026.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Thiele J, Orazi A, et al. Proposals and rationale for revision of the World Health Organization diagnostic criteria for polycythemia vera, essential thrombocythemia, and primary myelofibrosis: recommendations from an ad hoc international expert panel. Blood. 2007;110(4):1092–1097.
   PubMed Web of Science ®Google Scholar
• Courcoutsakis N, Spanoudaki A, Maris TG, et al. Perfusion parameters analysis of the vertebral bone marrow in patients with Ph1- chronic myeloproliferative neoplasms (Ph(neg) MPN): a dynamic contrast- enhanced MRI (DCE-MRI) study. J Magn Reson Imaging. 2012;35(3):696–702.
   Web of Science ®Google Scholar
• Hussein K, Van Dyke DL, Tefferi A. Conventional cytogenetics in myelofibrosis: literature review and discussion. Eur J Haematol. 2009;82(5):329–338.
   PubMed Web of Science ®Google Scholar
• Zhao R, Xing S, Li Z, et al. Identification of an acquired JAK2 mutation in polycythemia vera. J Biol Chem. 2005;280(24):22788–22792.
   PubMed Web of Science ®Google Scholar
• Barosi G, Mesa RA, Thiele J, et al. 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(2):437–438.
   PubMed Web of Science ®Google Scholar
• Cervantes F, Alvarez-Larran A, Hernandez-Boluda JC, et al. Darbepoetin-alpha for the anaemia of myelofibrosis with myeloid metaplasia. Br J Haematol. 2006;134(2):184–186.
   PubMed Web of Science ®Google Scholar
• Silver RT, Jenkins DE Jr, Engle RL Jr. Use of testosterone and busulfan in the treatment of myelofibrosis with myeloid metaplasia. Blood. 1964;23:341–353.
   Web of Science ®Google Scholar
• Kennedy BJ. Effect of androgenic hormone in myelofibrosis. JAMA. 1962;182:116–119.
   PubMed Web of Science ®Google Scholar
• Hast R, Engstedt L, Jameson S, et al. Oxymetholone treatment in myelofibrosis. Blut. 1978;37(1):19–26.
   PubMedGoogle Scholar
• Besa EC, Nowell PC, Geller NL, Gardner FH. Analysis of the androgen response of 23 patients with agnogenic myeloid metaplasia: the value of chromosomal studies in predicting response and survival. Cancer. 1982;49(2):308–313.
   Web of Science ®Google Scholar
• Brubaker LH, Briere J, Laszlo J, et al. Treatment of anemia in myeloproliferative disorders: a randomized study of fluoxymesterone v transfusions only. Arch Intern Med. 1982;142(8):1533–1537.
   PubMedGoogle Scholar
• Levy V, Bourgarit A, Delmer A, et al. Treatment of agnogenic myeloid metaplasia with danazol: a report of four cases. Am J Hematol. 1996;53(4):239–241.
   Web of Science ®Google Scholar
• Cervantes F, Hernandez-Boluda JC, Alvarez A, Nadal E, Montserrat E. Danazol treatment of idiopathic myelofibrosis with severe anemia. Haematologica. 2000;85(6):595–599.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Cortes J, Verstovsek S, et al. Lenalidomide therapy in myelofibrosis with myeloid metaplasia. Blood. 2006;108(4):1158–1164.
   PubMed Web of Science ®Google Scholar
• Quintas-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. 2009;27(28):4760–4766.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Yao X, Cripe LD, et al. Lenalidomide and prednisone for myelofibrosis: Eastern Cooperative Oncology Group (ECOG) phase 2 trial E4903. Blood. 2010;116(22):4436–4438.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Lasho TL, Mesa RA, Pardanani A, Ketterling RP, Hanson CA. Lenalidomide therapy in del(5)(q31)-associated myelofibrosis: cytogenetic and JAK2V617F molecular remissions. Leukemia. 2007;21(8):1827–1828.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Elliott MA, Schroeder G, Tefferi A. Durable responses to thalidomide-based drug therapy for myelofibrosis with myeloid metaplasia. Mayo Clin Proc. 2004;79(7):883–889.
   PubMed Web of Science ®Google Scholar
• Thomas DA, Giles FJ, Albitar M, et al. Thalidomide therapy for myelofibrosis with myeloid metaplasia. Cancer. 2006;106(9):1974–1984.
   PubMed Web of Science ®Google Scholar
• 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. 2004;22(3):424–431.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Verstovsek S, Barosi G, et al. Pomalidomide is active in the treatment of anemia associated with myelofibrosis. J Clin Oncol. 2009;27(27):4563–4569.
   PubMed Web of Science ®Google Scholar
• Begna KH, Pardanani A, Mesa RA, et al. Long-term outcome of pomalidomide therapy in myelofibrosis. Am J Hematol. 2012;87(1):66–68.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Pardanani AD, Hussein K, et al. Phase1/-2 study of pomalidomide in myelofibrosis. Am J Hematol. 2010;85(2):129–130.
   PubMed Web of Science ®Google Scholar
• Kroger N, Mesa RA. Choosing between stem cell therapy and drugs in myelofibrosis. Leukemia. 2008;22(3):474–486.
   Web of Science ®Google Scholar
• Mesa RA. Transplantation for myelofibrosis: time for a randomized trial. LeukRes. 2011;35(8):987–988.
   Web of Science ®Google Scholar
• Martinez-Trillos A, Gaya A, Maffioli M, et al. Efficacy and tolerability of hydroxyurea in the treatment of the hyperproliferative manifestations of myelofibrosis: results in 40 patients. Ann Hematol. 2010;89(12):1233–1237.
   PubMed Web of Science ®Google Scholar
• Mesa RA. How I treat symptomatic splenomegaly in patients with myelofibrosis. Blood. 2009;113(22):5394–5400.
   PubMed Web of Science ®Google Scholar
• Silver RT, Vandris K, Goldman JJ. Recombinant interferon-a may retard progression of early primary myelofibrosis: a preliminary report. Blood. 2011;117(24):6669–6672.
   PubMed Web of Science ®Google Scholar
• Ianotto JC, Kiladjian JJ, Demory JL, et al. PEG-IFN-alpha-2a therapy in patients with myelofibrosis: a study of the French Groupe d’Etudes des Myelofibroses (GEM) and France Intergroupe des syndromes Myeloproliferatifs (FIM). Br J Haematol. 2009;146(2):223–225.
   PubMedGoogle Scholar
• Abdel-Wahab O, Manshouri T, Patel J, et al. Genetic analysis of transforming events that convert chronic myeloproliferative neoplasms to leukemias. Cancer Res. 2010;70(2):447–452.
   PubMed Web of Science ®Google Scholar
• Thepot S, Itzykson R, Seegers V et al. Treatment of progression of Philadelphia-negative myeloproliferative neoplasms to myelodysplastic syndrome or acute myeloid leukemia by azacitidine: a report on 54 cases on the behalf of the Groupe Francophone des Myelodysplasies (GFM). Blood. 2010;116(19):3735–3742.
   PubMed Web of Science ®Google Scholar
• Tibes R, Mesa RA. Myeloproliferative neoplasms 5 years after discovery of JAK2V617F: what is the impact of JAK2 inhibitor therapy? LeukLymphoma. 2011;52(7):1178–1187.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Kantarjian H, Mesa RA, et al. Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med. 2010;363(12):1117–1127.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa RA, Gotlib JR, et al. Results of COMFORT-I, a randomized double-blind phase III trial of JAK1/2 inhibitor INCB18424 (424) versus placebo (PB) for patients with myelofibrosis (MF) [abstract]. J Clin Oncol. 2011;29 Suppl:6500.
   Google Scholar
• Harrison CN, Kiladjian JJ, Al-Ali HK, et al. Results of a randomized study of the JAK inhibitor ruxolitinib (INC424) versus best available therapy (BAT) in primary myelofibrosis (PMF), post-polycythemia vera-myelofibrosis (PPV-MF) or post-essential thrombocythemia- myelofibrosis (PET-MF) [abstract]. J Clin Oncol. 2011 ;29 Suppl: LBA6501.
   Google Scholar
• Verstovsek S, Kiladjian J, Waltzman RJ, et al. RESPONSE: a randomized, open label, phase III study of INC424 in polycythemia vera (PV) patients resistant to or intolerant of hydroxyurea (HU) [abstract]. J Clin Oncol. 2011;29 Suppl:TPS203.
   Web of Science ®Google Scholar
• Tyner JW, Bumm TG, Deininger J, et al. CYT387, a novel JAK2 inhibitor, induces hematologic responses and normalizes inflammatory cytokines in murine myeloproliferative neoplasms. Blood. 2010;115(25):5232–5240.
   PubMed Web of Science ®Google Scholar
• Pardanani A, George G, Lasho T, et al. A phase I/II study of CYT387, an oral JAK-1/2 inhibitor, in myelofibrosis: significant response rates in anemia, splenomegaly, and constitutional symptoms. Paper presented at: 52 nd American Society of Hematology Annual Meeting. December 4–6, 2010; Orlando, FL.
   Google Scholar
• Ma L, Zhao B, Walgren RA, et al. Efficacy of LY2784544, a small molecule inhibitor selective for mutant JAK2 kinase, in JAK2 V617F-induced hematologic malignancy models. Paper presented at: 5 2nd American Society of Hematology Annual Meeting. December 4–6, 2010; Orlando, FL.
   Google Scholar
• Shide K, Kameda T, Markovtsov V et al. Efficacy of R723, a potent and selective JAK2 inhibitor, in JAK2V617F-induced murine MPD model. Paper presented at: 51st American Society of Hematology Annual Meeting. December 5–8, 2009; New Orleans, LA.
   Google Scholar
• Shide K, Nakaya Y, Kameda T, et al. NS-018, a potent novel JAK2 inhibitor, effectively treats murine MPN induced by the Janus kinase 2 (JAK2) V617F mutant. Paper presented at: 52nd American Society of Hematology Annual Meeting. December 4–6, 2010; Orlando, FL.
   Google Scholar
• Mascarenhas J, Wang X, Rodriguez A, et al. A phase I study of LBH589, a novel histone deacetylase inhibitor in patients with primary myelofibrosis (PMF) and post-polycythemia/essential thrombocythemia (post-PV/ET MF). Paper presented at: 51st American Society of Hematology Annual Meeting. December 5–8, 2009; New Orleans, LA.
   Google Scholar
• Rambaldi A, Dellacasa CM, Salmoiraghi S, et al. A phase 2A study of the histone-deacetylase inhibitor ITF23 57 in patients with JAK2V617F positive chronic myeloproliferative neoplasms. Paper presented at: 50th American Society of Hematology Annual Meeting. December 6–9, 2008; San Francisco, CA.
   Google Scholar
• Vannucchi AM, Guglielmelli P, Lupo L, et al. A phase 1/2 study of RAD001, a mTOR inhibitor, in patients with myelofibrosis: final results. Paper presented at: 52nd American Society of Hematology Annual Meeting. December 4–6, 2010; Orlando, FL.
   Google Scholar
• Barosi G, Elliott M, Canepa L, et al. Thalidomide in myelofibrosis with myeloid metaplasia: a pooled-analysis of individual patient data from five studies. Leuk Lymphoma. 2002;43(12):2301–2307.
   PubMed Web of Science ®Google Scholar