Treatment of polycythemia vera

Česká verzia

Authors: Petr Dulíček
Authors place of work: IV. interní hematologická klinika LF UK a FN Hradec Králové
Published in the journal: Vnitř Lék 2018; 64(10): 955-960
Category: Přehledné referáty

Summary

Polycythemia vera is a chronic myeloproliferative neoplasm characterized by hematopoietic stem cell-derived clonal myeloproliferation resulting in erythrocytosis, leukocytosis and thrombocytosis. Survival is reduced compared with general population. Main reasons of death include thrombohemorrhagic complications, fibrotic progression and leukaemic transformation. Presence of Janus kinase (JAK2) gene mutations is a diagnostic marker and standard diagnostic criterion. World Health Organization 2016 diagnostic criteria focusing on hemoglobin levels, hematocrit, red cell mass and bone marrow morphology are mandatory. Therapeutic approach depends on stratification of patients according age and personal risk of thrombosis. Low-risk patients are treated first line with low-dose aspirin and phlebotomy. Cytoreduction is indicated in high-risk patients. Interferon-α has demonstrated efficacy in many clinical trials. Its pegylated form is well tolerated, enabling less frequent administration than standard interferon. Therefore it is therapy of choice based on Central European Myeloproliferative Neoplasm Organisation recommendation. Ropeginterferon α-2b has been shown to be more efficacious than hydroxyurea. Hydroxyurea is suspected of leukemogenic potential. JAK1/JAK2 inhibitor ruxolitinib is approved for hydroxyurea resistant/intolerant patients.

Key words:

diagnosis – polycythemia vera – therapy

Zdroje

Tefferi A, Guglielmelli P, Larson DR et al. Long-term survival and blast transformation in molecularly annotated essential thrombocythemia, polycythemia vera, and myelofibrosis. Blood 2014; 124(16): 2507–2513. Dostupné z DOI: <http://dx.doi.org/10.1182/blood-2014–05–579136>.
Tefferi A, Rumi E, Finazzi G et al. Survival and prognosis among 1545 patients with contemporary polycythemia vera: an international study. Leukemia 2013; 27(9): 1874–1881. <http://dx.doi.org/10.1038/leu.2013.163>.
Barbui T, Vannucchi AM, Carobbio A et al. Patterns of presentation and thrombosis outcome in patients with polycythemia vera strictly defined by WHO-criteria and stratified by calendar period of diagnosis. Am J Hematol 2015; 90(5): 434–437. Dostupné z DOI: <http://dx.doi.org/10.1002/ajh.23970>.
Finazzi G. Low-dose aspirin in p. A prospective analysis of thrombotic events in the European collaboration study on low-dose aspirin in polycythemia (ECLAP). Pathol Biol (Paris) 2004; 52(5): 285–288. Dostupné z DOI: <http://dx.doi.org/10.1016/j.patbio.2004.02.007>.
Barbui T, Carobbio A, Rumi E et al. In contemporary patients with polycythemia vera, rates of thrombosis and risk factors delineate a new clinical epidemiology. Blood 2014; 124(19): 3021–3023. Dostupné z DOI: <http://dx.doi.org/10.1182/blood-2014–07–591610>.
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. Dostupné z DOI: <http://dx.doi.org/10.1056/NEJMoa1208500>.
Schwarz J, Ovesná P, Černá O et al. Thrombosis in thrombocythemic Ph-myeloproliferations is associated with higher platelet count prior to the event: results of analyses of prothrombotic risk factors from a registry of patients treated with anagrelide. Eur J Haematol 2016; 96(1): 98–106. <http://dx.doi.org/10.1111/ejh.12554.
Chievitz E, Thiede T Complications and causes of death in polycythaemia vera. Acta Med Scand 1962; 172: 513–523.
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): 114–124. Dostupné z DOI: <http://dx.doi.org/10.1056/NEJMoa035572>.
Finazzi G, Caruso V, Marchioli R et al. Acute leukemia in polycythemia vera. An analysis of 1,638 patients enrolled in a prospective observational study. Blood 2005; 105(7): 2664–2670. Dostupné z DOI: <http://dx.doi.org/10.1182/blood-2004–09–3426>.
Alvarez-Larran A, Kerguelen A, Hernandez-Boluda JC et al. Frequency and prognostic value of resistance/intolerance to hydroxycarbamide in 890 patients with polycythemia vera. Br J Haematol 2016; 172(5): 786–793. Dostupné z DOI: <http://dx.doi.org/10.1111/bjh.13886>.
Quintás-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): 5418–5424. Dostupné z DOI: <http://dx.doi.org/10.1200/JCO.2009.23.6075>.
Gisslinger H, Zagrijtschuk O, Buxhofer-Ausch V et al. Ropeginterferon alfa-2b, a novel IFN alfa-2b, induces high response rates with low toxicity in patients with polycythemia vera. Blood 2015; 126(15): 1762–1769. Dostupné z DOI: <http://dx.doi.org/10.1182/blood-2015–04–637280>.
Vannucchi AM. Ruxolitinib versus standard therapy for the treatment of polycythemia vera. N Eng J Med 2015; 372(5): 426–435. Dostupné z DOI: <http://dx.doi.org/10.1056/NEJMoa1409002>.
Vannucchi AM, Verstovsek S, Guglielmelli P et al. Ruxolitinib reduces JAK2p.V617F allele burden in patients with polycythemia vera enrolled in the RESPONSE study. Ann Hematol 2017; 96(7): 1113–1120. Dostupné z DOI: <http://dx.doi.org/10.1007/s00277–017–2994-x>.
Burgstaller S, Buxhofer-Ausch V, Sliwa T et al. Austrian recommendations for the management of polycythemia vera. Wien Klin Wochenschr 2018; 130(17–18): 535–542. Dostupné z DOI: <http://dx.doi.org/10.1007/s00508–018–1359–3>.
Hatalova A, Schwarz J, Gotic M et al. Recommendations for the diagnosis and treatment of patients with polycythaemia vera. Eur J Haematol 2018. Dostupné z DOI: <http://dx.doi.org/10.1111/ejh.13156>.
Vaa BE, Tefferi A, Gangat N et al. Pruritus in primary myelofibrosis: management options in theera of JAK inhibitors. Ann Hematol 2016; 95(7): 1185–1189. Dostupné z DOI: <http://dx.doi.org/10.1007/s00277–016–2674–2>.
Guglielmelli P, Barosi G, Rambaldi A et al. Safety and efficacy of everolimus, a mTOR inhibitor, as single agent in a phase 1/2 study in patients with myelofibrosis. Blood 2011; 118(8): 2069–2076. Dostupné z DOI: <http://dx.doi.org/10.1182/blood-2011–01–330563>.
Lanotto JC, Couturier MA, Galinat H et al. Administration of direct oral anticoagulants in patients with myeloproliferative neoplasms. Int J Hematol 2017; 106(4): 517–521. Dostupné z DOI: <http://dx.doi.org/10.1007/s12185–017–2282–5>.