Clinical Roundtable Monograph

Myelodysplastic syndromes (MDS) represent a collection of heterogeneous malignant bone marrow stem cell disorders that result in the production of dysplastic and ineffective blood cells. The disease is marked by gradually worsening cytopenias and a variable risk for the eventual transformation to acute myelogenous leukemia (AML). The risk of developing MDS increases with age, and disease onset before 50 years is unusual. Several morphologic subtypes of MDS have been identified. Each of these subtypes has specific prognostic and morphologic and/or cytogenetic features which make it unique. The International Prognostic Scoring System (IPSS) was developed to aid in determining the prognosis of patients with MDS; this system categorizes patients into four risk groups for both overall survival and transformation to AML: low, intermediate-1, intermediate-2, and high. The management of MDS is based on the goal of controlling cytopenia-related symptoms, improving survival, improving quality of life, and decreasing risk of progression to AML. Treatment strategies include supportive care, iron chelation, treatment with hematopoietic growth factors, immunosuppressive therapies including lenalidomide, antithymocyte globulin, chemotherapy (eg, azacitidine, decitabine, low-dose Ara-C, 7+3 chemotherapy), and stem cell transplantation. However, selecting the appropriate therapy for each individual patient is critical to optimize clinical benefit. This monograph discusses treatment selection for the MDS patient, including a discussion of the overall survival and maintenance of MDS patients, how an appropriate therapy should be chosen in the community setting, and how MDS classification and risk stratification impacts treatment decisions. Supported through funding from Celgene Corporation. Disclaimer Funding for this Clinical Roundtable Monograph has been provided by Celgene Corporation. Support of this monograph does not imply the supporter’s agreement with the views expressed herein. Every effort has been made to ensure that drug usage and other information are presented accurately; however, the ultimate responsibility rests with the prescribing physician. Millennium Medical Publishing, Inc, the supporter, and the participants shall not be held responsible for errors or for any consequences arising from the use of information contained herein. Readers are strongly urged to consult any relevant primary literature. No claims or endorsements are made for any drug or compound at present under clinical investigation. ©2009 Millennium Medical Publishing, Inc. 611 Broadway, Suite 310, New York, NY 10012. Printed in the USA. All rights reserved, including the right of reproduction, in whole or in part, in any form. Overall Survival and Maintenance of MDS Patients Lewis R, Silverman, MD 3 Choosing an Appropriate Therapy for Lower-risk MDS in the Community Setting Roger M. Lyons, MD, FACP 5 MDS Classification and Risk Stratification Jamile M. Shammo, MD, FASCP 8 Combination Therapies for MDS Bart L. Scott, MD 13 Table of