Seminars in Hematology
Volume 42, Issue 4 , Pages 259-265 , October 2005

Clinical Trials in Myeloproliferative Disorders: Looking Forward

  • Roberto Marchioli

      Affiliations

    • Consorzio Mario Negri Sud, Via Nazionale, Chieti, Italy.
    • Corresponding Author InformationAddress correspondence to Roberto Marchioli, MD, Laboratory of Epidemiology of Cardiovascular Disease, Department of Clinical Pharmacology and Epidemiology, Consorzio Mario Negri Sud, Via Nazionale, 8, 66030 Santa Maria Imbaro, Chieti, Italy.
    • ,
  • Guido Finazzi

      Affiliations

    • Division of Hematology, Ospedali Riuniti, Bergamo, Italy.
    • ,
  • Rosa Maria Marfisi

      Affiliations

    • Consorzio Mario Negri Sud, Via Nazionale, Chieti, Italy.
    • ,
  • Gianni Tognoni

      Affiliations

    • Consorzio Mario Negri Sud, Via Nazionale, Chieti, Italy.
    • ,
  • Tiziano Barbui

      Affiliations

    • Division of Hematology, Ospedali Riuniti, Bergamo, Italy.

References 

  1. International Study of Infarct Survival Collaborative Group . Randomised trial of intravenous atenolol among 16027 cases of suspected acute myocardial infarction (ISIS-1) . Lancet . 1986;2:57–66
  2. Gruppo Italiano per lo Studio della Streptokinasi nell’Infarto Miocardico (GISSI) . Effectiveness of intravenous thrombolytic treatment in acute myocardial infarction . Lancet . 1986;1:397–401
  3. Second International Study of Infarct Survival Collaborative Group . Randomised trial of intravenous streptokinase, oral aspirin, both, or neither among 17187 cases of suspected acute myocardial infarction (ISIS-2) . Lancet . 1988;2:349–360
  4. The GUSTO Angiographic Investigators . The effect of tissue plasminogen activator, streptokinase, or both on coronary-artery patency, ventricular function, and survival after acute myocardial infarction . N Engl J Med . 1993;329:1615–1622
  5. Cardiac Arrythmias Suppression Trial . Preliminary report (Effect of encainide and flecainide on mortality in a randomized trial of arrhythmia suppression after myocardial infarction) . N Engl J Med . 1989;321:406–412
  6. Marchioli R , Prieto JC , Tognoni G . Surrogate end-points (The case of trials on coronary atherosclerotic plaque regression) . Clin Trials Metaanalysis . 1994;29:139–176
  7. Tavazzi L , Maggioni AP , Tognoni G . Participation versus education (The GISSI story and beyond) . Am Heart J . 2004;148:222–229
  8. Marchioli R , Barbui T , Landolfi R , Tognoni G . Feasibility of randomised clinical trials in rare diseases (The case of polycythemia vera) . Leuk Lymphoma . 1996;22:S121–S127
  9. Berk PD , Goldberg JD , Donovan PB , Fruchtman SM , Berlin NI , Wasserman LR . Therapeutic recommendations in polycythemia vera based on polycythemia vera study group protocols . Semin Hematol . 1986;23:132–143
  10. Berk P , Goldberg J , Silverstein M , Weinfeld A , Donovan PB , Ellis JT , et al.   Increased incidence of acute leukemia in polycythemia vera associated with chlorambucil therapy . N Engl J Med . 1981;304:441–447
  11. Najean Y , Dresch C , Rain JD . The very-long-term course of polycythaemia (A complement to the previously published data of the Polycythaemia Vera Study Group) . Br J Haematol . 1994;86:233–235
  12. Spivak JL , Barosi G , Tognoni G , Barbui T , Finazzi G , Marchioli R , et al.   Chronic Myeloproliferative Disorders . Hematology (Am Soc Hematol Educ Program) . 2003;200–224
  13. Marchioli R , Finazzi G , Landolfi R , Kutti J , Gisslinger H , Patrono C , et al.   Vascular and neoplastic risk in a large cohort of patients with polycythemia vera . J Clin Oncol . 2005;23:1–9
  14. Landolfi R , Marchioli R , Kutti J , Gisslinger H , Tognoni G , Patrono C , et al.   Efficacy and safety of low-dose aspirin in polycythemia vera . N Engl J Med . 2004;350:114–124
  15. 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;105:2264–2270
  16. Gruppo Italiano Studio Policitemia Vera . Natural history of polycythemia vera (Clinical epidemiology of 1213 patients followed-up over 20 years) . Ann Intern Med . 1995;123:656–664
  17. Barbui T , Barosi G , Grossi A , Gugliotta L , Liberato LN , Marchetti M , et al.   Practice guidelines for the therapy of essential thrombocythemia. A statement from the Italian Society of Hematology, the Italian Society of Experimental Hematology and the Italian Group for Bone Marrow Transplantation . Haematologica . 2004;89:215–232
  18. Tefferi A , Fonseca R , Pereira D , Hoagland HC . A long-term retrospective study of young women with essential thrombocythemia . Mayo Clin Proc . 2001;76:22–28
  19. Cortelazzo S , Viero P , Finazzi G , D’Emilio A , Rodeghiero F , Barbui T . Incidence and risk factors for thrombotic complications in a historical cohort of 100 patients with essential thrombocythemia . J Clin Oncol . 1990;8:556–562
  20. Randi ML , Luzzatto G , Fabris F . Low-risk thrombocythemia in patients younger than 40 . Br J Haematol . 1999;104:928–936
  21. Besses C , Cervantes F , Pereira A , Florensa L , Sole F , Hernandez-Boluda JC , et al.   Major vascular complications in essential thrombocythemia (A study of the predictive factors in a series of 148 patients) . Leukemia . 1999;13:150–154
  22. Cortelazzo S , Finazzi G , Ruggeri M , Vestri O , Galli M , Rodeghiero F , et al.   Hydroxyurea for patients with essential thrombocythemia and a high risk of thrombosis . N Engl J Med . 1995;332:1132–1136
  23. Fleissz JL . In: Statistical Methods for Rates and Proportions . ed 2. New York, NY: Wiley; 1981;
  24. Armitage P , Berry G . In: Statistical Methods in Medical Research . ed 3. Cambridge, MA: Blackwell Scientific; 1994;
  25. Machin D , Campbell M , Fayers P , Pinol A . In: Sample Size Tables for Clinical Studies . ed 2. Oxford, UK: Blackwell Science; 1997;
  26. Landolfi R , Ciabattoni G , Patrignani P , Castellani MAL , Pogliani E , Bizzi B , et al.   Increased thromboxane byosynthesis in patients with polycythemia vera (Evidence for aspirin-suppressible platelet activation in vivo) . Blood . 1992;80:1965–1971
  27. Anagrelide Study Group . Anagrelide, a therapy for thrombocythemic states (Experience in 577 patients) . Am J Med . 1992;92:69–76
  28. Silver RT . Interferon-α2B (A new treatment for polycythemia vera) . Ann Intern Med . 1993;119:1091–1092
  29. Griesshammer M , Klippel S , Strunck E , Temerinac S , Mohr U , Heimpel H , et al.   PRV-1 mRNA expression discriminates two types of essential thrombocythemia . Ann Hematol . 2004;83:364–370
  30. Goerttler P , Doerner E , Kutti J , Moliterno AR , Spivak JL , Pahl HL . Quantification of c-Mpl protein expression and PRV-1 mRNA levels distinguish four distinct subpopulations of patients with ET . Blood . 2002;100:797a; (abstr)
  31. Moliterno AR , Spivak JL . Posttranslational processing of the thrombopoietin receptor is impaired in polycythemia vera . Blood . 1999;94:2555–2561
  32. Klippel S , Strunck E , Busse CE , Behringer D , Pahl HL . Biochemical characterization of PRV-1, a novel hematopoietic cell surface receptor, which is overexpressed in polycythemia rubra vera . Blood . 2002;100:2441
  33. Chiusolo P , La Barbera EO , Laurenti L , Piccirillo N , Sora F , Giordana G , et al.   Clonal hemapoiesis and risk of thrombosis in young female patients with essential thrombocythemia . Exp Hematol . 2001;29:670–676
  34. Wasserman LR , Berk PD , Berlin NI . Polycythemia Vera and the Myeloproliferative Disorders . Philadelphia, PA: Saunders; 1995;
  35. Gruppo Italiano Studio Policitemia Vera . Feasibility of a randomised clinical trial with low-dose aspirin in polycythemia vera . Br J Haematol . 1997;97:453–456

PII: S0037-1963(05)00081-8

doi: 10.1053/j.seminhematol.2005.05.021

Seminars in Hematology
Volume 42, Issue 4 , Pages 259-265 , October 2005

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