Ruxolitinib versus standard therapy for the treatment of polycythemia vera

New England Journal of Medicine

Volumn 372, Issue 5, 2015, Pages 426-435

  Vannucchi, Alessandro M ^a   Kiladjian, Jean Jacques ^d   Griesshammer, Martin ^e   Masszi, Tamas ^f   Durrant, Simon ^g   Passamonti, Francesco ^b   Harrison, Claire N ^h   Pane, Fabrizio ^c   Zachee, Pierre ⁱ   Mesa, Ruben ^j   He, Shui ^k   Jones, Mark M ^k   Garrett, William ^k   Li, Jingjin ^l   Pirron, Ulrich ^l   Habr, Dany ^l   Verstovsek, Srdan ^m  

^a CAREGGI UNIVERSITY HOSPITAL   (Italy)

^b OSPEDALE DI CIRCOLO   (Italy)

^c UNIVERSITY OF NAPLES FEDERICO II   (Italy)

^d UNIVERSITÉ PARIS DESCARTES   (France)

^e KLINIKUM MINDEN   (Germany)

^f SEMMELWEIS UNIVERSITY   (Hungary)

^g ROYAL BRISBANE AND WOMEN'S HOSPITAL   (Australia)

^h GUY'S AND ST THOMAS' NHS FOUNDATION TRUST   (United Kingdom)

ⁱ ZiekenhuisNetwerk Antwerpen   (Belgium)

^j MAYO CLINIC   (United States)

^k Incyte ^*  (United States)

^l NOVARTIS PHARMA AG   (Switzerland)

^m UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE AMINOTRANSFERASE; ANAGRELIDE; ASPARTATE AMINOTRANSFERASE; HYDROXYUREA; JANUS KINASE 2; LENALIDOMIDE; PEGINTERFERON; PIPOBROMAN; RUXOLITINIB; THALIDOMIDE; ANTINEOPLASTIC AGENT; JANUS KINASE; PYRAZOLE DERIVATIVE;

ABDOMINAL PAIN; ADULT; AGED; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ANEMIA; ARTICLE; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; ASTHENIA; CONTROLLED STUDY; CROSSOVER PROCEDURE; DIARRHEA; DIZZINESS; DRUG DOSE INCREASE; DRUG EFFICACY; DRUG SAFETY; DRUG WITHDRAWAL; DYSPNEA; FATIGUE; FEMALE; HEADACHE; HEMATOCRIT; HERPES ZOSTER; HUMAN; HYPERCHOLESTEROLEMIA; HYPERTRANSAMINASEMIA; HYPERTRIGLYCERIDEMIA; HYPERURICEMIA; JAK2 GENE; LEUKOCYTE COUNT; LEUKOPENIA; LYMPHOCYTOPENIA; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; MUSCLE SPASM; MYELOFIBROSIS; NEUTROPENIA; NON MELANOMA SKIN CANCER; PHASE 3 CLINICAL TRIAL; PHLEBOTOMY; POLYCYTHEMIA VERA; PRIORITY JOURNAL; PRURITUS; RANDOMIZED CONTROLLED TRIAL; SPLEEN SIZE; THROMBOCYTE COUNT; THROMBOCYTOPENIA; TREATMENT RESPONSE; VERY ELDERLY; ANTAGONISTS AND INHIBITORS; BLOOD CELL COUNT; CHEMICALLY INDUCED; CLINICAL TRIAL; COMPARATIVE STUDY; DRUG EFFECTS; KAPLAN MEIER METHOD; MIDDLE AGED; ORGAN SIZE; PATHOLOGY; REMISSION; SPLEEN;

ADULT; AGED; AGED, 80 AND OVER; ANTINEOPLASTIC AGENTS; BLOOD CELL COUNT; FEMALE; HUMANS; HYDROXYUREA; JANUS KINASES; KAPLAN-MEIER ESTIMATE; MALE; MIDDLE AGED; ORGAN SIZE; POLYCYTHEMIA VERA; PYRAZOLES; REMISSION INDUCTION; SPLEEN; THROMBOCYTOPENIA;

EID: 84921891315     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1409002     Document Type: Article

References (31)

1. Spivak JL. Polycythemia vera: myths, mechanisms, and management. Blood 2002; 100: 4272-90.
2. Tefferi A. Polycythemia vera and essential thrombocythemia: 2013 update on diagnosis, risk-stratification, and management. Am J Hematol 2013; 88: 507-16.
3. Geyer HL, Scherber RM, Dueck AC, et al. Distinct clustering of symptomatic burden among myeloproliferative neoplasm patients: retrospective assessment in 1470 patients. Blood 2014; 123: 3803-10.
4. Stuart BJ, Viera AJ. Polycythemia vera. Am Fam Physician 2004; 69: 2139-44.
5. Tefferi A, Rumi E, Finazzi G, et al. Survival and prognosis among 1545 patients with contemporary polycythemia vera: an international study. Leukemia 2013; 27: 1874-81.
6. 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: 761-70.
7. Landolfi R, Marchioli R, Kutti J, et al. Efficacy and safety of low-dose aspirin in polycythemia vera. N Engl J Med 2004; 350: 114-24.
8. Marchioli R, Finazzi G, Specchia G, et al. Cardiovascular events and intensity of treatment in polycythemia vera. N Engl J Med 2013; 368: 22-33.
9. Prchal JT, Gordeuk VR. Treatment target in polycythemia vera. N Engl J Med 2013; 368: 1555-6.
10. Kim J, Wessling-Resnick M. Iron and mechanisms of emotional behavior. J Nutr Biochem 2014; 25: 1101-7.
11. Tobiasson M, Alyass B, S.derlund S, Birgeg.rd G. High prevalence of restless legs syndrome among patients with polycytemia vera treated with venesectio. Med Oncol 2010; 27: 105-7.
12. 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: 961-3.
13. 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: 1363-9.
14. Verstovsek S, Passamonti F, Rambaldi A, et al. A phase 2 study of ruxolitinib, an oral JAK1 and JAK2 Inhibitor, in patients with advanced polycythemia vera who are refractory or intolerant to hydroxyurea. Cancer 2014; 120: 513-20.
15. Tibes R, Mesa RA. Emerging drugs for polycythemia vera. Expert Opin Emerg Drugs 2013; 18: 393-404.
16. Sever M, Newberry KJ, Verstovsek S. Therapeutic options for patients with polycythemia vera and essential thrombocythemia refractory/resistant to hydroxyurea. Leuk Lymphoma 2014.
17. Abelsson J, Andréasson B, Samuelsson J, et al. Patients with polycythemia vera have worst impairment of quality of life among patients with newly diagnosed myeloproliferative neoplasms. Leuk Lymphoma 2013; 54: 2226-30.
18. Vannucchi AM, Kiladjian JJ, Griesshammer M, et al. Baseline characteristics and symptom burden in RESPONSE: a randomized, open-label, phase 3 study of ruxolitinib in polycythemia vera patients resistant to or intolerant of hydroxyurea. Blood 2013; 122: 4071. abstract.
19. Stauffer Larsen T, Iversen KF, Hansen E, 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: 1041-5.
20. 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: 5418-24.
21. 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: 3065-72.
22. Kiladjian JJ, Mesa RA, Hoffman R. The renaissance of interferon therapy for the treatment of myeloid malignancies. Blood 2011; 117: 4706-15.
23. Kiladjian JJ, Chomienne C, Fenaux P. Interferon-alpha therapy in bcr-abl-negative myeloproliferative neoplasms. Leukemia 2008; 22: 1990-8.
24. Nordic MPN Study Group. Nordic guidelines on the diagnosis and treatment of patients with myeloproliferative neoplasms, 2013 (http://nmpn.org/index.php/guidelines/1-nmpn-guidelines-2013/file).
25. Bj.rkholm M, Derolf AR, 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: 2410-5.
26. 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; 29: 3907-13.
27. Barbui T, Carobbio A, Rambaldi A, Finazzi G. Perspectives on thrombosis in essential thrombocythemia and polycythemia vera: is leukocytosis a causative factor? Blood 2009; 114: 759-63.
28. Landolfi R, Di Gennaro L, Barbui T, et al. Leukocytosis as a major thrombotic risk factor in patients with polycythemia vera. Blood 2007; 109: 2446-52.
29. Barbui T, Carobbio A, Finazzi G, et al. Inflammation and thrombosis in essential thrombocythemia and polycythemia vera: different role of C-reactive protein and pentraxin 3. Haematologica 2011; 96: 315-8.
30. Finazzi G, Caruso V, Marchioli R, et al. Acute leukemia in polycythemia vera: an analysis of 1638 patients enrolled in a prospective observational study. Blood 2005; 105: 2664-70.
31. 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: 755-61.