@article{3090275, title = "Incorporating novel agents in the treatment of myelodysplastic syndromes", author = "Anargyrou, K. and Vassilakopoulos, T.P. and Angelopoulou, M.K. and Terpos, E.", journal = "Leukemia Research", year = "2010", volume = "34", number = "1", pages = "6-17", issn = "0145-2126", doi = "10.1016/j.leukres.2009.07.021", keywords = "4 phenylbutyric acid; 5 aza 2' deoxycytidine; angiogenesis inhibitor; arsenic trioxide; azacitidine; bevacizumab; deferasirox; deferiprone; DNA methyltransferase inhibitor; eltrombopag; etanercept; histone deacetylase inhibitor; imatinib; immunomodulating agent; iron chelating agent; lenalidomide; lonafarnib; n (2 aminophenyl) 4 (3 pyridinylmethoxycarbonylaminomethyl)benzamide; n (2 aminophenyl) 4 [4 (3 pyridinyl) 2 pyrimidinylaminomethyl]benzamide; novel erythropoiesis stimulating protein; placebo; protein farnesyltransferase inhibitor; recombinant erythropoietin; romidepsin; romiplostim; romiplostin; sndx 275; thalidomide; thalomide; tipifarnib; tumor necrosis factor alpha inhibitor; unclassified drug; unindexed drug; valproic acid; vorinostat, abdominal pain; abnormal laboratory result; acute granulocytic leukemia; allogeneic stem cell transplantation; anemia; anorexia; arthralgia; arthritis; biliary cirrhosis; blast cell; bone marrow suppression; cellulitis; cerebrovascular accident; chronic myelomonocytic leukemia; clinical trial; comorbidity; congestive heart failure; constipation; continuous infusion; cutaneous T cell lymphoma; cytogenetics; dementia; diarrhea; dizziness; dosage schedule comparison; dose response; drug absorption; drug bioavailability; drug blood level; drug dose comparison; drug dose escalation; drug dose increase; drug dose reduction; drug fatality; drug half life; drug mechanism; drug megadose; drug response; drug safety; drug tolerability; drug withdrawal; dyspepsia; dyspnea; epistaxis; face edema; fatigue; febrile neutropenia; fever; gastrointestinal symptom; gastrointestinal toxicity; gene deletion; headache; hematologic malignancy; hemosiderosis; human; hyperbilirubinemia; hypertension; hypophosphatemia; idiopathic thrombocytopenic purpura; infection; injection site erythema; injection site pain; injection site reaction; iron overload; kidney failure; leg pain; leukemia; liver cirrhosis; liver dysfunction; loading drug dose; low drug dose; multiple cycle treatment; myalgia; myelodysplastic syndrome; myeloma; nausea; nephrotoxicity; neurotoxicity; neutropenia; nonhuman; optimal drug dose; pain; palliative therapy; pneumonia; priority journal; pruritus; rash; recommended drug dose; refractory anemia with excess blasts; refractory anemia with excess blasts in transformation; remission; repeated drug dose; review; rhinopharyngitis; rigor; sedation; shoulder pain; side effect; sore throat; survival rate; thrombocytopenia; thromboembolism; tremor; upper respiratory tract infection; urticaria; vertigo; vomiting; weight reduction, Antineoplastic Agents; Humans; Myelodysplastic Syndromes", abstract = "Myelodysplastic syndromes (MDS) are a group of heterogeneous clonal stem cell (SC) disorders that mainly affect the elderly population. They are characterized by ineffective hematopoiesis which results in quantitative and qualitative cellular defects and high incidence of leukemic transformation. Recent advances in MDS research have led to the development of novel agents which appears to improve remission rates and survival when compared to best supportive care. Currently azacitidine, decitabine, and lenalidomide are approved by the US FDA for the treatment of MDS, while the activity of other novel agents such as histone deacetylase inhibitors, farnesyl-transferase inhibitors, novel thrombopoietic agents, and anti-angiogenesis molecules is under evaluation. Erythropoietin-stimulating agents, iron chelating therapy and thrombopoietin receptor ligands may also improve quality of life and possibly prolong survival in MDS patients. The only treatment modality that can achieve long-term survival is the allogeneic SC transplantation which is given only in selected patients. Moreover the heterogeneity of MDS and the patient's advanced age and co-morbidity are significant factors besides cytogenetics, IPSS and WPSS that should be taken into account during the decision-making process. Therefore clinicians should treat patients with MDS on an individual basis aiming the increase of the response rates and the decrease of treatment-associated toxicities. This can only be achieved through the better understanding of the MDS subgroups. If we can better define MDS subgroups we will be able to identify patients who will benefit from the incorporation of the novel agents, as monotherapy or in combinations regimens along with supportive care. © 2009 Elsevier Ltd. All rights reserved." }