Polycythemia vera: Current pharmacotherapy and future directions

Expert Opinion on Pharmacotherapy

Volumn 14, Issue 5, 2013, Pages 609-617

  Hensley, Benjamin ^a   Geyer, Holly ^a   Mesa, Ruben ^a  

^a MAYO CLINIC   (United States)

Author keywords

JAK2 inhibitors; Myeloproliferative neoplasms; Polycythemia vera; Ruxolitinib

Indexed keywords

ACETYLSALICYLIC ACID; ALPHA INTERFERON; BUSULFAN; CHLORAMBUCIL; GANDOTINIB; HEMOGLOBIN; HYDROXYUREA; JANUS KINASE 2; N TERT BUTYL 3 [5 METHYL 2 [4 [2 (1 PYRROLIDINYL)ETHOXY]PHENYLAMINO] 4 PYRIMIDINYLAMINO]BENZENESULFONAMIDE; PEGINTERFERON ALPHA2A; PEGINTERFERON ALPHA2B; PIPOBROMAN; PLACEBO; RUXOLITINIB;

ANTICOAGULANT THERAPY; BLEEDING; DRUG EFFICACY; DRUG HYPERSENSITIVITY; DRUG MEGADOSE; DRUG SAFETY; ERYTHROCYTOSIS; FATIGUE; FEVER; GASTRITIS; HEADACHE; HEMATOCRIT; HEMATOPOIESIS; HEMOGLOBIN BLOOD LEVEL; HIGH RISK PATIENT; HUMAN; IMMOBILIZATION; IRON DEFICIENCY; LEUKOCYTE COUNT; LOW DRUG DOSE; MEGALOCYTOSIS; NIGHT SWEAT; PARESTHESIA; PEPTIC ULCER; PHLEBOTOMY; PNEUMONIA; POLYCYTHEMIA VERA; PRURITUS; REVIEW; RISK ASSESSMENT; SATIETY; SKIN DEFECT; SPLENOMEGALY; SYMPTOM ASSESSMENT; THROMBOCYTOSIS; UPPER GASTROINTESTINAL BLEEDING;

ALKYLATING AGENTS; ASPIRIN; HEMATOCRIT; HEMATOLOGIC AGENTS; HEMATOPOIESIS; HUMANS; HYDROXYUREA; INTERFERON-ALPHA; JANUS KINASE 2; POLYCYTHEMIA VERA; POLYETHYLENE GLYCOLS; PREDICTIVE VALUE OF TESTS; PROTEIN KINASE INHIBITORS; PYRAZOLES; RECOMBINANT PROTEINS; RISK ASSESSMENT; RISK FACTORS; TREATMENT OUTCOME;

EID: 84874959297     PISSN: 14656566     EISSN: 17447666     Source Type: Journal    
DOI: 10.1517/14656566.2013.779671     Document Type: Review

References (60)

1. Vaquez LH. Sur une forme speciale de cyanose s'accompagnant d'hyperglobulie excessive et persistante. C R Soc Biol 1892;44:384-8
2. Dameshek W. Some speculations on the myeloproliferative syndromes. Blood 1951;6(4):372-5.
3. Tefferi A, Vardiman JW. Classification and diagnosis of myeloproliferative neoplasms: the 2008 World Health Organization criteria and point-of-care diagnostic algorithms. Leukemia 2008;22(1):14-22.
4. Silverstein MN, Lanier AP. Polycythemia vera, 1935-1969: an epidemiologic survey in Rochester, Minnesota. Mayo Clin Proc 1971;46(11):751-3
5. Polycythemia vera: the natural history of 1213 patients followed for 20 years. Gruppo Italiano Studio Policitemia. Ann Intern Med 1995;123(9):656-64
6. 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
7. Vannucchi AM, Antonioli E, Guglielmelli P, et al. Clinical correlates of JAK2V617F presence or allele burden in myeloproliferative neoplasms: a critical reappraisal. Leukemia 2008;22(7):1299-307
8. Passamonti F, Rumi E, Pietra D, et al. Relation between JAK2 (V617F) mutation status, granulocyte activation, and constitutive mobilization of CD34+ cells into peripheral blood in myeloproliferative disorders. Blood 2006;107(9):3676-82
9. Larsen TS, Pallisgaard N, Christensen JH, et al. New molecular markers within the chronic myeloproliferative disorders. II: the JAK2 mutation. Ugeskr Laeger 2006;168(39):3299-303.
10. Mossuz P, Girodon F, Donnard M, et al. Diagnostic value of serum erythropoietin level in patients with absolute erythrocytosis. Haematologica 2004;89(10):1194-8
11. Tefferi A. Polycythemia vera and essential thrombocythemia: 2012 update on diagnosis, risk stratification, and management. Am J Hematol 2012;87(3):285-93
12. Barbui T, Thiele J, Passamonti F, et al. Survival and disease progression in essential thrombocythemia are significantly influenced by accurate morphologic diagnosis: an international study. J Clin Oncol 2011;29(23):3179-84
13. 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
14. Bircher AJ, Meier-Ruge W. Aquagenic pruritus. Water-induced activation of acetylcholinesterase. Arch Dermatol 1988;124(1):84-9
15. Steinman HK, Greaves MW. Aquagenic pruritus. J Am Acad Dermatol 1985;13(1):91-6
16. Pieri L, Bogani C, Guglielmelli P, et al. The JAK2V617 mutation induces constitutive activation and agonist hypersensitivity in basophils from patients with polycythemia vera. Haematologica 2009;94(11):1537-45
17. 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
18. Tefferi A, Elliott M. Thrombosis in myeloproliferative disorders: prevalence, prognostic factors, and the role of leukocytes and JAK2V617F. Semin Thromb Hemost 2007;33(4):313-20
19. Michiels JJ, Berneman Z, Schroyens W, et al. The paradox of platelet activation and impaired function: platelet-von Willebrand factor interactions, and the etiology of thrombotic and hemorrhagic manifestations in essential thrombocythemia and polycythemia vera. Semin Thromb Hemost 2006;32(6):589-604
20. Scherber R, Dueck A, Kiladjian J-J, et al. The myeloproliferative neoplasm symptom assessment form (MPN-SAF) derived total symptom score (TSS): an international trial of 1433 patients with myeloproliferative neoplasms (MPNs). Blood 2011;116(Suppl):abstract 3839.
21. Passamonti F, Rumi E, Caramella M, et al. A dynamic prognostic model to predict survival in post-polycythemia vera myelofibrosis. Blood 2008;111(7):3383-7
22. Gangat N, Strand J, Li CY, et al. Leucocytosis in polycythaemia vera predicts both inferior survival and leukaemic transformation. Br J Haematol 2007;138(3):354-8
23. 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
24. 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):1574-9
25. Cervantes F, Passamonti F, Barosi G. Life expectancy and prognostic factors in the classic BCR/ABL-negative myeloproliferative disorders. Leukemia 2008;22(5):905-14
26. Passamonti F, Elena C, Schnittger S, et al. Molecular and clinical features of the myeloproliferative neoplasm associated with JAK2 exon 12 mutations. Blood 2011;117(10):2813-16
27. Tefferi A, Rumi E, Finazz G, et al. Survival and prognosis among 1,263 patients with polycythemia vera: an international study. Blood (ASH Annual Meeting Abstracts) 2011;118:abstract 277.
28. Girodon F, Abdulkarim K, Bonicelli G, et al. Leukocytosis and thrombosis at diagnosis are associated with poor survival in polycythemia vera: a population-based study on 327 patients. Blood (ASH Annual Meeting Abstracts) 2011;118:abstract 2819
29. Finazz G, Rumi E, Vannucchi A, et al. Risk factors for thrombosis among 1,545 patients with polycythemia vera: an international study. Blood (ASH Annual Meeting Abstracts) 2012;120:abstract 2849
30. Di Nisio M, Barbui T, Di Gennaro L, et al. The haematocrit and platelet target in polycythemia vera. Br J Haematol 2007;136(2):249-59
31. Pearson TC, Wetherley-Mein G. Vascular occlusive episodes and venous haematocrit in primary proliferative polycythaemia. Lancet 1978;2(8102):1219-22
32. Thomas DJ, du Boulay GH, Marshall J, et al. Cerebral blood-flow in polycythaemia. Lancet 1977;2(8030):161-3
33. Wasserman LR, Balcerzak SP, Berk PD, et al. Influence of therapy on causes of death in polycythemia vera. Trans Assoc Am Physicians 1981;94:30-8
34. 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.
35. Landolfi R, Ciabattoni G, Patrignani P, et al. Increased thromboxane biosynthesis in patients with polycythemia vera: evidence for aspirin-suppressible platelet activation in vivo. Blood 1992;80(8):1965-71
36. 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.
37. Tartaglia AP, Goldberg JD, Berk PD, et al. Adverse effects of antiaggregating platelet therapy in the treatment of polycythemia vera. Semin Hematol 1986;23(3):172-6
38. 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
39. Bjorkholm M, Derolf AR, Hultcrantz M, et al. Treatment-related risk factors for transformation to acute myeloid leukemia and myelodysplastic syndromes in myeloproliferative neoplasms. J Clin Oncol 2011;29(17):2410-15
40. Najean Y, Rain JD. Treatment of polycythemia vera: use of 32P alone or in combination with maintenance therapy using hydroxyurea in 461 patients greater than 65 years of age. The French Polycythemia Study Group. Blood 1997;89(7):2319-27
41. 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
42. Antonioli E, Guglielmelli P, Pieri L, et al. Hydroxyurea-related toxicity in 3,411 patients with Ph'-negative MPN. Am J Hematol 2012;87(5):552-4.
43. Barosi G, Birgegard G, Finazzi G, et al. Response criteria for essential thrombocythemia and polycythemia vera: result of a European LeukemiaNet consensus conference. Blood 2009;113(20):4829-33
44. Alvarez-Larran A, Pereira A, Cervantes F, et al. Assessment and prognostic value of the European LeukemiaNet criteria for clinicohematologic response, resistance, and intolerance to hydroxyurea in polycythemia vera. Blood 2012;119(6):1363-9
45. 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
46. Jabbour E, Kantarjian H, Cortes J, et al. PEG-IFN-alpha-2b therapy in BCR-ABL-negative myeloproliferative disorders: final result of a phase 2 study. Cancer 2007;110(9):2012-18
47. Samuelsson J, Hasselbalch H, Bruserud O, et al. A phase II trial of pegylated interferon alpha-2b therapy for polycythemia vera and essential thrombocythemia: feasibility, clinical and biologic effects, and impact on quality of life. Cancer 2006;106(11):2397-405
48. Kiladjian JJ, Mesa RA, Hoffman R. The renaissance of interferon therapy for the treatment of myeloid malignancies. Blood 2011;117(18):4706-15
49. Gilbert HS. Long term treatment of myeloproliferative disease with interferonalpha- 2b: feasibility and efficacy. Cancer 1998;83(6):1205-13
50. Silver RT. Long-term effects of the treatment of polycythemia vera with recombinant interferon-alpha. Cancer 2006;107(3):451-8
51. Sacchi S, Leoni P, Liberati M, et al. A prospective comparison between treatment with phlebotomy alone and with interferon-alpha in patients with polycythemia vera. Ann Hematol 1994;68(5):247-50
52. Kiladjian JJ, Cassinat B, Chevret S, et al. Pegylated interferon-alfa-2a induces complete hematologic and molecular responses with low toxicity in polycythemia vera. Blood 2008;112(8):3065-72.
53. 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
54. Bai J, Xue YP, Zhang L, et al. Interferon-alpha-2b induces molecular responses of patients with polycythemia vera and its post-polycythemic myelofibrosis. Zhongguo Shi Yan Xue Ye Xue Za Zhi 2011;19(2):444-9
55. Gisslinger H, Kralovics R, Schoder R. Open-abel, prospective, multicenter, phase I/II study of AOP2014, a novel PEG-proline-interferon alpha-2b in patients with polycythemia vera: update from an ongoing study. Blood (ASH Annual Meeting Abstracts) 2011;118:abstract 1747
56. Verstovsek S, Passamonti F, Rambaldi A, et al. Long-term efficacy and safety results from a phase II study of ruxolitinib in patients with polycythemia vera. Blood (ASH Annual Meeting Abstracts) 2012;120:abstract 804.
57. Wernig G, Kharas MG, Okabe R, et al. Efficacy of TG101348, a selective JAK2 inhibitor, in treatment of a murine model of JAK2V617F-induced polycythemia vera. Cancer Cell 2008;13(4):311-20
58. Tefferi A, Cortes J, Hochhaus A, et al. A randomized phase II, open-label trial of orally administered SAR302503 in patients with polycythemia vera (PV) or essential thrombocythemia (ET) who are resistant or intolerant to hydroxyurea. J Clin Oncol 2012;30(Suppl):abstract TPS6641
59. Verstovsek S, Mesa R, Rhoades S, et al. Phase I study of the JAK2 V617F inhibitor, LY2784544, in patients with myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET). Blood (ASH Annual Meeting) 2011;118:2814
60. Najean Y, Rain JD. Treatment of polycythemia vera: the use of hydroxyurea and pipobroman in 292 patients under the age of 65 years. Blood 1997;90(9):3370-7