Abstract
Living in low-resource countries, we have diverse geographical, political, and sociocultural profiles, but at the same time, we have similar economic and development constraints. We face major challenges, which include appropriate utilization of resources and limited trained workforce for providing maximal benefit by our treatments to our patients. This is especially true for cancer patients for whom the cost of diagnosis, treatment, and monitoring is prohibitively expensive. Pragmatic constraints affect our decisions for use of diagnostic tests; molecular pathway profiling currently is of academic interest. Financial constraints prevent us from delivering optimal treatments to our patients from the lower strata. What is appropriate diagnostic/treatment and follow-up options in the developed world are not practical for us working in developing countries due to aforementioned reasons.
No data are available on Philadelphia-negative myeloproliferative neoplasms (MPNs) with regard to epidemiology, survival, and treatment options for developing countries. A vast majority of patients have symptomatic high-risk disease requiring immediate therapy. Since treatment for all Philadelphia-negative neoplasms are palliative, practical decisions for diagnosis, therapy, and monitoring of patients are required.
For patients who present with chronic myeloid leukemia (CML), Philadelphia-positive initial treatment with imatinib is now the gold standard even in developing countries. The goal of therapy with tyrosine kinase inhibitors (TKIs) is now dose optimization to produce maximum and optimal responses with minimum toxicities. Majority of patients diagnosed with CML and receiving TKIs will probably die of causes unrelated to CML or its treatment.
This is a preview of subscription content, log in via an institution to check access.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
References
Mehta J, Wang H, Iqbal SU, Mesa R et al (2014) Epidemiology of myeloproliferative neoplasms in the United States. Leuk Lymphoma 55(3):595–600
Tefferi A, Vardiman JW (2008) Classification and diagnosis of myeloproliferative neoplasms: the 2008 World Health Organization criteria and point-of-care diagnostic algorithms. Leukemia 22:14–22
Baxter EJ, Scott LM, Campbell PJ, et al; Cancer Genome Project (2005) Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders. Lancet 365(9464):1054–1061
Tefferi A, Rumi E, Finazzi G et al (2013) Survival and prognosis among 1545 patients with contemporary polycythemia vera: an international study. Leukemia 27(9):1874–1881
Huang J, Li CY, Mesa RA et al (2008) Risk factors for leukemic transformation in patients with primary myelofibrosis. Cancer 112(12):2726–2732
Alvarez-Larran A, Bellosillo B, Martinez-Aveiles L et al (2009) Post polycythaemic myelofibrosis: frequency and risk factors for this complication in 116 patients. Br J Haematol 146(5):504–509
Tefferi A, Thiele J, Vannucchi AM, Barbui T et al (2014) An overview on CALR and CSF3R mutations and a proposal for revision of WHO diagnostic criteria for myeloproliferative neoplasm. Leukemia 28(7):1407–1413
Tefferi A, Elliot M (2007) Thrombosis in myeloproliferative disorders: prevalence, prognostic factors, and the role of leukocytes and JAK2V617F. Semin Thromb Hemost 33:313–320
Landolifi R, Marchioli R et al (2004) Efficacy and safety of Low-Dose Aspirin in polycythemia vera. N Engl J Med 350:114–124
Geyer HL, Mesa RA (2014) Therapy for myeloproliferative neoplasms: when, which agent, and how? Blood 124(24):3529–3537
Kiladjian JJ, Chevret S, Dosquet C et al (2011) Treatment of polycythemia vera with hydroxyurea and pipobroman: final results of a randomized trial initiated in 1980. J Clin Oncol 29(29):3907–3913
Quintas-Cardama A, Abdel-Wahab O, Manshouri T et al (2013) Molecular analysis of patients with polycythemia vera or essential thrombocythemia receiving pegylated interferon alpha-2a. Blood 122(6):893–901
Srdan V, Pssamonti F et al (2014) 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 120:513–520
Smalberg JH, Arends LR, Valla DC et al (2012) Myeloproliferative neoplasms in Budd-Chiari syndrome and portal vein thrombosis: a meta-analysis. Blood 120(25):4921–4928
Ruggeri M, Rodeghiero F, Tosetto A, et al, Gruppo Italiano Malattie Ematologiche dell, Adulto (GIMEMA) Chronic Myeloproliferative Diseases Working Party (2008) Postsurgery outcomes in patients with polycythemia vera and essential thrombocythemia: a retrospective survey. Blood 111(2):666–671
Vannucchi MA (2014) How I treat polycythemia vera. Blood 124:3212–3220
Hoffman R, Benz EJ Jr, Shattil SJ et al (2000) Hematology: basic principles and practice, 3rd edn. Churchill Livingstone, New York, pp 1106–1155, 1172–1205
Tefferi A, Solberg LA, Silverstein MN et al (2000) A clinical update on polycythemia vera and essential thrombocythemia. Am J Med 109:141–149
Klampfl T, Gisslinger H, Harutyunyan AS et al (2013) Somatic mutations of calreticulin in myeloproliferative neoplasms. N Engl J Med 369(25):2379–2390
Beer PA, Erber WN, Campbell PJ et al (2011) How I treat thrombocythemia. Blood 117(5):1472–1482
Gisslinger H, Gotic M, Holowiecki J et al (2013) Anagrelide compared with hydroxyurea in WHO-classified essential thrombocythemia: the ANAHYDRET study, a randomized controlled trial. Blood 121(10):1720–1728
Rumi E, Pietra D, Pascutto C et al (2014) Clinical effect of driver mutations of JAK2, CALR or MPL in primary myelofibrosis. Blood 124(7):1062–1069
Hussein K, Pardani A, Van Dyke DL et al (2010) International prognostic scoring system-independent cytogenetic risk categorization in primary myelofibrosis. Blood 115:496–499
Passamonti F, Cervantes F, Vannucchi AM et al (2010) 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
Barbui T, Barosi G, Birgegard G et al (2011) Philadelphia-negative classical myeloproliferative neoplasms: critical concepts and management recommendations from European LeukemiaNet. J Clin Oncol 20(29):6761–6770
Verstovsek S, Mesa A, Gotlib JA et al (2012) Double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med 366:799–807
Bacigalupo A, Soraru M, Dominietto A et al (2010) Allogeneic hemopoietic SCT for patients with primary myelofibrosis: a predictive transplant score based on transfusion requirement, spleen size and donor type. Bone Marrow Transplant 45(3):458–463
GLOBOCAN 2012: Estimated Cancer Incidence, Mortality and Prevalence Worldwide, 2012. www.globocan.iarc.fr. Accessed 21 Nov 2014
Aziz Z, Iqbal J et al (2007) Treatment of CML in the Imatinib era: perspective from a developing country. Cancer 109(6):1138–1145
O’Brien SG, Guihot F, Larson RA et al (2003) Imatinib compared with interferon and low-dose cytarabine for newly diagnosed chronic-phase chronic myeloid leukemia. N Engl J Med 348(11):994–1004
Ruiz-Argelles GJ et al (2008) Therapeutic choices in patients with ph-positive CML living in Mexico in the tyrosine kinase era: SCT or TKIs? Bone Marrow Transplant 42:23–28
Author information
Authors and Affiliations
Corresponding author
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2015 Springer International Publishing Switzerland
About this chapter
Cite this chapter
Aziz, Z. (2015). Myeloproliferative Neoplasms. In: Droz, JP., Carme, B., Couppié, P., Nacher, M., Thiéblemont, C. (eds) Tropical Hemato-Oncology. Springer, Cham. https://doi.org/10.1007/978-3-319-18257-5_26
Download citation
DOI: https://doi.org/10.1007/978-3-319-18257-5_26
Publisher Name: Springer, Cham
Print ISBN: 978-3-319-18256-8
Online ISBN: 978-3-319-18257-5
eBook Packages: MedicineMedicine (R0)