TY - CHAP M1 - Book, Section TI - Myelodysplastic Syndromes A1 - Bates, Jill S. A2 - DiPiro, Joseph T. A2 - Yee, Gary C. A2 - Posey, L. Michael A2 - Haines, Stuart T. A2 - Nolin, Thomas D. A2 - Ellingrod, Vicki PY - 2020 T2 - Pharmacotherapy: A Pathophysiologic Approach, 11e AB - KEY CONCEPTSMyelodysplastic syndromes (MDS) primarily affect elderly adults.The exact cause of MDS is unknown and is probably multifactorial. MDS have been associated with host-specific characteristics, environmental, lifestyle, and therapeutic exposures.Genomic instability drives MDS development and progression. 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, infection, bleeding/bruising, lethargy, or other symptoms related to cytopenias.The prognosis of patients with MDS is variable and depends on the biology of the MDS and host characteristics. 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 goals of therapy for MDS are to change the natural history of the disease, reduce the number of red blood cell transfusions, and improve quality of life.Lenalidomide should be considered for patients with MDS that harbors a del(5q) clone and is particularly beneficial in those with symptomatic anemia.Patients with lower-risk MDS and symptomatic anemia who have a serum erythropoietin level ≤500 mU/mL (U/L) are suitable candidates for an erythropoietin stimulating agent with or without growth factor support.A subset of lower-risk MDS patients respond well to antithymocyte globulin (ie, immunosuppressive therapy), which is most effective in patients who have a hypocellular marrow, MDS that expresses HLA DR15 with trisomy 8 as the sole cytogenetic abnormality, refractory anemia, and are younger than 60 years old.Further evaluation is required to determine optimal hypomethylating agent treatment regimens.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=1182476816 ER -