Patients with MF have significant disease burden, with debilitating symptoms that are progressive, relentless, and detrimental to QOL.3,11
Splenomegaly, present in approximately 85% of patients with MF, occurs because the inability of the bone marrow to produce normal blood cells forces the spleen to do so.2,12
Splenomegaly > 8 cm below the left costal margin and progressive anemia are common in patients with MF. Some patients may require blood transfusions.13-15
MPN-related symptoms are driven by the increased production of circulating inflammatory cytokines resulting from the abnormal activation of JAK signaling.5
Splenomegaly, cytopenias, and MPN-related symptoms generally worsen over the course of the disease.3,14-16
- Swerdlow SH, Campo E, Harrison NL, et al. WHO classification of tumours of haematopoietic and lymphoid tissues. Vol 2. 4th ed. Geneva: World Health Organization; 2008.
- Abdel-Wahab OI, Levine RL. Primary myelofibrosis: Update on definition, pathogenesis, and treatment. Annu Rev Med. 2009;60:233-245.
- Mesa RA, Schwager S, Radia D, et al. The myelofibrosis symptom assessment form (MFSAF): An evidence-based brief inventory to measure quality of life and symptomatic response to treatment in myelofibrosis. Leuk Res. 2009;33(9):1199-1203.
- Girodon F, Bonicelli G, Schaeffer C, et al. Significant increase in the apparent incidence of essential thrombocythemia related to new WHO diagnostic criteria: A population-based study. Haematologica. 2009;94(6):865-869.
- Hasselbalch HC. The role of cytokines in the initiation and progression of myelofibrosis. Cytokine Growth Factor Rev. 2013;24(2):133-145.
- Levine RL, Pardanani A, Tefferi A, Gilliland DG. Role of JAK2 in the pathogenesis and therapy of myeloproliferative disorders. Nat Rev Cancer. 2007;7(9):673-683.
- Klampfl T, Gisslinger H, Harutyunyan AS, et al. Somatic mutations of calreticulin in myeloproliferative neoplasms. N Engl J Med. 2013;369(25):2379-2390.
- 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. 2009;113(13):2895-2901.
- Tefferi A. Essential thrombocythemia, polycythemia vera, and myelofibrosis: Current management and the prospect of targeted therapy. Am J Hematol. 2008;83(6):491-497.
- Cervantes F, Barosi G. Myelofibrosis with myeloid metaplasia: Diagnosis, prognostic factors, and staging. Semin Oncol. 2005;32(4):395-402.
- Scherber R, Dueck AC, Johansson P, et al. The myeloproliferative neoplasm symptom assessment form (MPN-SAF): International prospective validation and reliability trial in 402 patients. Blood. 2011;118(2):401-408.
- Barosi G. Myelofibrosis with myeloid metaplasia: Diagnostic definition and prognostic classification for clinical studies and treatment guidelines. J Clin Oncol. 1999;17(9):2954-2970.
- O'Reilly RA. Splenomegaly at a united states county hospital: Diagnostic evaluation of 170 patients. Am J Med Sci. 1996;312(4):160-165.
- Thiele J, Kvasnicka HM. Grade of bone marrow fibrosis is associated with relevant hematological findings-a clinicopathological study on 865 patients with chronic idiopathic myelofibrosis. Ann Hematol. 2006;85(4):226-232.
- Tefferi A, Lasho TL, Jimma T, et al. One thousand patients with primary myelofibrosis: The mayo clinic experience. Mayo Clin Proc. 2012;87(1):25-33.
- Barosi G, Rosti V, Bonetti E, et al. Evidence that prefibrotic myelofibrosis is aligned along a clinical and biological continuum featuring primary myelofibrosis. PLoS One. 2012;7(4):e35631.
- Emanuel RM, Dueck AC, Geyer HL, et al. Myeloproliferative neoplasm (MPN) symptom assessment form total symptom score: Prospective international assessment of an abbreviated symptom burden scoring system among patients with MPNs. J Clin Oncol. 2012;30(33):4098-4103.
- Silver RT, Chow W, Orazi A, Arles SP, Goldsmith SJ. Evaluation of WHO criteria for diagnosis of polycythemia vera: A prospective analysis. Blood. 2013;122:1881-1886.
- Tefferi A. How I treat myelofibrosis. Blood. 2011;117(13):3494-3504.
- Mascarenhas JO, Orazi A, Bhalla KN, Champlin RE, Harrison C, Hoffman R. Advances in myelofibrosis: A clinical case approach. Haematologica. 2013;98(10):1499-1509.
- 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. 2009;114(5):937-951.
- Tefferi A. Mutations galore in myeloproliferative neoplasms: Would the real spartacus please stand up? Leukemia. 2011;25(7):1059-1063.
- 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. 2012;87(5):562-568.
- Rumi E, Pietra D, Guglielmelli P, et al. Acquired copy-neutral loss of heterozygosity of chromosome 1p as a molecular event associated with marrow fibrosis in MPL-mutated myeloproliferative neoplasms. Blood. 2013;121:4388-4395.
- Rumi E, Pietra D, Pascutto C, et al. Clinical effect of driver mutations of JAK2, CALR or MPL in primary myelofibrosis. Blood. 2014; 124:1062-1069.
- Nangalia J, Massie CE, Baxter EJ, et al. Somatic CALR mutations in myeloproliferative neoplasms with nonmutated JAK2. N Engl J Med. 2013;369(25):2391-2405.
- 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. 2010;115(9):1703-1708.
- Vannucchi AM, Lasho TL, Guglielmelli P, et al. Mutations and prognosis in primary myelofibrosis. Leukemia. 2013;27:1861-1869.
- Guglielmelli P, Lasho TL, Rotunno G, et al. The number of prognostically detrimental mutations and prognosis in primary myelofibrosis: An international study of 797 patients. Leukemia. 2014;28:1804-1810.
- Tefferi A, Lasho TL, Finke CM, et al. CALR vs JAK2 vs MPL-mutated or triple-negative myelofibrosis: Clinical, cytogenetic and molecular comparisons. Leukemia. 2014;28(7):1472-1477.
- Tefferi A, Guglielmelli P, Lasho TL, et al. CALR and ASXL1 mutations-based molecular prognostication in primary myelofibrosis: An international study of 570 patients. Leukemia. 2014;1494-1500.