TY - CHAP M1 - Book, Section TI - Myelodysplastic Syndromes A1 - McCullough, Kristen B. A1 - Merten, Julianna A. A2 - DiPiro, Joseph T. A2 - Talbert, Robert L. A2 - Yee, Gary C. A2 - Matzke, Gary R. A2 - Wells, Barbara G. A2 - Posey, L. Michael Y1 - 2017 N1 - T2 - Pharmacotherapy: A Pathophysiologic Approach, 10e AB - KEY CONCEPTS Myelodysplastic syndromes (MDS) primarily affect elderly adults, with median age at diagnosis of 76 years. MDS are associated with environmental, occupational, and therapeutic exposures to chemicals or radiation. The clonal population of cells manifested as MDS results from enhanced self-renewal of a hematopoietic stem cell or acquisition of self-renewal in a progenitor cell, increased proliferative capacity in the abnormal clone, impaired cell differentiation, evasion of immune regulation, and antiapoptotic mechanisms in the disease-sustaining cell. Most patients with MDS present with fatigue and lethargy or symptoms related to anemia-induced tissue hypoxia. The prognosis of patients with MDS is variable. Overall survival time ranges from a few months to several years and is most accurately estimated with the International Prognostic Scoring System—Revised (IPSS-R). The primary goal of therapy is hematologic improvement for lower-risk patients and alteration in the natural course of the disease for higher-risk patients. Palliation of symptoms and improvement in quality of life are goals of therapy for all patients. Current guidelines recommend erythropoietin (EPO) or darbepoetin with or without filgrastim for management of anemia in patients with lower-risk MDS. Hypomethylating agents are appropriate for patients with lower-risk MDS with clinically significant neutropenia or thrombocytopenia, patients with anemia who are unlikely to respond to or have not responded to a trial of EPO or immunosuppressive therapy. Antithymocyte globulin is appropriate treatment for patients with lower risk, HLA DR15 positive expressing MDS who have symptomatic anemia that is unlikely to respond to erythropoietic agents. Lenalidomide is recommended for initial treatment of lower-risk 5q- syndrome accompanied by symptomatic anemia. Allogeneic hematopoietic stem cell transplantation offers potentially curative therapy to patients with MDS who have a donor and are healthy enough for the procedure. SN - PB - McGraw-Hill Education CY - New York, NY Y2 - 2024/03/29 UR - accesspharmacy.mhmedical.com/content.aspx?aid=1145222969 ER -