title: Current therapy of myelodysplastic syndromes.
abstract: After being a neglected and poorly-understood disorder for many years, there has been a recent explosion of data regarding the complex pathogenesis of myelodysplastic syndromes (MDS). On the therapeutic front, the approval of azacitidine, decitabine, and lenalidomide in the last decade was a major breakthrough. Nonetheless, the responses to these agents are limited and most patients progress within 2 years. Allogeneic stem cell transplantation remains the only potentially curative therapy, but it is associated with significant toxicity and limited efficacy. Lack or loss of response after standard therapies is associated with dismal outcomes. Many unanswered questions remain regarding the optimal use of current therapies including patient selection, response prediction, therapy sequencing and combinations, and management of resistance. It is hoped that the improved understanding of the underpinnings of the complex mechanisms of pathogenesis will be translated into novel therapeutic approaches and better prognostic/predictive tools that would facilitate accurate risk-adaptive therapy.
authors: Department of Oncology, the Sidney Kimmel Comprehensive Cancer Center at the Johns Hopkins University, Baltimore, MD 21287, USA. azeidan1@jhmi.edu
authors: Amer M
authors: AM
authors: Zeidan
authors: Yuliya
authors: Y
authors: Linhares
authors: Steven D
authors: SD
authors: Gore
mesh_terms: D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use; D003131:Combined Modality Therapy; D018450:Disease Progression; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D009190:Myelodysplastic Syndromes / Q000628:therapy*; D011379:Prognosis; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents
keywords: Allogeneic stem cell transplantation; Azacitidine; Chemotherapy; Decitabine; Erythropoiesis-stimulating agents; Hypomethylating agents; Immunosuppressive therapy; Lenalidomide; Myelodysplastic syndromes (MDS)
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