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Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematopoietic stem cell disorders that impair normal hematopoiesis, resulting in a variable number of cytopenias and a potential to evolve into acute myeloid leukaemia (AML).1 With a median age at diagnosis around 70 years, MDS typically affects the elderly.1,2 Hence, there is much morbidity and mortality associated with this patient population, as patients frequently suffer from complications due to cytopenias as well as other comorbidities. The two systems used for classifying MDS are the French American and British (FAB) criteria and the more recently revised World Health Organization (WHO) classification system. A third system, the International Prognostic Scoring System (IPSS), can predict survival based on percentage of bone marrow blasts, karyotype and number of peripheral blood cytopenias3 and is the most widely used prognostic tool for assisting with treatment decisions.
For many years, supportive care with blood products (red blood cell [RBC] and platelet transfusions), hematopoietic growth factors and antibiotics remained the only treatment modality for MDS patients, until the development of three novel agents that may alter the natural history of this disease. Within the past decade, the US Food and Drug Administration (FDA) has approved an immunomodulatory agent, lenalidomide (Revlimid™, Celgene) and two hypomethylating agents, decitabine (Dacogen™, Eisai, Inc.) and azacitidine (Vidaza™, Celgene) for the treatment of patients with MDS. In simple practice, therapy is tailored to IPSS score with an emphasis on supportive care therapies or lenalidomide for lower-risk patients (IPSS low or intermediate [int]-1) and more intensive therapies such as conventional chemotherapy, allogeneic hematopoietic stem cell transplant (HST) and clinical trials, as well as hypomethylating agents, for patients with higher-risk disease (IPSS int-2 or high) or lower-risk patients with progressive disease.3