Myelofibrosis patients in Belgium: disease characteristics

References

• Vardiman JW, Thiele J, Arber DA, Brunning RD, Borowitz MJ, Porwit A, et al. The 2008 revision of the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia: rationale and important changes. Blood. 2009;114(5):937–51.
   PubMed Web of Science ®Google Scholar
• 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.
   Web of Science ®Google Scholar
• Tefferi A. Myelofibrosis with myeloid metaplasia. N Engl J Med. 2000;342(17):1255–65.
   Web of Science ®Google Scholar
• 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.
   Web of Science ®Google Scholar
• Maynadie M, Girodon F, Manivet-Janoray I, Mounier M, Mugneret F, Bailly F, et al. Twenty-five years of epidemiological recording on myeloid malignancies: data from the specialized registry of hematologic malignancies of Cote d'Or (Burgundy, France). Haematologica. 2011;96(1):55–61.
   Web of Science ®Google Scholar
• 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.
   Web of Science ®Google Scholar
• Johansson P, Kutti J, Andreasson B, Safai-Kutti S, Vilen L, Wedel H, et al. Trends in the incidence of chronic Philadelphia chromosome negative (Ph-) myeloproliferative disorders in the city of Goteborg, Sweden, during 1983–99. J Intern Med. 2004;256(2):161–5.
   Web of Science ®Google Scholar
• Osca-Gelis G, Puig-Vives M, Saez M, Gallardo D, Lloveras N, Marcos-Gragera R. Population-based incidence of myeloid malignancies: fifteen years of epidemiological data in the province of Girona, Spain. Haematologica. 2013;98(8):e95–7.
   Google Scholar
• Phekoo KJ, Richards MA, Moller H, Schey SA. The incidence and outcome of myeloid malignancies in 2,112 adult patients in southeast England. Haematologica. 2006;91(10):1400–4.
   Web of Science ®Google Scholar
• Visser O, Trama A, Maynadie M, Stiller C, Marcos-Gragera R, De Angelis R, et al. Incidence, survival and prevalence of myeloid malignancies in Europe. Eur J Cancer. 2012;48(17):3257–66.
   Web of Science ®Google Scholar
• Passamonti F, Cervantes F, Vannucchi AM, Morra E, Rumi E, Pereira A, 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. 2010;115(9):1703–8.
   Web of Science ®Google Scholar
• Gangat N, Caramazza D, Vaidya R, George G, Begna K, Schwager S, 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. 2011;29(4):392–7.
   Web of Science ®Google Scholar
• Deeg HJ, Gooley TA, Flowers ME, Sale GE, Slattery JT, Anasetti C, et al. Allogeneic hematopoietic stem cell transplantation for myelofibrosis. Blood. 2003;102(12):3912–8.
   Web of Science ®Google Scholar
• Ditschkowski M, Beelen DW, Trenschel R, Koldehoff M, Elmaagacli AH. Outcome of allogeneic stem cell transplantation in patients with myelofibrosis. Bone Marrow Transplant. 2004;34(9):807–13.
   Web of Science ®Google Scholar
• Kroger N, Holler E, Kobbe G, Bornhauser M, Schwerdtfeger R, Baurmann H, et al. Allogeneic stem cell transplantation after reduced-intensity conditioning in patients with myelofibrosis: a prospective, multicenter study of the Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation. Blood. 2009;114(26):5264–70.
   Web of Science ®Google Scholar
• Ballen KK, Shrestha S, Sobocinski KA, Zhang MJ, Bashey A, Bolwell BJ, et al. Outcome of transplantation for myelofibrosis. Biol Blood Marrow Transplant. 2010;16(3):358–67.
   Web of Science ®Google Scholar
• Abdel-Wahab OI, Levine RL. Primary myelofibrosis: update on definition, pathogenesis, and treatment. Annu Rev Med. 2009;60:233–45.
   PubMed Web of Science ®Google Scholar
• Tefferi A. How I treat myelofibrosis. Blood. 2011;117(13):3494–504.
   Web of Science ®Google Scholar
• Mesa RA, Nagorney DS, Schwager S, Allred J, Tefferi A. Palliative goals, patient selection, and perioperative platelet management: outcomes and lessons from 3 decades of splenectomy for myelofibrosis with myeloid metaplasia at the Mayo Clinic. Cancer. 2006;107(2):361–70.
   Web of Science ®Google Scholar
• Mesa RA. How I treat symptomatic splenomegaly in patients with myelofibrosis. Blood. 2009;113(22):5394–400.
   Web of Science ®Google Scholar
• Baxter EJ, Scott LM, Campbell PJ, East C, Fourouclas N, et al. Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders. Lancet. 2005;365(9464):1054–61.
   PubMed Web of Science ®Google Scholar
• 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.
   Web of Science ®Google Scholar
• Kralovics R, Passamonti F, Buser AS, Teo SS, Tiedt R, Passweg JR, et al. A gain-of-function mutation of JAK2 in myeloproliferative disorders. N Engl J Med. 2005;352(17):1779–90.
   Web of Science ®Google Scholar
• 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.
   Web of Science ®Google Scholar
• Tam CS, Verstovsek S. Investigational Janus kinase inhibitors. Expert Opin Investig Drugs. 2013;22(6):687–99.
   Web of Science ®Google Scholar
• Sonbol MB, Firwana B, Zarzour A, Morad M, Rana V, Tiu RV. Comprehensive review of JAK inhibitors in myeloproliferative neoplasms. Ther Adv Hematol. 2013;4(1):15–35.
   Google Scholar
• Komrokji R, Verstovsek S. Assessing efficacy in myelofibrosis treatment: a focus on JAK inhibition. Expert Rev Hematol. 2012;5(6):631–41.
   Web of Science ®Google Scholar
• 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.
   Web of Science ®Google Scholar
• Reilly JT, McMullin MF, Beer PA, Butt N, Conneally E, Duncombe A, et al. Writing group: British Committee for Standards in H. Guideline for the diagnosis and management of myelofibrosis. Br J Haematol. 2012;158(4):453–71.
   Web of Science ®Google Scholar
• Barosi G, Lupo L, Rosti V. Management of myeloproliferative neoplasms: from academic guidelines to clinical practice. Curr Hematol Malig Rep. 2012;7(1):50–6.
   Google Scholar
• Tefferi A. Primary myelofibrosis: 2013 update on diagnosis, risk-stratification, and management. Am J Hematol. 2013;88(2):141–50.
   Web of Science ®Google Scholar
• Mascarenhas J, Hoffman R. A comprehensive review and analysis of the effect of ruxolitinib therapy on the survival of patients with myelofibrosis. Blood. 2013;121(24):4832–7.
   Web of Science ®Google Scholar
• 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.
   Web of Science ®Google Scholar
• Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med. 2012:366(9):799–807.
   Web of Science ®Google Scholar
• Mascarenhas J, Hoffman R. Ruxolitinib: the first FDA approved therapy for the treatment of myelofibrosis. Clin Cancer Res. 2012;18(11):3008–14.
   Web of Science ®Google Scholar
• Pardanani A, Laborde RR, Lasho TL, Finke C, Begna K, Al-Kali A, et al. Safety and efficacy of CYT387, a JAK1 and JAK2 inhibitor, in myelofibrosis. Leukemia. 2013;27(6):1322–7.
   PubMed Web of Science ®Google Scholar
• Orphanet Report Series. Prevalence of rare diseases: Bibliographic data 2011. Available from: http://www.orpha.net (accessed 17 March 2014).
   Google Scholar
• Statistical Overview of Belgium. Key figures 2012. Available from: http://statbel.fgov.be (accessed 17 March 2014).
   Google Scholar
• Komrokji RS, Wadleigh M, Seymour JF, Roberts AW, Bik L, Zhu HJ, et al. Results of a Phase 2 Study of Pacritinib (SB1518), a Novel Oral JAK2 Inhibitor, in Patients with Primary, Post-Polycythemia Vera, and Post-Essential Thrombocythemia Myelofibrosis. Paper presented at the ASH Annl Meet; 2011 Dec 10–13; San Diego, CA, USA.
   Google Scholar
• Santos FP, Verstovsek S. What is next beyond janus kinase 2 inhibitors for primary myelofibrosis? Curr Opin Hematol. 2013;20(2):123–9.
   Web of Science ®Google Scholar
• Verstovsek S, Kantarjian HM, Estrov Z, Cortes JE, Thomas DA, Kadia T, et al. Long-term outcomes of 107 patients with myelofibrosis receiving JAK1/JAK2 inhibitor ruxolitinib: survival advantage in comparison to matched historical controls. Blood. 2012;120(6):1202–9.
   Web of Science ®Google Scholar
• Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, Dipersio JF, et al. Efficacy, safety and survival with ruxolitinib in patients with myelofibrosis: results of a median 2-year follow-up of COMFORT-I. Haematologica. 2013;98(12):1865–71.
   Google Scholar
• Cervantes F, Vannucchi AM, Kiladjian JJ, Al-Ali HK, Sirulnik A, Stalbovskaya V, et al. Three-year efficacy, safety, and survival findings from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for myelofibrosis. Blood. 2013;122(25):4047–53.
   Web of Science ®Google Scholar
• Talpaz M, Paquette R, Afrin L, Hamburg SI, Prchal JT, Jamieson K, et al. Interim analysis of safety and efficacy of ruxolitinib in patients with myelofibrosis and low platelet counts. J Hematol Oncol. 2013;6(1):81.
   Google Scholar
• Pardanani A, Gotlib JR, Jamieson C, Cortes JE, Talpaz M, Stone RM, et al. Safety and efficacy of TG101348, a selective JAK2 inhibitor, in myelofibrosis. J Clin Oncol. 2011;29(7):789–796.
   Web of Science ®Google Scholar
• Tefferi A, Verstovsek S, Barosi G, Passamonti F, Roboz GJ, Gisslinger H, et al. Pomalidomide is active in the treatment of anemia associated with myelofibrosis. J Clin Oncol. 2009;27(27):4563–4569.
   Web of Science ®Google Scholar
• Tefferi A, Passamonti F, Barbui T, Barosi G, Begna K, Cazzola M, et al. Phase 3 Study Of Pomalidomide In Myeloproliferative Neoplasm (MPN)-Associated Myelofibrosis With RBC-Transfusion-Dependence. Paper presented at the ASH Annl Meet; 2013 Dec 7–10; New Orleans, LA, USA.
   Google Scholar
• Keohane C, Radia DH, Harrison CN. Treatment and management of myelofibrosis in the era of JAK inhibitors. Biologics. 2013;7:189–98.
   PubMedGoogle Scholar