Idiopathic myelofibrosis.
Idiopathic myelofibrosis (IMF) is characterized by anemia, progressive splenomegaly, bone marrow fibrosis, and extramedullary hematopoiesis. However, patients with a transitional myeloproliferative disorder (MPD), a prefibrotic form of myelofibrosis, or myelofibrosis with a fatty bone marrow share many features of IMF but have clinical characteristics that deviate from the classical description of IMF. A phenomenon that serves as a unique biomarker of IMF is the constitutive mobilization of hematopoietic progenitor cells (HPCs) and/or endothelial progenitor cells (EPCs) from the bone marrow to the peripheral blood and other extramedullary sites. Using such parameters as hemoglobin level, white blood cell count, and number of blasts in the peripheral blood, prognostic scores can be developed by which to base therapeutic decisions. Androgens, recombinant human erythropoietin (rHuEpo), and thalidomide are effective modalities of treatment of the anemia of IMF. Systemic symptoms of excess myeloproliferation are the primary indication for treatment with chemotherapeutic agents. Hydroxyurea is the most commonly used drug. Ablation of the abnormal hematopoietic clone with high-dose chemotherapy and allogeneic stem cell transplantation offers an opportunity to cure patients with IMF. The use of fully myeloablative conditioning regimens, with or without total body irradiation, is associated with a high transplant-related mortality rate (27% to 48%), especially in patients with advanced disease and in the elderly. The use of reduced intensity conditioning (RIC) regimens has resulted in prolonged survival and lower transplant-related mortality.
Barosi
Giovanni
G
Laboratory of Clinical Epidemiology, IRCCS Policlinico S. Matteo, Pavia, Italy.
Hoffman
Ronald
R
D015415:Biomarkers / Q000097:blood; D001853:Bone Marrow / Q000378:metabolism / Q000473:pathology; D042783:Endothelial Cells / Q000378:metabolism / Q000473:pathology; D006412:Hematopoietic Stem Cells / Q000378:metabolism / Q000473:pathology; D006801:Humans; D055728:Primary Myelofibrosis / Q000097:blood / Q000473:pathology / Q000628:therapy
D015415:Biomarkers