Optimal therapy for polycythemia vera and essential thrombocythemia: Preferred use of interferon therapy based on phase 2 trials

References

• Vannucchi AM. How I treat polycythemia vera. Blood. 2014;124(22):3212–20. doi: 10.1182/blood-2014-07-551929
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Mesa R, Prchal J, Hoffman R. Optimal therapy for polycythemia vera and essential thrombocythemia can only be determined by the completion of randomized clinical trials. Haematologica. 2014;99(6):945–9. http://dx.doi.org/10.3324/haematol.2014.106013.
   PubMed Web of Science ®Google Scholar
• Nelson HD, Haney EM, Dana T, Bougatsos C, Chou R, U.S. Preventive Services Task Force. Screening for breast cancer: an update for the U.S. Preventive Services Task Force. Ann Intern Med. 2009;151(10):727–37, W237–42. doi: 10.7326/0003-4819-151-10-200911170-00009
   PubMed Web of Science ®Google Scholar
• Gøtzsche PC, Jørgensen KJ. Screening for breast cancer with mammography. Cochrane Database Syst Rev. 2013;6:CD001877.
   PubMed Web of Science ®Google Scholar
• Marmot MG, Altman DG, Cameron DA, Dewar JA, Thompson SG, Wilcox M. The benefits and harms of breast cancer screening: an independent review. Br J Cancer. 2013;108(11):2205–40. http://dx.doi.org/10.1038/bjc.2013.177.
   PubMed Web of Science ®Google Scholar
• Dagher R, Johnson J, Williams G, Keegan P, Pazdur R. Accelerated approval of oncology products: a decade of experience. J Natl Cancer Inst. 2004;96(20):1500–9. doi: 10.1093/jnci/djh279
   PubMedGoogle Scholar
• Hoffman R, Silver RT, Harrison C, Alessandro R, Myeloproliferative Disorders-Research Consortium (MPD-RC), MPD-RC 112: Randomized trial of pegylated interferon alfa-2a versus hydroxyurea in polycythemia vera (PV) and essential thrombocythemia (ET). In: ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2000 [cited 2012 June 15]. Available from: http://clinicaltrials.gov/ct2/show/NCT01259856.
   Google Scholar
• Cassinat B, Laguillier C, Gardin C, de Beco V, Burcheri S, Fenaux P, et al. PV-Nord Group. Classification of myeloproliferative disorders in the JAK2 era: is there a role for red cell mass? Leukemia. 2008;22(2):452–3. http://dx.doi.org/10.1038/sj.leu.2404908.
   PubMed Web of Science ®Google Scholar
• Johansson PL, Safai-Kutti S, Kutti J. An elevated venous haemoglobin concentration cannot be used as a surrogate marker for absolute erythrocytosis: a study of patients with polycythaemia vera and apparent polycythaemia. Br J Haematol. 2005;129(5):701–5. doi: 10.1111/j.1365-2141.2005.05517.x
   PubMed Web of Science ®Google Scholar
• Spivak JL, Silver RT. The treatment of essential thrombocytosis revisited. Blood. 2011;118(4):1179–80. http://dx.doi.org/10.1182/blood-2011-03-341925.
   PubMed Web of Science ®Google Scholar
• Alvarez-Larrán A, Ancochea A, Angona A, Pedro C, García-Pallarols F, Martínez-Avilés L, et al. Red cell mass measurement in patients with clinically suspected diagnosis of polycythemia vera or essential thrombocythemia. Haematologica. 2012;97(11):1704–7. doi: 10.3324/haematol.2012.067348
   PubMed Web of Science ®Google Scholar
• Silver RT, Chow W, Orazi A, Arles SP, Goldsmith SJ. Evaluation of WHO criteria for diagnosis of polycythemia vera: a prospective analysis. Blood. 2013;122(11):1881–6. http://dx.doi.org/10.1182/blood-2013-06-508416
   PubMed Web of Science ®Google Scholar
• Silver RT, Gjoni S. The hematocrit value in polycythemia vera: caveat utilitor. Leuk Lymphoma. 2014;30:1–2.
   Google Scholar
• Barbui T, Thiele J, Gisslinger H, Finazzi G, Carobbio A, Rumi E, et al. Masked polycythemia vera (mPV): results of an international study. Am J Hematol. 2014;89:52–4. doi: 10.1002/ajh.23585
   PubMed Web of Science ®Google Scholar
• Barbui T, Thiele J, Vannucchi AM, Tefferi A. Rethinking the diagnostic criteria of polycythemia vera. Leukemia. 2014;28(6):1191–5. http://dx.doi.org/10.1038/leu.2013.380.
   PubMed Web of Science ®Google Scholar
• Hwang TJ, Bourgeois FT, Seeger JD. Drug safety in the digital age. N Engl J Med. 2014;370(26):2460–2. doi: 10.1056/NEJMp1401767
   PubMed Web of Science ®Google Scholar
• Berlin NI. Prologue: polycythemia vera. The closing of the Wasserman-Polycythemia Vera Study Group era. Semin Hematol. 1997;34(1):1–5.
   PubMed Web of Science ®Google Scholar
• Najean Y, Rain JD. The very long-term evolution of polycythemia vera: an analysis of 318 patients initially treated by phlebotomy or 32P between 1969 and 1981. Semin Hematol. 1997;34(1):6–16.
   PubMed Web of Science ®Google Scholar
• Fretham SJ, Carlson ES, Georgieff MK. The role of iron in learning and memory. Adv Nutr. 2011;2(2):112–21. http://dx.doi.org/10.3945/an.110.000190.
   PubMed Web of Science ®Google Scholar
• Balci K, Utku U, Asil T, Büyükkoyuncu N. Deep cerebral vein thrombosis associated with iron deficiency anaemia in adults. J Clin Neurosci. 2007;14(2):181–4. doi: 10.1016/j.jocn.2005.09.020
   PubMed Web of Science ®Google Scholar
• Avni T, Leibovici L, Gafter-Gvili A. Iron supplementation for the treatment of chronic heart failure and iron deficiency: systematic review and meta-analysis. Eur J Heart Fail. 2012;14(4):423–9. http://dx.doi.org/10.1093/eurjhf/hfs017.
   PubMed Web of Science ®Google Scholar
• Donovan PB, Kaplan ME, Goldberg JD, Tatarsky I, Najean Y, Silberstein EB, et al. Treatment of polycythemia vera with hydroxyurea. Am J Hematol. 1984;17(4):329–34. doi: 10.1002/ajh.2830170402
   PubMed Web of Science ®Google Scholar
• Spivak JL, Hasselbalch H. Hydroxycarbamide: a user's guide for chronic myeloproliferative disorders. Expert Rev Anticancer Ther. 2011;11(3):403–14. http://dx.doi.org/10.1586/era.11.10.
   PubMed Web of Science ®Google Scholar
• Donehower RC. An overview of the clinical experience with hydroxyurea. Semin Oncol. 1992;19(3):11–9.
   PubMed Web of Science ®Google Scholar
• Estève E, Georgescu V, Heitzmann P, Martin L. Multiple skin and mouth squamous cell carcinomas related to long-term treatment with hydroxyurea. Ann Dermatol Venereol. 2001;128(8–9):919–21.
   PubMed Web of Science ®Google Scholar
• Latagliata R, Spadea A, Cedrone M, Di Giandomenico J, De Muro M, Villivà N, et al. Symptomatic mucocutaneous toxicity of hydroxyurea in Philadelphia chromosome-negative myeloproliferative neoplasms: the Mister Hyde face of a safe drug. Cancer. 2012;118(2):404–9. doi: 10.1002/cncr.26194
   PubMed Web of Science ®Google Scholar
• Finazzi G, Caruso V, Marchioli R, Capnist G, Chisesi T, Finelli C, et al. Acute leukemia in polycythemia vera: an analysis of 1638 patients enrolled in a prospective observational study. Blood. 2005;105(7):2664–70. http://dx.doi.org/10.1182/blood-2004-09-3426.
   PubMed Web of Science ®Google Scholar
• Kiladjian JJ, Chevret S, Dosquet C, Chomienne C, Rain JD. Treatment of polycythemia vera with hydroxyurea and pipobroman: final results of a randomized trial initiated in 1980. J Clin Oncol. 2011;129(29):3907–13. doi: 10.1200/JCO.2011.36.0792
   Web of Science ®Google Scholar
• Busque L, Patel JP, Figueroa ME, Vasanthakumar A, Provost S, Hamilou Z, et al. Recurrent somatic TET2 mutations in normal elderly individuals with clonal hematopoiesis. Nat Genet. 2012;44(11):1179–81. http://dx.doi.org/10.1038/ng.2413.
   PubMed Web of Science ®Google Scholar
• Jaiswal S, Fontanillas P, Flannick J, Manning A, Grauman PV, Mar BG, et al. Age-related clonal hematopoiesis associated with adverse outcomes. N Engl J Med. 2014;371(26):2488–98. doi: 10.1056/NEJMoa1408617
   PubMed Web of Science ®Google Scholar
• Abkowitz JL. Clone wars – the emergence of neoplastic blood-cell clones with aging. N Engl J Med. 2014;371(26):2523–5. http://dx.doi.org/10.1056/NEJMe1412902.
   PubMed Web of Science ®Google Scholar
• Wong TE, Brandow AM, Lim W, Lottenberg R. Update on the use of hydroxyurea therapy in sickle cell disease. Blood. 2014;124(26):3850–7. doi: 10.1182/blood-2014-08-435768
   PubMed Web of Science ®Google Scholar
• Lanzkron S, Haywood C Jr, Hassell KL, Rand C. Provider barriers to hydroxyurea use in adults with sickle cell disease: a survey of the Sickle Cell Disease Adult Provider Network. J Natl Med Assoc. 2008;100(8):968–73. http://dx.doi.org/10.1016/S0027-9684(15)31420-6.
   PubMed Web of Science ®Google Scholar
• Essers MA, Offner S, Blanco-Bose WE, Waibler Z, Kalinke U, Duchosal MA, et al. IFNa activates dormant haematopoietic stem cells in vivo. Nature. 2009;458(7240):904–8. doi: 10.1038/nature07815
   PubMed Web of Science ®Google Scholar
• Hasan S, Lacout C, Marty C, Cuingnet M, Solary E, Vainchenker W, et al. JAK2V617F expression in mice amplifies early hematopoietic cells and gives them a competitive advantage that is hampered by IFNa. Blood. 2013;122(8):1464–77. http://dx.doi.org/10.1182/blood-2013-04-498956.
   PubMed Web of Science ®Google Scholar
• Mullally A, Bruedigam C, Poveromo L, Heidel FH, Purdon A, Vu T, et al. Depletion of Jak2V617F myeloproliferative neoplasm-propagating stem cells by interferon-a in a murine model of polycythemia vera. Blood. 2013;121(18):3692–702. doi: 10.1182/blood-2012-05-432989
   PubMed Web of Science ®Google Scholar
• Chott A, Gisslinger H, Thiele J, Fritz E, Linkesch W, Radaszkiewicz T, et al. Interferon-alpha-induced morphological changes of megakaryocytes: a histomorphometrical study on bone marrow biopsies in chronic myeloproliferative disorders with excessive thrombocytosis. Br J Haematol. 1990;74(1):10–6. http://dx.doi.org/10.1111/j.1365-2141.1990.tb02531.x.
   PubMed Web of Science ®Google Scholar
• Silver RT, Vandris K, Goldman JJ. Recombinant interferon-α may retard progression of early primary myelofibrosis: a preliminary report. Blood. 2011;117(24):6669–72. doi: 10.1182/blood-2010-11-320069
   PubMed Web of Science ®Google Scholar
• Kiladjian JJ, Cassinat B, Chevret S, Turlure P, Cambier N, Roussel M, et al. Pegylated interferon-alfa-2a induces complete hematologic and molecular responses with low toxicity in polycythemia vera. Blood. 2008;112:3065–72. http://dx.doi.org/10.1182/blood-2008-03-143537.
   PubMed Web of Science ®Google Scholar
• Quintás-Cardama A, Kantarjian H, Manshouri T, Luthra R, Estrov Z, Pierce S, 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;27:5418–24. doi: 10.1200/JCO.2009.23.6075
   PubMed Web of Science ®Google Scholar
• Larsen TS, Iversen KF, Hansen E, Mathiasen AB, Marcher C, Frederiksen M, et al. Long term molecular responses in a cohort of Danish patients with essential thrombocythemia, polycythemia vera and myelofibrosis treated with recombinant interferon alpha. Leuk Res. 2013;37(9):1041–5. http://dx.doi.org/10.1016/j.leukres.2013.06.012.
   PubMed Web of Science ®Google Scholar
• Quintas-Quardama A, Abdel-Wahab O, Manshouri T, Kilpivaara O, Cortes J, Roupie AL, et al. Molecular analysis of patients with polycythemia vera or essential thrombocythemia receiving pegylated interferon α-2a. Blood. 2013;122(6):893–901. doi: 10.1182/blood-2012-07-442012
   PubMed Web of Science ®Google Scholar
• Cassinat B, Verger E, Kiladjian JJ. Interferon alfa therapy in CALR-mutated essential thrombocythemia. N Engl J Med. 2014;371(2):188–9. http://dx.doi.org/10.1056/NEJMc1401255.
   PubMed Web of Science ®Google Scholar
• Silver RT. Long-term effects of the treatment of polycythemia vera and recombinant interferon-alpha. Cancer. 2006;107(3):451–8. doi: 10.1002/cncr.22026
   PubMed Web of Science ®Google Scholar
• Langer C, Lengfelder E, Thiele J, Kvasnicka HM, Pahl HL, Beneke H, et al. Pegylated interferon for the treatment of high risk essential thrombocythemia: results of a Phase II study. Haematologica. 2005;90:1333–8.
   PubMed Web of Science ®Google Scholar
• Samuelsson J, Hasselbalch H, Bruserod O, Temerinac S, Brandberg Y, Merup M, et al. A Phase II trial of pegylated interferon a-2b therapy for polycythemia vera and essential thrombocythemia. Feasibility, clinical and biologic effects, and impact on quality of life. Cancer. 2006;106:2397–405. http://dx.doi.org/10.1002/cncr.21900.
   PubMed Web of Science ®Google Scholar
• Hasselbalch HC. A new era for IFN-α in the treatment of Philadelphia-negative chronic myeloproliferative neoplasms. Expert Rev Hematol. 2011;4(6):637–55. doi: 10.1586/ehm.11.63
   PubMed Web of Science ®Google Scholar
• Silver RT, Kiladjian JJ, Hasselbalch HC. Interferon and the treatment of polycythemia vera, essential thrombocythemia and myelofibrosis. Expert Rev Hematol. 2013;6(1):49–58. http://dx.doi.org/10.1586/ehm.12.69.
   PubMed Web of Science ®Google Scholar
• Hasselbalch HC, Silver RT. Interferon in polycythemia vera and related neoplasms. Can it become the treatment of choice without a randomized trial? Expert Rev Hematol. 2014;8(4):439–45. doi: 10.1586/17474086.2015.1045409
   Web of Science ®Google Scholar