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PURPOSE In contrast to 5-azacytidine (5-aza), allogeneic stem-cell transplantation (HSCT) represents a curative treatment strategy for patients with myelodysplastic syndromes (MDS), but therapy-related mortality (TRM) limits its broader use in elderly patients with MDS. The present prospective multicenter study compared HSCT following 5-aza pretreatment with continuous 5-aza treatment in patients...

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    In a prospective study of AZA pretreatment followed by HSCT in patients with available donor there was a significant improvement in EFS when compared to AZA alone, despite a 19% TRM at 1 year. A significant number of patients dropped off during the AZA pretreatment phase due to progression, death or toxicity; highlighting the need for more effective therapy.