title: Myelodysplastic syndromes.
abstract: Efforts made during the last few years have helped unravel the complex pathogenesis of the myelodysplastic syndromes (MDS). A large number of studies, made possible by the introduction of newer technologies, have led to major progress in understanding the heterogeneous genetic and biological abnormalities contributing to the development and progression of myelodysplasia. Better insights into these pathogenetic processes will aid the development of newer and more successful therapies for MDS patients. The identification of specific genes involved in the emergence and progression of the myelodysplastic clone has extended biological findings into the clinic. Recently, several clinical trials have used selective compounds to target and inhibit the disrupted signal transduction pathway in myelodysplastic patients. The demonstration of genetic abnormalities present not only in MDS patients but also in acute myeloid leukemia (AML) patients or in chronic myeloproliferative disorders (CMPD) has prompted extension of a number of clinical trials from AML and CMPD to MDS patients. In spite of this, the more complex and heterogeneous pathogenesis underlying the myelodysplastic process is responsible for the often different and in same cases worse clinical results obtained in MDS patients. Finally, the identification of myelodysplasia-associated antigens that may be targeted by an immunotherapeutic approach represents a future perspective in tailored therapy for MDS patients.
authors.affiliation: Dept. of Clinical and Biological Sciences of the University of Turin, San Luigi Hospital, Gonzole 10, 10043 Orbassano-Torino, Italy. daniela.cilloni@unito.it
authors.forename: Daniela
authors.initials: D
authors.lastname: Cilloni
authors.forename: Francesca
authors.initials: F
authors.lastname: Messa
authors.forename: Sonia
authors.initials: S
authors.lastname: Carturan
authors.forename: Francesca
authors.initials: F
authors.lastname: Arruga
authors.forename: Ilaria
authors.initials: I
authors.lastname: Defilippi
authors.forename: Emanuela
authors.initials: E
authors.lastname: Messa
authors.forename: Enrico
authors.initials: E
authors.lastname: Gottardi
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Saglio
mesh_terms: D000818:Animals; D002869:Chromosome Aberrations; D002986:Clinical Trials as Topic; D000939:Epitopes / Q000737:chemistry; D006801:Humans; D007167:Immunotherapy / Q000379:methods; D009190:Myelodysplastic Syndromes / Q000175:diagnosis* / Q000235:genetics* / Q000628:therapy; D015398:Signal Transduction; D014178:Translocation, Genetic; D025721:WT1 Proteins / Q000378:metabolism
chemical_list: D000939:Epitopes; D025721:WT1 Proteins