Anagrelide compared with hydroxyurea in WHO-classified essential thrombocythemia: The ANAHYDRET Study, a randomized controlled trial

Blood

Volumn 121, Issue 10, 2013, Pages 1720-1728

  Gisslinger, Heinz ^a   Gotic, Mirjana ^b   Holowiecki, Jerzy ^c   Penka, Miroslav ^d   Thiele, Juergen ^e   Kvasnicka, Hans Michael ^f   Kralovics, Robert ^a,g   Petrides, Petro E ^h  

^a MEDICAL UNIVERSITY OF VIENNA   (Austria)

^b CLINICAL CENTER OF SERBIA   (Serbia)

^c MEDICAL UNIVERSITY OF SILESIA   (Poland)

^d UNIVERSITY HOSPITAL BRNO   (Czech Republic)

^e UNIVERSITY HOSPITAL OF COLOGNE   (Germany)

^f GOETHE UNIVERSITY   (Germany)

^g ERICH SCHMID INSTITUTE OF MATERIALS SCIENCE   (Austria)

^h UNIVERSITY OF MUNICH   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

ACETYLSALICYLIC ACID; ANAGRELIDE; HEMOGLOBIN; HYDROXYUREA; JANUS KINASE 2; LACTATE DEHYDROGENASE;

ABDOMINAL PAIN; ADULT; AGED; ALOPECIA; ANEMIA; ARTERY THROMBOSIS; ARTICLE; BLEEDING; BRONCHITIS; CLINICAL OBSERVATION; COHORT ANALYSIS; CONTROLLED STUDY; DIARRHEA; DISEASE CLASSIFICATION; DIZZINESS; DRUG DOSE INCREASE; DRUG DOSE REDUCTION; DRUG EFFICACY; DRUG INDUCED HEADACHE; DRUG TOLERABILITY; DRUG WITHDRAWAL; EPISTAXIS; FEMALE; FLU LIKE SYNDROME; GASTROINTESTINAL SYMPTOM; HEART PALPITATION; HERPES LABIALIS; HERPES SIMPLEX; HERPES ZOSTER; HUMAN; HYPERTENSION; LEUKOCYTE COUNT; LEUKOPENIA; MAJOR CLINICAL STUDY; MALE; MYELOFIBROSIS; OUTCOME ASSESSMENT; PRIORITY JOURNAL; PROSPECTIVE STUDY; RANDOMIZED CONTROLLED TRIAL; SIDE EFFECT; SINGLE BLIND PROCEDURE; SKIN DISEASE; SPLENOMEGALY; TACHYCARDIA; THROMBOCYTE COUNT; THROMBOCYTHEMIA; VEIN THROMBOSIS; VERTIGO; VIRUS INFECTION;

EID: 84876515240     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2012-07-443770     Document Type: Article

References (49)

1. Fenaux P, Simon M, Caulier MT, et al. Clinical course of essential thrombocythemia in 147 cases. Cancer. 1990;66(3): 549-556. (Pubitemid 20233455)
2. Cortelazzo S, Viero P, Finazzi G, et al. Incidence and risk factors for thrombotic complications in a historical cohort of 100 patients with essential thrombocythemia. J Clin Oncol. 1990;8(3): 556-562. (Pubitemid 20087557)
3. Passamonti F, Rumi E, Arcaini L, et al. Prognostic factors for thrombosis, myelofibrosis, and leukemia in essential thrombocythemia: a study of 605 patients. Haematologica. 2008;93(11): 1645-1651.
4. Besses C, Cervantes F, Pereira A, et al. Major vascular complications in essential thrombocythemia: a study of the predictive factors in a series of 148 patients. Leukemia. 1999;13(2): 150-154. (Pubitemid 29092645)
5. Barbui T, Barosi G, Birgegard G, et al. European LeukemiaNet. Philadelphia-negative classical myeloproliferative neoplasms: critical concepts and management recommendations from European LeukemiaNet. J Clin Oncol. 2011;29(6): 761-770.
6. Cortelazzo S, Finazzi G, Ruggeri M, et al. Hydroxyurea for patients with essential thrombocythemia and a high risk of thrombosis. N Engl J Med. 1995;332(17):1132-1136.
7. Tefferi A, Gangat N, Wolanskyj AP. Management of extreme thrombocytosis in otherwise low-risk essential thrombocythemia; does number matter? Blood. 2006;108(7):2493-2494. (Pubitemid 44497538)
8. Ruggeri M, Finazzi G, Tosetto A, et al. No treatment for low-risk thrombocythaemia: results from a prospective study. Br J Haematol. 1998; 103(3):772-777. (Pubitemid 28545314)
9. Millard FE, Hunter CS, Anderson M, et al. Clinical manifestations of essential thrombocythemia in young adults. Am J Hematol. 1990;33(1):27-31. (Pubitemid 20031289)
10. Johnson M, Gernsheimer T, Johansen K. Essential thrombocytosis: underemphasized cause of large-vessel thrombosis. J Vasc Surg. 1995;22(4):443-447, discussion 448-449.
11. Tefferi A, Gangat N, Wolanskyj A. The interaction between leukocytosis and other risk factors for thrombosis in essential thrombocythemia. Blood. 2007;109(9):4105. (Pubitemid 46641767)
12. Carobbio A, Finazzi G, Guerini V, et al. Leukocytosis is a risk factor for thrombosis in essential thrombocythemia: interaction with treatment, standard risk factors, and Jak2 mutation status. Blood. 2007;109(6):2310-2313. (Pubitemid 46425868)
13. Harrison CN, Campbell PJ, Buck G, et al. United Kingdom Medical Research Council Primary Thrombocythemia 1 Study. Hydroxyurea compared with anagrelide in high-risk essential thrombocythemia. N Engl J Med. 2005;353(1):33-45. (Pubitemid 41007805)
14. Murphy S. Diagnostic criteria and prognosis in polycythemia vera and essential thrombocythemia. Semin Hematol. 1999; 36(1 Suppl 2):9-13. (Pubitemid 29045497)
15. Campbell PJ, Bareford D, Erber WN, et al. Reticulin accumulation in essential thrombocythemia: prognostic significance and relationship to therapy. J Clin Oncol. 2009;27(18): 2991-2999.
16. Imbert M, Pierre R, Thiele J, et al. Essential thrombocythaemia. In: Jaffe ES, Harris NL, Stein H, et al, eds. Pathology and Genetics: Tumours of haematopoietic and lymphoid tissues. World Health Organization. Classification of Tumours. Lyon, France: IARC Press; 2001:39-41.
17. Thiele J, Kvasnicka HM, Orazi A, et al. Essential thrombocythaemia. In: Swerdlow SH, Campo E, Lee Harris N, et al, eds. WHO classification of tumours of haematopoietic and lymphoid tissues. 4th ed. Lyon, France: International Agency for Research on Cancer; 2008:48-50.
18. Thiele J, Kvasnicka HM. Clinicopathological criteria for differential diagnosis of thrombocythemias in various myeloproliferative disorders. Semin Thromb Hemost. 2006;32(3): 219-230.
19. Carobbio A, Antonioli E, Guglielmelli P, et al. Leukocytosis and risk stratification assessment in essential thrombocythemia. J Clin Oncol. 2008; 26(16):2732-2736.
20. Carobbio A, Thiele J, Passamonti F, et al. Risk factors for arterial and venous thrombosis in WHO-defined essential thrombocythemia: an international study of 891 patients. Blood. 2011; 117(22):5857-5859.
21. Steurer M, Gastl G, Jedrzejczak WW, et al. Anagrelide for thrombocytosis in myeloproliferative disorders: a prospective study to assess efficacy and adverse event profile. Cancer. 2004;101(10):2239-2246. (Pubitemid 39458679)
22. Kralovics R, Teo SS, Li S, et al. Acquisition of the V617F mutation of JAK2 is a late genetic event in a subset of patients with myeloproliferative disorders. Blood. 2006;108(4):1377-1380. (Pubitemid 44232040)
23. Petrides PE, Gisslinger H, Steurer M, et al. Pharmacokinetics, bioequivalence, tolerability, and effects on platelet counts of two formulations of anagrelide in healthy volunteers and patients with thrombocythemia associated with chronic myeloproliferation. Clin Ther. 2009;31(2): 386-398.
24. Jabaily J, Iland HJ, Laszlo J, et al. Neurologic manifestations of essential thrombocythemia. Ann Intern Med. 1983;99(4):513-518. (Pubitemid 13009218)
25. Okazaki H, Doi T, Izumikawa M, et al. Pulsatile tinnitus as a first symptom of essential thrombocythemia. Am J Otolaryngol. 2011;32(3): 263-264.
26. Barosi G, Mesa RA, Thiele J, et al. International Working Group for Myelofibrosis Research and Treatment (IWG-MRT). Proposed criteria for the diagnosis of post-polycythemia vera and postessential thrombocythemia myelofibrosis: a consensus statement from the International Working Group for Myelofibrosis Research and Treatment. Leukemia. 2008;22(2):437-438. (Pubitemid 351250553)
27. Maurer W, Hothorn LA, Lehmacher W. Multiple comparisons in drug clinical trials and preclinical assays: a-priori ordered hypotheses. In: Testing Principles in Clinical and Preclinical Trials, Hrsg. Von J. Vollmar, Biometrie in der chemischpharmazeutischen Industrie 6, Gustav Fischer, Stuttgart 1995, p. 3-18.
28. Su JQ, Wei LJ. Nonparametric estimation for the difference or ratio of median failure times. Biometrics. 1993;49(2):603-607. (Pubitemid 23217995)
29. Bauer P, Kieser M. Combining different phases in the development of medical treatments within a single trial. Stat Med. 1999;18(14):1833-1848. (Pubitemid 29364054)
30. Mantel N, Haenszel W. Statistical aspects of the analysis of data from retrospective studies of disease. J Natl Cancer Inst. 1959;22(4):719-748.
31. Koch GG, Edwards S. Clinical efficacy trials with categorical data. In: Peace KE, ed. Biopharmaceutical Statistics for Drug Development. New York: Marcel Dekker, Inc.; 1988:403-457.
32. Bauer P, Kǒhne K. Evaluation of experiments with adaptive interim analyses. Biometrics. 1994; 50(4):1029-1041.
33. Thiele J, Kvasnicka HM, Facchetti F, et al. European consensus on grading bone marrow fibrosis and assessment of cellularity. Haematologica. 2005;90(8):1128-1132. (Pubitemid 41323513)
34. 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. 2012; (Sept 14 Epub ahead of print)
35. Gugliotta L, Tieghi A, Tortorella G, et al. Low impact of cardiovascular adverse events on anagrelide treatment discontinuation in a cohort of 232 patients with essential thrombocythemia. Leuk Res. 2011;35(12):1557-1563.
36. Barbui T, Carobbio A, Rambaldi A, et al. Perspectives on thrombosis in essential thrombocythemia and polycythemia vera: is leukocytosis a causative factor? Blood. 2009; 114(4):759-763.
37. Campbell PJ, MacLean C, Beer PA, et al. Correlation of blood counts with vascular complications in essential thrombocythemia: analysis of the prospective PT1 cohort. Blood. 2012;120(7):1409-1411.
38. Buxhofer-Ausch V, Gisslinger H, Thiele J, et al. Leukocytosis as an important risk factor for arterial thrombosis in WHO-defined early/prefibrotic myelofibrosis: an international study of 264 patients. Am J Hematol. 2012;87(7):669-672.
39. Thiele J, Kvasnicka HM, Schmitt-Graeff A, et al. Follow-up examinations including sequential bone marrow biopsies in essential thrombocythemia (ET): a retrospective clinicopathological study of 120 patients. Am J Hematol. 2002;70(4):283-291. (Pubitemid 34810505)
40. Kvasnicka HM, Thiele J. The impact of clinicopathological studies on staging and survival in essential thrombocythemia, chronic idiopathic myelofibrosis, and polycythemia rubra vera. Semin Thromb Hemost. 2006;32(4 Pt 2):362-371.
41. 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):3179-3184.
42. Finazzi G, Carobbio A, Thiele J, et al. Incidence and risk factors for bleeding in 1104 patients with essential thrombocythemia or prefibrotic myelofibrosis diagnosed according to the 2008 WHO criteria. Leukemia. 2012;26(4):716-719.
43. Cortelazzo S, Marchetti M, Orlando E, et al. Aspirin increases the bleeding side effects in essential thrombocythemia independent of the cyclooxygenase pathway: role of the lipoxygenase pathway. Am J Hematol. 1998; 57(4):277-282. (Pubitemid 28158413)
44. Hussein K, Bock O, Theophile K, et al. JAK2(V617F) allele burden discriminates essential thrombocythemia from a subset of prefibrotic-stage primary myelofibrosis. Exp Hematol. 2009;37(10):1186-1193, e7.
45. Wilkins BS, Erber WN, Bareford D, et al. Bone marrow pathology in essential thrombocythemia: interobserver reliability and utility for identifying disease subtypes. Blood. 2008;111(1):60-70.
46. Florena AM, Tripodo C, Iannitto E, et al. Value of bone marrow biopsy in the diagnosis of essential thrombocythemia. Haematologica. 2004;89(8): 911-919.
47. Gianelli U, Vener C, Raviele PR, et al. Essential thrombocythemia or chronic idiopathic myelofibrosis? A single-center study based on hematopoietic bone marrow histology. Leuk Lymphoma. 2006;47(9):1774-1781. (Pubitemid 44649759)
48. Kreft A, Bǔche G, Ghalibafian M, et al. The incidence of myelofibrosis in essential thrombocythaemia, polycythaemia vera and chronic idiopathic myelofibrosis: a retrospective evaluation of sequential bone marrow biopsies. Acta Haematol. 2005;113(2):137-143. (Pubitemid 40522818)
49. Boiocchi L, Vener C, Savi F, et al. Increased expression of vascular endothelial growth factor receptor 1 correlates with VEGF and microvessel density in Philadelphia chromosome-negative myeloproliferative neoplasms. J Clin Pathol. 2011; 64(3):226-231.