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Nontransplant Treatment Options for Myelofibrosis: How to Treat Anemia, Splenomegaly, Constitutional Symptoms, and Extramedullary Disease

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Myeloproliferative Neoplasms

Part of the book series: Hematologic Malignancies ((HEMATOLOGIC))

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Abstract

Current drug therapy in PMF is not curative and has not been shown to prolong survival. Allogenic stem cell transplant (ASCT) is potentially curative but also associated with relatively high risk of death and morbidity. Therefore, one must first determine if a particular patient needs any form of therapy and carefully select the treatment strategy with the best chance of inducing disease control without compromising life expectancy (Table 15.1 and Fig. 15.1). According to the DIPSS-plus prognostic model (Gangat et al. 2011), the respective median survival of low- or intermediate-1-risk patients exceeds 15 and 6 years and even longer for patients below age 65 years. Therefore, the risk of allo-SCT-associated mortality and morbidity is not justified in such patients. Similarly, there is no evidence to support the value of conventional drug therapy in asymptomatic patients with low- or intermediate-1-risk disease. Therefore, a “watch and wait” treatment strategy is reasonable and usually preferred in asymptomatic low- or intermediate-risk patients with MF. MF patients with high- or intermediate-2-risk disease are usually symptomatic and can be managed by conventional drug therapy, splenectomy, radiotherapy, allo-SCT, or experimental drug therapy (Fig. 15.1). The goal of therapy in using allo-SCT is cure or prolongation of life. The goal of therapy in nontransplant candidates is disease palliation.

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  1. Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, MN, USA

    Ayalew Tefferi

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  1. Div. Ematologia, Ospedali Riuniti di Bergamo, Largo Barozzi 1, Bergamo, 24128, Italy

    Tiziano Barbui

  2. Dept. Internal Medicine, Div. Hematology, Mayo Clinic, First Street SW. 200, Rochester, 55905, Minnesota, USA

    Ayalew Tefferi

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Tefferi, A. (2012). Nontransplant Treatment Options for Myelofibrosis: How to Treat Anemia, Splenomegaly, Constitutional Symptoms, and Extramedullary Disease. In: Barbui, T., Tefferi, A. (eds) Myeloproliferative Neoplasms. Hematologic Malignancies. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-24989-1_15

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