By Hien K. Duong, MD
Today’s Education Program Session on myelodysplastic syndromes (MDS) from 7:30 to 9:00 a.m. (repeated this afternoon from 4:00 to 5:30 p.m.), in Auditorium ABC of the Ernest N. Morial Convention Center will summarize some of the exciting developments that have occurred in recent years in this active field. Since the publication of the International Prognostic Scoring System (IPSS) in 1997, there have been substantial advancements in the understanding of disease biology, as well as in the prognostication and treatment of patients with MDS.
What’s in a name? In Shakespeare’s lyrical tale of two star-crossed lovers, Juliet poses this question to Romeo, suggesting that names are a convention of little importance except in what they signify. Dr. David Steensma from the Dana-Farber Cancer Institute in Boston will explore the meaning of names with respect to the evolution of the definition of MDS and the classification of MDS subtypes. He will discuss several features of the 2008 World Health Organization (WHO) classification of myeloid neoplasms and MDS, compare newer prognostic systems to the IPSS, and survey the significance of several new molecular abnormalities in MDS, including loss-of-function mutations in TET2. For those bone marrow failure syndromes that do not meet WHO criteria for MDS, but also do not meet any other disease classification, a category of “idiopathic cytopenia(s) of undetermined significance” (ICUS) has been proposed; Dr. Steensma will discuss the proposed criteria for ICUS, the natural history of ICUS, and its implications for monitoring patients.
Although Romeo and Juliet failed to meet the end they had hoped for, their story is timeless. Following this theme of union, Dr. Mikkael Sekeres from the Cleveland Clinic Taussig Cancer Institute will discuss links between disease biology and therapy in patients with MDS. At the time of publication of the IPSS, few treatment options were available to patients aside from supportive care with transfusions, or stem cell transplantation. But now there are three drugs that have been specifically approved by the Food & Drug Administration (FDA) for MDS: azacitidine, lenalidomide, and decitabine. In addition, clinicians are using multiple hematopoietic growth factors, immunosuppressants, and cytotoxic agents for patients with MDS. Dr. Sekeres will discuss our current understanding of the disease biology of MDS and the most appropriate therapeutic strategies for patients with lower- and higher-risk MDS. He will review newer investigative agents, including thrombopoietin agonists that stimulate platelet production, and other drugs that target novel pathways in MDS or that show promise for combination therapies.
Dr. Heather Leitch from the University of British Columbia will close the session by exploring suggestive data about the importance of iron overload in patients with MDS. Since humans lack a physiologic mechanism for excretion of excess iron, iron accumulation due to chronic red blood cell transfusions in patients with marrow failure syndromes such as MDS can become a cause of morbidity and a contributing factor to mortality. Dr. Leitch will review the evidence supporting the idea that iron overload is harmful in MDS, and she will discuss both the potential for iron-lowering benefit from iron chelation therapy (ICT) and questions regarding long-term clinical benefit. In patients with beta-thalassemia major, ICT has been shown to help decrease morbidity and improve overall survival; whether this benefit is also seen in a clonal disorder such as MDS, where patients’ life expectancy is limited by disease progression or complications of cytopenias, is a question that requires careful clinical investigation.
Our collective understanding of MDS has improved significantly over the last several decades, but there is still a long way to go. As we continue to improve our understanding of these challenging disease processes, including better understanding of molecular mechanisms, we will be able to develop better classification and prognostication systems and more effective therapies, and, most importantly, improve outcomes for our patients.
Dr. Duong indicated no relevant conflicts of interest.
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