5-Azacytidine in myelodysplastic syndromes: A clinical practice guideline

SummaryBackground ysplastic syndrome (MDS) is a clonal disorder of hematopoiesis that results in peripheral blood cytopenias and a marked propensity to progress to acute myelogenous leukemia. With 40,000–76,000 new cases per year in the USA, MDS is the commonest of the hematological malignancies and represents a significant burden of morbidity and premature death. Although supportive or palliative measures such as blood transfusion have long been the mainstay of management of MDS, disease-modifying medical therapies have recently become available. The most extensively characterized of these is 5-azacytidine (5-Aza); however, no consensus exists on how this agent should be deployed in MDS. s rarching search of the literature identified 7019 citations investigating the treatment or management of MDS. Of those, six clinical articles of prospective phase 2–3 study design or meta-analyses were selected for inclusion in a systematic review of the evidence. sions nadian Consortium on Evidence-Based Care in MDS recommends 5-Aza as first line therapy in all MDS patients with IPSS high-intermediate and high risk scores including WHO-defined AML (20–30% blasts) who cannot proceed immediately to allogeneic stem cell transplant. is not recommended as first line therapy with MDS patients with IPSS Low and Low-intermediate risk scores as there is no evidence that it alters the natural history of the disease nor is superior to standard therapy. S consortium does not recommend combining 5-Aza with other agents at this time outside the context of a clinical trial.