Comparison of placebo and best available therapy for the treatment of myelofibrosis in the phase 3 COMFORT studies

Haematologica

Volumn 99, Issue 2, 2014, Pages 291-298

  Mesa, Ruben A ^a   Kiladjian, Jean Jacques ^b   Verstovsek, Srdan ^c   Al Ali, Haifa Kathrin ^d   Gotlib, Jason ^e   Gisslinger, Heinz ^f   Levy, Richard ^g   Siulnik, Andres ^h   Gupta, Vikas ⁱ   Khan, Mahmudul ^h   DiPersio, John F ^j   McQuitty, Mari ^h   Catalano, John V ^k   Hunter, Deborah S ^g   Knoops, Laurent ^l   Deininger, Michael ^m   Cervantes, Francisco ⁿ   Miller, Carole ^o   Vannucchi, Alessandro M ^p   Silver, Richard T ^q   Barbui, Tiziano ^r   Talpaz, Moshe ^s   Barosi, Giovanni ^t   Winton, Elliott F ^u   Mendeson, Estella ^h   Harvey Jr , Jimmie H ^v   Arcasoy, Murat O ^w   Hexner, Elizabeth ^x   Lyons, Roger M ^y   Paquette, Ronald ^z   Raza, Azra ^aa   Sun, William ^g   Sandor, Victor ^g   Kantarjian, Hagop M ^c   Harrison, Claire ^ab   more..

^a MAYO CLINIC   (United States)

^b UNIVERSITÉ PARIS DESCARTES   (France)

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

^d UNIVERSITY OF LEIPZIG   (Germany)

^e STANFORD CANCER INSTITUTE   (United States)

^f MEDICAL UNIVERSITY OF VIENNA   (Austria)

^g Drug Metabolism Pharmacokinetics Clinical Pharmacology   (United States)

^h NOVARTIS PHARMA AG   (Switzerland)

ⁱ UNIVERSITY OF TORONTO   (Canada)

^j WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^k FRANKSTON HOSPITAL   (Australia)

^l CLINIQUES UNIVERSITAIRES SAINT LUC   (Belgium)

^m OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

ⁿ HOSPITAL CLÍNIC   (Spain)

^o St Agnes Cancer Institute   (United States)

^p UNIVERSITY OF FLORENCE   (Italy)

^q WEILL CORNELL MEDICAL COLLEGE   (United States)

^r OSPEDALI RIUNITI   (Italy)

^s UNIVERSITY OF MICHIGAN   (United States)

^t FONDAZIONE IRCCS POLICLINICO SAN MATTEO   (Italy)

^u EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^v Birmingham Hematology Oncology   (United States)

^w DUKE UNIVERSITY HEALTH SYSTEM   (United States)

^x UNIVERSITY OF PENNSYLVANIA   (United States)

^y Developmental Therapeutics Committee and Co Chair of the Genitourinary Committee   (United States)

^z UNIVERSITY OF CALIFORNIA   (United States)

^aa Department of Orthopaedic Surgery   (United States)

^ab GUY S HOSPITAL   (United Kingdom)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ANDROGEN; CHLORMETHINE DERIVATIVE; GLUCOCORTICOID; HYDROXYUREA; IMMUNOMODULATING AGENT; PLACEBO; PURINE DERIVATIVE; PYRIMIDINE DERIVATIVE; RECOMBINANT ERYTHROPOIETIN; RUXOLITINIB; PROTEIN KINASE INHIBITOR;

ADULT; AGED; ARTICLE; COMPUTER ASSISTED TOMOGRAPHY; CONTROLLED STUDY; DOUBLE BLIND PROCEDURE; FEMALE; HUMAN; INCIDENCE; MALE; MULTICENTER STUDY; MYELOFIBROSIS; NUCLEAR MAGNETIC RESONANCE IMAGING; OPEN STUDY; QUALITY OF LIFE; QUESTIONNAIRE; RANDOMIZED CONTROLLED TRIAL; THROMBOCYTE COUNT; VERY ELDERLY; COMPARATIVE STUDY; CONTROLLED CLINICAL TRIAL; MIDDLE AGED; MYELOID METAPLASIA; ORGAN SIZE; PATHOLOGY; PATHOPHYSIOLOGY; PHASE 3 CLINICAL TRIAL; SPLEEN; SPLENOMEGALY; TIME;

ADULT; AGED; AGED, 80 AND OVER; DOUBLE-BLIND METHOD; FEMALE; HUMANS; MALE; MIDDLE AGED; ORGAN SIZE; PRIMARY MYELOFIBROSIS; PROTEIN KINASE INHIBITORS; QUALITY OF LIFE; SPLEEN; SPLENOMEGALY; TIME FACTORS;

EID: 84896739915     PISSN: 03906078     EISSN: 15928721     Source Type: Journal    
DOI: 10.3324/haematol.2013.087650     Document Type: Article

References (22)

1. Mesa RA, Verstovsek S, Cervantes F, Barosi G, Reilly JT, Dupriez B, et al. Primary myelofibrosis (PMF), post polycythemia vera myelofibrosis (post-PV MF), post essential thrombocythemia myelofibrosis (post-ET MF), blast phase PMF (PMF-BP): consensus on terminology by the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT). Leuk Res. 2007;31(6):737-40.
2. Cervantes F, Passamonti F, Barosi G. Life expectancy and prognostic factors in the classic BCR/ABL-negative myeloproliferative disorders. Leukemia. 2008;22(5):905-14.
3. Mesa RA, Niblack J, Wadleigh M, Verstovsek S, Camoriano J, Barnes S, et al. The burden of fatigue and quality of life in myeloproliferative disorders (MPDs): an international Internet-based survey of 1179 MPD patients. Cancer. 2007;109(1):68-76.
4. Cervantes F, Dupriez B, Pereira A, Passamonti F, Reilly JT, Morra E, et al. New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. Blood. 2009;113(13):2895-901.
5. Ballen K. How to manage the transplant question in myelofibrosis. Blood Cancer J. 2012;2(3):e59.
6. James C, Ugo V, Le Couedic JP, Staerk J, Delhommeau F, Lacout C, et al. A unique clonal JAK2 mutation leading to constitutive signalling causes polycythaemia vera. Nature. 2005;434(7037):1144-8.
7. Baxter EJ, Scott LM, Campbell PJ, East C, Fourouclas N, Swanton S, et al. Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders. Lancet. 2005;365(9464):1054-61.
8. Kralovics R, Passamonti F, Buser AS, Teo SS, Tiedt R, Passweg JR, et al. A gain-offunction mutation of JAK2 in myeloproliferative disorders. N Engl J Med. 2005;352 (17):1779-90.
9. Levine RL, Wadleigh M, Cools J, Ebert BL, Wernig G, Huntly BJ, et al. Activating mutation in the tyrosine kinase JAK2 in polycythemia vera, essential thrombocythemia, and myeloid metaplasia with myelofibrosis. Cancer Cell. 2005;7(4):387-97.
10. Jakafi (ruxolitinib) [package insert]. Wilmington, DE: Incyte Corporation; 2011.
11. Novartis drug Jakavi first medication to receive European Commission approval to treat patients with myelofibrosis [Internet]. Switzerland: Novartis Pharmaceuticals. [updated 28 August 2012; cited 15 May 2013]. Available from: http://www.novartis.com/newsroom/media-releases/en/2012/1636508.shtml.
12. Jakavi (ruxolitinib) product monograph. Dorval, QC, Canada: Novartis Pharmaceuticals Canada Inc; 2012.
13. Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio J, et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med. 2012;366(9): 799-807.
14. Harrison C, Kiladjian JJ, Al-Ali HK, Gisslinger H, Waltzman R, Stalbovskaya V, et al. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med. 2012;366(9):787-98.
15. Osoba D, Rodrigues G, Myles J, Zee B, Pater J. Interpreting the significance of changes in health-related quality-of-life scores. J Clin Oncol. 1998;16(1):139-44.
16. Mesa RA, Shields A, Hare T, Erickson-Viitanen S, Sun W, Sarlis NJ, et al. Progressive burden of myelofibrosis in untreated patients: assessment of patientreported outcomes in patients randomized to placebo in the COMFORT-I study. Leuk Res 2013;37(8):911-6.
17. Heuck G. Zwei fälle von leukämie mit eigenthümlichem blut-resp knochenmarksbefund. Arch Pathol Antat Physiol Virchows. 1879;78:475-96.
18. Barbui T, Barosi G, Birgegard G, Cervantes F, Finazzi G, Griesshammer M, et al. Philadelphia-negative classical myeloproliferative neoplasms: critical concepts and management recommendations from European LeukemiaNet. J Clin Oncol. 2011;29(6):761-70.
19. Mesa RA, Pardanani AD, Hussein K, Wu W, Schwager S, Litzow MR, et al. Phase1/-2 study of pomalidomide in myelofibrosis. Am J Hematol. 2009;85(2):129-30.
20. Martinez-Trillos A, Gaya A, Maffioli M, Arellano-Rodrigo E, Calvo X, Diaz-Beya M, et al. Efficacy and tolerability of hydroxy-urea in the treatment of the hyperproliferative manifestations of myelofibrosis: results in 40 patients. Ann Hematol. 2010;89(12): 1233-7.
21. Besses C, Martínez-Sellés M. Anagrelide and cardiovascular events. Much ado about nothing? Leuk Res. 2011;35(12):1543-4.
22. Mesa RA, Green A, Barosi G, Verstovsek S, Vardiman J, Gale RP. MPN-associated myelofibrosis (MPN-MF). Leuk Res. 2011; 35(1):12-3.