Therapeutic options for patients with polycythemia vera and essential thrombocythemia refractory/resistant to hydroxyurea

References

• Swerdlow SH, Campo E, Harris NL, et al. World Health Organization classification of tumours of haematopoietic and lymphoid tissues. Lyon: IARC; 2008.
   Google Scholar
• Tefferi A. Polycythemia vera and essential thrombocythemia: 2013 update on diagnosis, risk-stratification, and management. Am J Hematol 2013;88:507–516.
   PubMedGoogle Scholar
• Kiladjian J-J. The spectrum of JAK2-positive myeloproliferative neoplasms. Hematology Am Soc Hematol Educ Program 2012: 561–566.
   Google Scholar
• Vainchenker W, Delhommeau F, Constantinescu SN, et al. New mutations and pathogenesis of myeloproliferative neoplasms. Blood 2011;118:1723–1735.
   PubMed Web of Science ®Google Scholar
• Passamonti F, Rumi E, Pungolino E, et al. Life expectancy and prognostic factors for survival in patients with polycythemia vera and essential thrombocythemia. Am J Med 2004;117:755–761.
   PubMed Web of Science ®Google Scholar
• Mehta J, Wang H, Iqbal SU, et al. Epidemiology of myeloproliferative neoplasms in the United States. Leuk Lymphoma 2014;55:595–600.
   PubMed Web of Science ®Google Scholar
• 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;29:761–770.
   PubMed Web of Science ®Google Scholar
• Alvarez-Larrán 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;119:1363–1369.
   PubMed Web of Science ®Google Scholar
• Hernandez-Boluda JC, Alvarez-Larran A, Gomez M, et al. Clinical evaluation of the European LeukaemiaNet criteria for clinicohaematological response and resistance/intolerance to hydroxycarbamide in essential thrombocythaemia. Br J Haematol 2011;152:81–88.
   PubMed Web of Science ®Google Scholar
• Cervantes F, Passamonti F, Barosi G. Life expectancy and prognostic factors in the classic BCR/ABL-negative myeloproliferative disorders. Leukemia 2008;22:905–914.
   PubMed Web of Science ®Google Scholar
• 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;113:4829–4833.
   PubMed Web of Science ®Google Scholar
• Carobbio A, Finazzi G, Antonioli E, et al. Hydroxyurea in essential thrombocythemia: rate and clinical relevance of responses by European LeukemiaNet criteria. Blood 2010;116:1051–1055.
   PubMed Web of Science ®Google Scholar
• Hernandez-Boluda JC, Pereira A, Cervantes F, et al. Clinical evaluation of the European LeukemiaNet response criteria in patients with essential thrombocythemia treated with anagrelide. Ann Hematol 2013;92:771–775.
   PubMed Web of Science ®Google Scholar
• Barosi G, Mesa R, Finazzi G, et al. Revised response criteria for polycythemia vera and essential thrombocythemia: an ELN and IWG-MRT consensus project. Blood 2013;121:4778–4781.
   PubMed Web of Science ®Google Scholar
• Barosi G, Besses C, Birgegard G, et al. A unified definition of clinical resistance/intolerance to hydroxyurea in essential thrombocythemia: results of a consensus process by an international working group. Leukemia 2007;21:277–280.
   PubMed Web of Science ®Google Scholar
• 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;148:961–963.
   PubMed Web of Science ®Google Scholar
• Stein BL, Tiu RV. Biological rationale and clinical use of interferon in the classical BCR-ABL-negative myeloproliferative neoplasms. J Interferon Cytokine Res 2013;33:145–153.
   PubMed Web of Science ®Google Scholar
• Jonasch E, Haluska FG. Interferon in oncological practice: review of interferon biology, clinical applications, and toxicities. Oncologist 2001;6:34–55.
   PubMed Web of Science ®Google Scholar
• Kiladjian JJ, Chomienne C, Fenaux P. Interferon-alpha therapy in bcr-abl-negative myeloproliferative neoplasms. Leukemia 2008; 22:1990–1998.
   PubMed Web of Science ®Google Scholar
• Kozlowski A, Charles SA, Harris JM. Development of pegylated interferons for the treatment of chronic hepatitis C. BioDrugs 2001;15:419–429.
   PubMed Web of Science ®Google Scholar
• Bruno R, Sacchi P, Cima S, et al. Comparison of peginterferon pharmacokinetic and pharmacodynamic profiles. J Viral Hepatitis 2012;19:33–36.
   PubMed Web of Science ®Google Scholar
• Quintas-Cardama A, Kantarjian HM, Giles F, et al. Pegylated interferon therapy for patients with Philadelphia chromosome-negative myeloproliferative disorders. Semin Thromb Hemost 2006;32:409–416.
   PubMed Web of Science ®Google Scholar
• Foster GR. Pegylated interferons: chemical and clinical differences. Aliment Pharmacol Ther 2004;20:825–830.
   PubMed Web of Science ®Google Scholar
• Bailon P, Palleroni A, Schaffer CA, et al. Rational design of a potent, long-lasting form of interferon: a 40 kDa branched polyethylene glycol-conjugated interferon alpha-2a for the treatment of hepatitis C. Bioconjug Chem 2001;12:195–202.
   PubMed Web of Science ®Google Scholar
• Wang YS, Youngster S, Grace M, et al. Structural and biological characterization of pegylated recombinant interferon alpha-2b and its therapeutic implications. Adv Drug Deliv Rev 2002;54:547–570.
   PubMed Web of Science ®Google Scholar
• 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;112:3065–3072.
   PubMed Web of Science ®Google Scholar
• 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;27:5418–5424.
   PubMed Web of Science ®Google Scholar
• Quintás-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;122:893–901.
   PubMed Web of Science ®Google Scholar
• 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;97:1570–1573.
   PubMed Web of Science ®Google Scholar
• Alvarado Y, Cortes J, Verstovsek S, et al. Pilot study of pegylated interferon-alpha 2b in patients with essential thrombocythemia. Cancer Chemother Pharmacol 2003;51:81–86.
   PubMed Web of Science ®Google Scholar
• 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;90:1333–1338.
   PubMed Web of Science ®Google Scholar
• 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;106:2397–2405.
   PubMed Web of Science ®Google Scholar
• Jabbour E, Kantarjian H, Cortes J, et al. PEG-IFN-α-2b therapy in BCR-ABL-negative myeloproliferative disorders. Cancer 2007;110: 2012–2018.
   PubMed Web of Science ®Google Scholar
• Gisslinger H, Kralovics R, Gisslinger B, et al. AOP2014, a novel peg-proline-interferon alpha-2b with improved pharmacokinetic properties, is safe and well tolerated and shows promising efficacy in patients with polycythemia vera (PV). Blood 2012;120(Suppl. 1): Abstract 175.
   Google Scholar
• Harrison CN, Campbell PJ, Buck G, et al. Hydroxyurea compared with anagrelide in high-risk essential thrombocythemia. N Engl J Med 2005;353:33–45.
   PubMed Web of Science ®Google Scholar
• Ejerblad E, Kvasnicka HM, Thiele J, et al. Diagnosis according to World Health Organization determines the long-term prognosis in patients with myeloproliferative neoplasms treated with anagrelide: results of a prospective long-term follow-up. Hematology 2013;18:8–13.
   PubMed Web of Science ®Google Scholar
• Gisslinger H, Gotic M, Holowiecki J, et al. Anagrelide compared with hydroxyurea in WHO-classified essential thrombocythemia: the ANAHYDRET study, a randomized controlled trial. Blood 2013;121:1720–1728.
   PubMed Web of Science ®Google Scholar
• Christoforidou A, Pantelidou D, Anastasiadis A, et al. Hydroxyurea and anagrelide combination therapy in patients with chronic myeloproliferative diseases resistant or intolerant to monotherapy. Acta Haematol 2008;120:195–198.
   PubMed Web of Science ®Google Scholar
• D’Adda M, Micheletti M, Drera M, et al. The combined use of hydroxyurea and anagrelide allows satisfactory hematologic control in patients with chronic myeloproliferative disorders and thrombocytosis: a report on 13 patients with poor tolerance to hydroxyurea monotherapy. Leuk Lymphoma 2008;49:2216–2218.
   PubMed Web of Science ®Google Scholar
• Santos FP, Verstovsek S. Breakthroughs in myeloproliferative neoplasms. Hematology 2012;17(Suppl. 1):S55–S58.
   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;363:1117–1127.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Passamonti F, Rambaldi A, et al. A phase 2 study of INCB018424, an oral, selective JAK1/JAK2 inhibitor, in patients with advanced polycythemia vera (PV) and essential thrombocythemia (ET) refractory to hydroxyurea. Blood 2009;114(Suppl. 1): Abstract 311.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Passamonti F, Rambaldi A, et al. Durable responses with the JAK1/JAK2 inhibitor, INCB018424, in patients with polycythemia vera (PV) and essential thrombocythemia (ET) refractory or intolerant to hydroxyurea (HU). Blood 2010;116(Suppl. 1): Abstract 313.
   Google Scholar
• 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 2012;120(Suppl. 1): Abstract 804.
   Google Scholar
• 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;150:446–455.
   PubMed Web of Science ®Google Scholar
• 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;161:688–694.
   PubMed Web of Science ®Google Scholar
• Björkholm M, Derolf ÅR, Hultcrantz M, et al. Treatment-related risk factors for transformation to acute myeloid leukemia and myelodysplastic syndromes in myeloproliferative neoplasms. J Clin Oncol 2011;29:2410–2415.
   PubMed Web of Science ®Google Scholar