Practical management of patients with myelofibrosis receiving ruxolitinib

References

• Cervantes F, Dupriez B, Passamonti F et al. Improving survival trends in primary myelofibrosis: An international study. Haematologica 97(s1), 634 (2012).
   Google Scholar
• Cervantes F, Dupriez B, Pereira A et al. New prognostic scoring system for primary myelofibrosis based on a study of the international working group for myelofibrosis research and treatment. Blood 113(13), 2895–2901 (2009).
   PubMed Web of Science ®Google Scholar
• Vardiman JW, Thiele J, Arber DA et al. The 2008 revision of the world health organization (WHO) classification of myeloid neoplasms and acute leukemia: Rationale and important changes. Blood 114(5), 937–951 (2009).
   PubMed Web of Science ®Google Scholar
• Abdel-Wahab O, Pardanani A, Bernard OA et al. Unraveling the genetic underpinnings of myeloproliferative neoplasms and understanding their effect on disease course and response to therapy: proceedings from the 6th International Post-ASH Symposium. Am. J. Hematol. 87(5),562–568 (2012).
   PubMed Web of Science ®Google Scholar
• Abdel-Wahab OI, Levine RL. Primary myelofibrosis: update on definition, pathogenesis, and treatment. Annu. Rev. Med. 60, 233–245 (2009).
   PubMed Web of Science ®Google Scholar
• James C, Ugo V, Le Couedic JP et al. A unique clonal JAK2 mutation leading to constitutive signalling causes polycythaemia vera. Nature 434(7037), 1144–1148 (2005).
   PubMed Web of Science ®Google Scholar
• Levine RL, Wadleigh M, Cools J et al. Activating mutation in the tyrosine kinase JAK2 in polycythemia vera, essential thrombocythemia, and myeloid metaplasia with myelofibrosis. Cancer Cell 7(4), 387–397 (2005).
   PubMed Web of Science ®Google Scholar
• Baxter EJ, Scott LM, Campbell PJ et al. Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders. Lancet 365(9464), 1054–1061 (2005).
   PubMed Web of Science ®Google Scholar
• Kralovics R, Passamonti F, Buser AS et al. A gain-of-function mutation of JAK2 in myeloproliferative disorders. N. Engl. J. Med. 352(17), 1779–1790 (2005).
   PubMed Web of Science ®Google Scholar
• Tefferi A, Skoda R, Vardiman JW. Myeloproliferative neoplasms: contemporary diagnosis using histology and genetics. Nat. Rev. Clin. Oncol. 6(11), 627–637 (2009).
   PubMed Web of Science ®Google Scholar
• Scott LM, Tong W, Levine RL et al. JAK2 exon 12 mutations in polycythemia vera and idiopathic erythrocytosis. N. Engl. J. Med. 356(5), 459–468 (2007).
   PubMed Web of Science ®Google Scholar
• Oh ST, Simonds EF, Jones C et al. Novel mutations in the inhibitory adaptor protein LNK drive JAK-STAT signaling in patients with myeloproliferative neoplasms. Blood 116(6), 988–992 (2010).
   PubMed Web of Science ®Google Scholar
• Pikman Y, Lee BH, Mercher T et al. MPLW515L is a novel somatic activating mutation in myelofibrosis with myeloid metaplasia. PLoS Med. 3(7), e270 (2006).
   PubMed Web of Science ®Google Scholar
• Vainchenker W, Delhommeau F, Constantinescu SN, Bernard OA. New mutations and pathogenesis of myeloproliferative neoplasms. Blood 188(7), 1723–1735 (2011).
   Google Scholar
• Jakafi® (ruxolitinib), package insert. Incyte Corporation, DE, USA.
   Google Scholar
• Jakavi® (ruxolitinib), summary of product characteristics. Novartis Europharm Limited, Horsham, West Sussex, UK (2012).
   Google Scholar
• Jakavi® (ruxolitinib), package insert. Novartis Pharmaceuticals Canada Inc, Quebec, Canada.
   Google Scholar
• Harrison C, Kiladjian JJ, Al-Ali HK et al. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N. Engl. J. Med. 366(9), 787–798 (2012).
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa RA, Gotlib J et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N. Engl. J. Med. 366(9), 799–807 (2012).
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Kantarjian H, Mesa RA et al. Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N. Engl. J. Med. 363(12), 1117–1127 (2010).
   PubMed Web of Science ®Google Scholar
• Harrison C, Kiladijan JJ, Al-Ali HK et al. Health-related quality of life and symptoms in myelofibrosis patients treated with ruxolitinib versus best available therapy (abstract 795). Blood 118(21), 361–362 (2011).
   Web of Science ®Google Scholar
• Verstovsek S, Mesa R, Gotlib J et al. Long-term outcome of ruxolitinib treatment in patients with myelofibrosis: durable reductions in spleen volume, improvements in quality of life, and overall survival advantage in COMFORT-I. Blood 120(21), Abstract 800 (2012).
   Web of Science ®Google Scholar
• Cervantes F, Kiladjian J, Niederwieser D et al. Long-term efficacy, safety, and survival findings from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for the treatment of myelofibrosis. Blood 120(21), Abstract 801 (2012).
   Web of Science ®Google Scholar
• Passamonti F, Cervantes F, Vannucchi AM et al. A dynamic prognostic model to predict survival in primary myelofibrosis: a study by the IWG-MRT (International Working group for Myeloproliferative Neoplasms Research and Treatment). Blood 115(9), 1703–1708 (2010).
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa R, Gotlib J et al. Consistent benefit of ruxolitinib over placebo in spleen volume reduction and symptom improvement across subgroups and overall survival advantage: results from COMFORT-I (abstract 278). Blood 118(21), 129 (2011).
   PubMed Web of Science ®Google Scholar
• Harrison C, Kiladijan JJ, Gisslinger H et al. Ruxolitinib provides reductions in splenomegaly across subgroups: an analysis of spleen response in the COMFORT-II study (abstract 279). Blood 118(21), 129 (2011).
   PubMed Web of Science ®Google Scholar
• Mesa RA, Niblack J, Wadleigh M et al. The burden of fatigue and quality of life in myeloproliferative disorders (MPDs): an international internet-based survey of 1179 MPD patients. Cancer 109(1), 68–76 (2007).
   PubMed Web of Science ®Google Scholar
• Tefferi A, Lasho TL, Jimma T et al. One thousand patients with primary myelofibrosis: the Mayo Clinic experience. Mayo Clin. Proc. 87(1), 25–33 (2012).
   PubMed Web of Science ®Google Scholar
• Benjamini O, Jain P, Estrov Z, Kantarjian HM, Verstovsek S. Therapeutic effects of ruxolitinib in patients with myelofibrosis without clinically significant splenomegaly. Blood 120(13), 2768–2769 (2012).
   PubMed Web of Science ®Google Scholar
• Passamonti F, Cervantes F, Vannucchi AM et al. Dynamic International Prognostic Scoring System (DIPSS) predicts progression to acute myeloid leukemia in primary myelofibrosis. Blood 116(15), 2857–2858 (2010).
   PubMed Web of Science ®Google Scholar
• Tefferi A, Vaidya R, Caramazza D, Finke C, Lasho T, Pardanani A. Circulating interleukin (IL)-8, IL-2R, IL-12, and IL-15 levels are independently prognostic in primary myelofibrosis: a comprehensive cytokine profiling study. J. Clin. Oncol. 29(10), 1356–1363 (2011).
   PubMed Web of Science ®Google Scholar
• Verstovsek S. Therapeutic potential of janus-activated kinase-2 inhibitors for the management of myelofibrosis. Clin. Cancer Res. 16(7), 1988–1996 (2010).
   PubMed Web of Science ®Google Scholar
• Cervantes F. How I treat splenomegaly in myelofibrosis. Blood Cancer J. 1(10), e37 (2011).
   PubMed Web of Science ®Google Scholar
• Mesa RA. How I treat symptomatic splenomegaly in patients with myelofibrosis. Blood 113(22), 5394–5400 (2009).
   PubMed Web of Science ®Google Scholar
• Abu-Hilal M, Tawaker J. Portal hypertension secondary to myelofibrosis with myeloid metaplasia: a study of 13 cases. World J. Gastroenterol. 15(25), 3128–3133 (2009).
   PubMed Web of Science ®Google Scholar
• Koschmieder S, Koppelle A, Seifert H. Ruxolitinib for myelofibrosis. N. Engl. J. Med. 366(21), 2031–2032, author reply 2032–2034 (2012).
   PubMed Web of Science ®Google Scholar
• Eghtedar A, Verstovsek S, Estrov Z et al. Phase II study of the JAK kinase inhibitor ruxolitinib in patients with refractory leukemias, including post myeloproliferative neoplasms (MPN) acute myeloid leukemia (AML). Blood 119(20), 4614–4618 (2012).
   PubMed Web of Science ®Google Scholar
• Gangat N, Caramazza D, Vaidya R et al. DIPSS plus: a refined dynamic international prognostic scoring system for primary myelofibrosis that incorporates prognostic information from karyotype, platelet count, and transfusion status. J. Clin. Oncol. 29(4), 392–397 (2011).
   PubMed Web of Science ®Google Scholar
• McMullin MF, Harrison CN, Niederwieser D et al. The use of erythropoietic-stimulating agents (ESAs) with ruxolitinib in patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), and post-essential thrombocythemia myelofibrosis (PET-MF). Blood 120(21), Abstract 2838 (2012).
   PubMed Web of Science ®Google Scholar
• Reilly JT, McMullin MF, Beer PA et al. Guideline for the diagnosis and management of myelofibrosis. Br. J. Haematol. 158(4), 453–471 (2012).
   PubMed Web of Science ®Google Scholar
• Mesa RA, Barosi G, Cervantes F, Reilly JT, Tefferi A. Myelofibrosis with myeloid metaplasia: disease overview and non-transplant treatment options. Best Pract. Res. Clin. Haematol. 19(3), 495–517 (2006).
   PubMed Web of Science ®Google Scholar
• Reilly JT, Snowden JA, Spearing RL et al. Cytogenetic abnormalities and their prognostic significance in idiopathic myelofibrosis: a study of 106 cases. Br. J. Haematol. 98(1), 96–102 (1997).
   PubMed Web of Science ®Google Scholar
• Huang J, Li CY, Mesa RA et al. Risk factors for leukemic transformation in patients with primary myelofibrosis. Cancer 112(12), 2726–2732 (2008).
   PubMed Web of Science ®Google Scholar
• Talpaz M, Hamburg S, Jamieson K et al. Preliminary safety and efficacy from a phase II study of ruxolitinib in patients with primary and secondary myelofibrosis with platelet counts of 50–100 × 109/L (abstract 0597). Haematologica 97(s1), 245 (2012).
   Google Scholar
• Gisslinger H, McMullin MF, Jakel N et al. A phase 1b, open-label, dose-finding study of ruxolitinib in patients with myelofibrosis and baseline platelet (PLT) counts between 50 and <100 × 109/L (abstract 0369). Haematologica 97(s1), 149 (2012).
   Google Scholar
• Shilling AD, Nedza FM, Emm T et al. Metabolism, excretion and pharmacokinetics of 14C-INCB018424, a selective JAK1/2 inhibitor, in humans. Drug Metab. Dispos. 38(11), 2023–2031 (2010).
   PubMed Web of Science ®Google Scholar
• Shi JG, Chen X, McGee RF et al. The pharmacokinetics, pharmacodynamics, and safety of orally dosed INCB018424 phosphate in healthy volunteers. J. Clin. Pharmacol. 51(12), 1644–1654 (2011).
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Navada S, Malone A, Rodriguez A, Najfeld V, Hoffman R. Therapeutic options for patients with myelofibrosis in blast phase. Leuk. Res. 34(9), 1246–1249 (2010).
   PubMed Web of Science ®Google Scholar
• Mesa RA, Li CY, Ketterling RP, Schroeder GS, Knudson RA, Tefferi A. Leukemic transformation in myelofibrosis with myeloid metaplasia: a single-institution experience with 91 cases. Blood 105(3), 973–977 (2005).
   PubMed Web of Science ®Google Scholar
• Thepot S, Itzykson R, Seegers V et al. Treatment of progression of Philadelphia-negative myeloproliferative neoplasms to myelodysplastic syndrome or acute myeloid leukemia by azacitidine: a report on 54 cases on the behalf of the Groupe Francophone des Myelodysplasies (GFM). Blood 116(19), 3735–3742 (2010).
   PubMed Web of Science ®Google Scholar
• Quintas-Cardama A, Tong W, Kantarjian H et al. A phase II study of 5-azacitidine for patients with primary and post-essential thrombocythemia/polycythemia vera myelofibrosis. Leukemia 22(5), 965–970 (2008).
   PubMed Web of Science ®Google Scholar
• Tefferi A, Pardanani A. Serious adverse events during ruxolitinib treatment discontinuation in patients with myelofibrosis. Mayo Clin. Proc. 86(12), 1188–1191 (2011).
   PubMed Web of Science ®Google Scholar
• Haidar S, Ortiz-Neira C, Shroff M, Gilday D, Blaser S. Intracranial involvement in extramedullary hematopoiesis: case report and review of the literature. Pediatr. Radiol. 35(6), 630–634 (2005).
   PubMed Web of Science ®Google Scholar
• Lee CM, Salzman KL, Blumenthal DT, Gaffney DK. Intracranial extramedullary hematopoiesis: brief review of response to radiation therapy. Am. J. Hematol. 78(2), 151–152 (2005).
   PubMed Web of Science ®Google Scholar
• Zherebitskiy V, Morales C, Del Bigio MR. Extramedullary hematopoiesis involving the central nervous system and surrounding structures. Hum. Pathol. 42(10), 1524–1530 (2011).
   PubMed Web of Science ®Google Scholar
• Ayyildiz O, Isikdogan A, Celik M, Muftuoglu E. Intracranial meningeal extramedullary hematopoiesis inducing serious headache in a patient with idiopathic myelofibrosis. J. Pediatr. Hematol. Oncol. 26(1), 28–29 (2004).
   PubMed Web of Science ®Google Scholar