396
Views
7
CrossRef citations to date
0
Altmetric
Reviews

Emerging drugs for polycythemia vera

, MD PhD & , MD FACP
Pages 393-404 | Published online: 22 Aug 2013

Bibliography

  • Tibes R, Singh G, Mesa R. Myeloproliferative neoplasms: chronic myelogenous leukemia, polycythemia vera, essential thrombocythemia, and primary myelofibrosis. In: Raghavan D, Blanke CD, Johnson DH, et al. editors. Section 8: hematological malignancies. 4th edition. Wiley-Blackwell, Hoboken NJ; 2012. p. 647-58
  • 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;30(33):4098-103
  • Barbui T, Barosi G, Birgegard G, et al. Philadelphia-negative classical myeloproliferative neoplasms: critical concepts and management recommendations from European LeukemiaNet. J Clin Oncol 2011;29(6):761-70
  • Gowin K, Thapaliya P, Samuelson J, et al. Experience with pegylated interferon alpha-2a in advanced myeloproliferative neoplasms in an international cohort of 118 patients. Haematologica 2012;97(10):1570-3
  • Tibes R, Bogenberger JM, Geyer HL, Mesa RA. JAK2 inhibitors in the treatment of myeloproliferative neoplasms. Expert Opin Investig Drugs 2012;21(12):1755-74
  • Kiladjian JJ, Mesa RA, Hoffman R. The renaissance of interferon therapy for the treatment of myeloid malignancies. Blood 2011;117(18):4706-15
  • Mesa RA, Silverstein MN, Jacobsen SJ, et al. Population-based incidence and survival figures in essential thrombocythemia and agnogenic myeloid metaplasia: an Olmsted County Study, 1976-1995. Am J Hematol 1999;61(1):10-15
  • Moulard O, Mehta J, Olivares R, et al. Epidemiology of Myelofibrosis (MF), Polycythemia Vera (PV) and Essential Thrombocythemia (ET) in the European Union (EU). ASH Annu Meet Abstr 2012;120(21):1496-97
  • Berglund S, Zettervall O. Incidence of polycythemia vera in a defined population. Eur J Haematol 1992;48(1):20-6
  • Fenaux P, Simon M, Caulier MT, et al. Clinical course of essential thrombocythemia in 147 cases. Cancer 1990;66(3):549-56
  • Cortelazzo S, Viero P, Finazzi G, et al. Incidence and risk factors for thrombotic complications in a historical cohort of 100 patients with essential thrombocythemia. J Clin Oncol 1990;8(3):556-62
  • Rozman C, Giralt M, Feliu E, et al. Life expectancy of patients with chronic nonleukemic myeloproliferative disorders. Cancer 1991;67(10):2658-63
  • Landgren O, Goldin LR, Kristinsson SY, et al. Increased risks of polycythemia vera, essential thrombocythemia, and myelofibrosis among 24,577 first-degree relatives of 11,039 patients with myeloproliferative neoplasms in Sweden. Blood 2008;112(6):2199-204
  • 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
  • Tefferi A, Vainchenker W. Myeloproliferative neoplasms: molecular pathophysiology, essential clinical understanding, and treatment strategies. J Clin Oncol 2011;29(5):573-82
  • Vannucchi AM, Guglielmelli P, Tefferi A. Advances in understanding and management of myeloproliferative neoplasms. CA Cancer J Clin 2009;59(3):171-91
  • Mesa RA, Tibes R. MPN blast phase: clinical challenge and assessing response. Leuk Res 2012;36(12):1496-7
  • Cherington C, Slack JL, Leis J, et al. Allogeneic stem cell transplantation for myeloproliferative neoplasm in blast phase. Leuk Res 2012;36(9):1147-51
  • Kiladjian JJ, Chevret S, Dosquet C, et al. Treatment of polycythemia vera with hydroxyurea and pipobroman: final results of a randomized trial initiated in 1980. J Clin Oncol 2011;29(29):3907-13
  • 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):937-51
  • Marchioli R, Finazzi G, Landolfi R, et al. Vascular and neoplastic risk in a large cohort of patients with polycythemia vera. J Clin Oncol 2005;23(10):2224-32
  • McMullin MF, Bareford D, Campbell P, et al. Guidelines for the diagnosis, investigation and management of polycythaemia/erythrocytosis. Br J Haematol 2005;130(2):174-95
  • Landolfi R, Marchioli R, Kutti J, et al. Efficacy and Safety of Low-Dose Aspirin in Polycythemia Vera. N Engl J Med 2004;350(2):114-24
  • Marchioli R, Finazzi G, Specchia G, et al. Cardiovascular events and intensity of treatment in polycythemia vera. N Engl J Med 2013;368(1):22-33
  • Harrison CN, Campbell PJ, Buck G, et al. Hydroxyurea Compared with Anagrelide in High-Risk Essential Thrombocythemia. N Engl J Med 2005;353(1):33-45
  • Cortelazzo S, Finazzi G, Ruggeri M, et al. Hydroxyurea for patients with essential thrombocythemia and a high risk of thrombosis. N Engl J Med 1995;332(17):1132-6
  • Finazzi G, Caruso V, Marchioli R, et al. Acute leukemia in polycythemia vera: an analysis of 1638 patients enrolled in a prospective observational study. Blood 2005;105(7):2664-70
  • Barosi G, Birgegard G, Finazzi G, et al. A unified definition of clinical resistance and intolerance to hydroxycarbamide in polycythaemia vera and primary myelofibrosis: results of a European LeukemiaNet (ELN) consensus process. Br J Haematol 2010;148(6):961-3
  • Mesa R, Mehta J, Wang H, et al. Epidemiology of Myeloproliferative Disorders in US - A Real World Analysis [Oral and poster Abstracts #2834]. 54th ASH Annual Meeting and Exposition #2834 2012 December 9; 2012
  • US Census Bureau Data 2012 Economic Indicators. Available from: http://www.census.gov/ [Cited 2012]
  • Tibes R, Mesa RA. Blood consult: resistant and progressive essential thrombocythemia. Blood 2011;118(2):240-2
  • Tibes R, Trent J, Kurzrock R. Tyrosine kinase inhibitors and the dawn of molecular cancer therapeutics. Annu Rev Pharmacol Toxicol 2005;45:357-84
  • Tibes R, Mesa RA. Myeloproliferative neoplasms 5 years after discovery of JAK2V617F: what is the impact of JAK2 inhibitor therapy? Leuk Lymphoma 2011;52(7):1178-87
  • Roder S, Steimle C, Meinhardt G, Pahl HL. STAT3 is constitutively active in some patients with Polycythemia rubra vera. Exp Hematol 2001;29(6):694-702
  • Kirito K, Uchida M, Yamada M, et al. A distinct function of STAT proteins in erythropoietin signal transduction. J Biol Chem 1997;272(26):16507-13
  • 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
  • Dai CH, Krantz SB, Means RT Jr, et al. Polycythemia vera blood burst-forming units-erythroid are hypersensitive to interleukin-3. J Clin Invest 1991;87(2):391-6
  • Baxter EJ, Scott LM, Campbell PJ, et al. Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders. Lancet 2005;365(9464):1054-61
  • 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):1144-8
  • 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):387-97
  • Zhao R, Xing S, Li Z, et al. Identification of an acquired JAK2 mutation in polycythemia vera. J Biol Chem 2005;280(24):22788-92
  • Pardanani A, Lasho TL, Finke C, et al. Prevalence and clinicopathologic correlates of JAK2 exon 12 mutations in JAK2V617F-negative polycythemia vera. Leukemia 2007;21(9):1960-3
  • Langemeijer SM, Kuiper RP, Berends M, et al. Acquired mutations in TET2 are common in myelodysplastic syndromes. Nat Genet 2009;41(7):838-42
  • Delhommeau F, Dupont S, Della Valle V, et al. Mutation in TET2 in myeloid cancers. N Engl J Med 2009;360(22):2289-301
  • Abdel-Wahab O, Mullally A, Hedvat C, et al. Genetic characterization of TET1, TET2, and TET3 alterations in myeloid malignancies. Blood 2009;114(1):144-7
  • Stegelmann F, Bullinger L, Schlenk RF, et al. DNMT3A mutations in myeloproliferative neoplasms. Leukemia 2011;25(7):1217-19
  • Brecqueville M, Rey J, Bertucci F, et al. Mutation analysis of ASXL1, CBL, DNMT3A, IDH1, IDH2, JAK2, MPL, NF1, SF3B1, SUZ12, and TET2 in myeloproliferative neoplasms. Genes Chromosomes Cancer 2012;51(8):743-55
  • Ernst T, Chase AJ, Score J, et al. Inactivating mutations of the histone methyltransferase gene EZH2 in myeloid disorders. Nat Genet 2010;42(8):722-6
  • Teofili L, Martini M, Cenci T, et al. Epigenetic alteration of SOCS family members is a possible pathogenetic mechanism in JAK2 wild type myeloproliferative diseases. Int J Cancer 2008;123(7):1586-92
  • Fourouclas N, Li J, Gilby DC, et al. Methylation of the suppressor of cytokine signaling 3 gene (SOCS3) in myeloproliferative disorders. Haematologica 2008;93(11):1635-44
  • Suessmuth Y, Elliott J, Percy MJ, et al. A new polycythaemia vera-associated SOCS3 SH2 mutant (SOCS3F136L) cannot regulate erythropoietin responses. Br J Haematol 2009;147(4):450-8
  • 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):1140-51
  • Platanias LC. Mechanisms of type-I- and type-II-interferon-mediated signalling. Nat Rev Immunol 2005;5(5):375-86
  • Barosi G, Liberato LN, Costa A, Ascari E. Cytoreductive effect of recombinant alpha interferon in patients with myelofibrosis with myeloid metaplasia. Blut 1989;58(6):271-4
  • Kiladjian JJ, Chomienne C, Fenaux P. Interferon-alpha therapy in bcr-abl-negative myeloproliferative neoplasms. Leukemia 2008;22(11):1990-8
  • Quintas-Cardama A, Kantarjian H, Manshouri T, et al. Pegylated interferon alfa-2a yields high rates of hematologic and molecular response in patients with advanced essential thrombocythemia and polycythemia vera. J Clin Oncol 2009;27(32):5418-24
  • Quintas-Cardama A, Abdel-Wahab O, Manshouri T, et al. Molecular analysis of patients with polycythemia vera or essential thrombocythemia receiving pegylated interferon alpha-2a. Blood 2013; [Epub ahead of print]
  • Mookerjee B. Switch Study From Hydroxyurea to Ruxolitinib for RELIEF of Polycythemia Vera Symptoms: The Relief Study. [ClinicalTrails.gov U.S. National Institutes of Health]. 2012. Available from: http://clinicaltrials.gov/ct2/show/record/NCT01632904 [Cited 2013 01/10]
  • Verstovsek S, Mookerjee B. Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 (INCB018424) Tablets Versus Best Available Care. 2010. Available from: http://clinicaltrials.gov/ct2/show/record/NCT01243944 [Cited 2013 01/10]
  • Besses C, Alvarez-Larran A, Martinez-Aviles L, et al. Modulation of JAK2 V617F allele burden dynamics by hydroxycarbamide in polycythaemia vera and essential thrombocythaemia patients. Br J Haematol 2011;152(4):413-19
  • Antonioli E, Carobbio A, Pieri L, et al. Hydroxyurea does not appreciably reduce JAK2 V617F allele burden in patients with polycythemia vera or essential thrombocythemia. Haematologica 2010;95(8):1435-8
  • Shih AH, Abdel-Wahab O, Patel JP, Levine RL. The role of mutations in epigenetic regulators in myeloid malignancies. Nat Rev Cancer 2012;12(9):599-612
  • 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):3735-42
  • Mesa RA, Verstovsek S, Rivera C, et al. 5-Azacitidine has limited therapeutic activity in myelofibrosis. Leukemia 2009;23(1):180-2
  • Rambaldi A, Dellacasa CM, Finazzi G, et al. A pilot study of the Histone-Deacetylase inhibitor Givinostat in patients with JAK2V617F positive chronic myeloproliferative neoplasms. Br J Haematol 2010;150(4):446-55
  • Finazzi G, Vannucchi AM, Martinelli V, et al. A phase II study of Givinostat in combination with hydroxycarbamide in patients with polycythaemia vera unresponsive to hydroxycarbamide monotherapy. Br J Haematol 2013;161(5):688-94
  • Andersen CL, McMullin MF, Ejerblad E, et al. A phase II study of vorinostat (MK-0683) in patients with polycythaemia vera and essential thrombocythaemia. Br J Haematol 2013.162(4): 498-508. Available from: http://www.ncbi.nlm.nih.gov/pubmed/23758082
  • Nussenzveig RH, Cortes J, Sever M, et al. Imatinib mesylate therapy for polycythemia vera: final result of a phase II study initiated in 2001. Int J Hematol 2009;90(1):58-63
  • Langer C, Lengfelder E, Thiele J, et al. Pegylated interferon for the treatment of high risk essential thrombocythemia: results of a phase II study. Haematologica 2005;90(10):1333-8
  • 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):1117-27
  • Taylor T, Gyves P, Burgunder JM. Thyroid hormone regulation of TRH mRNA levels in rat paraventricular nucleus of the hypothalamus changes during ontogeny. Neuroendocrinology 1990;52(3):262-7
  • Pardanani A, Gotlib JR, Jamieson C, et al. Safety and efficacy of TG101348, a selective JAK2 inhibitor, in myelofibrosis. J Clin Oncol 2011;29(7):789-96

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.