title: New therapeutic approaches in myelodysplastic syndromes: Hypomethylating agents and lenalidomide.
abstract: Recent advances in the treatment of myelodysplastic syndromes have come from the use of the hypomethylating agents decitabine and azacitidine as well as the immunomodulatory drug lenalidomide. Their clinical benefit has been demonstrated by randomized phase III clinical trials, mostly in high-risk and del(5q) myelodysplastic syndromes, respectively. Neither drug, however, appears to eradicate myelodysplastic stem cells, and thus they currently do not represent curative options. Here, we review data from both clinical and translational research on those drugs to identify their molecular and cellular mechanisms of action and to delineate paths for improved treatment allocation and further therapeutic advances in myelodysplastic syndromes.
authors.affiliation: Department of Hematology, Saint-Louis Hospital, Assistance Publique, Hopitaux de Paris, Paris Diderot University, Paris, France.
authors.forename: Clémence
authors.initials: C
authors.lastname: Loiseau
authors.affiliation: Institut National de la Santé et de la Recherche Médicale, Saint-Louis Institute, Paris, France.
authors.forename: Ashfaq
authors.initials: A
authors.lastname: Ali
authors.affiliation: Department of Hematology, Saint-Louis Hospital, Assistance Publique, Hopitaux de Paris, Paris Diderot University, Paris, France; Institut National de la Santé et de la Recherche Médicale, Saint-Louis Institute, Paris, France. Electronic address: raphael.itzykson@aphp.fr.
authors.forename: Raphael
authors.initials: R
authors.lastname: Itzykson
mesh_terms: D020533:Angiogenesis Inhibitors / Q000627:therapeutic use*; D000818:Animals; D000964:Antimetabolites, Antineoplastic / Q000627:therapeutic use*; D001374:Azacitidine / Q000031:analogs & derivatives* / Q000627:therapeutic use*; D017326:Clinical Trials, Phase III as Topic; D000077209:Decitabine; D006801:Humans; D000077269:Lenalidomide; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000473:pathology; D016032:Randomized Controlled Trials as Topic; D013792:Thalidomide / Q000031:analogs & derivatives* / Q000627:therapeutic use; D057170:Translational Research, Biomedical
chemical_list: D020533:Angiogenesis Inhibitors; D000964:Antimetabolites, Antineoplastic; D013792:Thalidomide; D000077209:Decitabine; D000077269:Lenalidomide; D001374:Azacitidine