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Hematopoietic Cell Transplant for Myelofibrosis

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Abstract

Myelofibrosis (MF) can present as primary myelofibrosis (PMF) or evolve from polycythemia vera (PV) or essential thrombocythemia (ET). Regardless of the etiology, MF is characterized as a clonal stem cell disorder associated with elevated levels of pro-inflammatory and pro-angiogenic cytokines such as tumor necrosis factor-alpha (TNF-α), interleukin-6 (IL-6) and interferon-gamma (IFN-γ), resulting in a bone marrow stromal reaction that includes varying degrees of reticulin and collagen fibrosis and osteosclerosis. The median age at diagnosis of MF is 60–65 years. The clinical course is heterogeneous with a median life expectancy in nontransplanted patients ranging from less than 2 years to more than 10 years. MF frequently involves the spleen, resulting in massive splenomegaly, severe constitutional symptoms, a hypermetabolic state, and cachexia. Worsening cytopenias and increasing numbers of circulating blasts and eventually leukemic transformation commonly mark disease progression. Allogeneic hematopoietic cell transplantation (HCT) is currently the only treatment with proven curative potential for patients with MF. In the last decade, the numbers of patients undergoing HCT for MF have more than doubled as reduced intensity conditioning (RIC) and non-sibling donor sources have increased accessibility. The following chapter will review the staging systems and other disease and non-disease related risk factors that guide decision-making, as well as choice of donor sources and conditioning regimens, and post-transplant complications which contribute to outcomes following transplantation.

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Authors and Affiliations

  1. Fred Hutchinson Cancer Research Center, University of Washington Medical Center, Seattle, WA, USA

    Rachel B. Salit

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  1. Center for Hematologic Malignancies Adult Blood and Marrow Transplant & Cell Therapy Program, Knight Cancer Institute Oregon Health & Science University, Portland, OR, USA

    Richard T. Maziarz

  2. Center for Hematologic Malignancies Adult Blood and Marrow Transplant & Cell Therapy Program, Knight Cancer Institute Oregon Health & Science University, Portland, OR, USA

    Susan Schubach Slater

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Salit, R.B. (2021). Hematopoietic Cell Transplant for Myelofibrosis. In: Maziarz, R.T., Slater, S.S. (eds) Blood and Marrow Transplant Handbook. Springer, Cham. https://doi.org/10.1007/978-3-030-53626-8_20

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