Azacitidine for treatment of patients with myelodysplastic syndromes (MDS): practical recommendations of the German MDS Study Group

Abstract

To cite this version:Katharina Götze, Uwe Platzbecker, Aristoteles Giagounidis, Detlef Haase, Michael Lübbert, et al.. Azacitidine for treatment of patients with myelodysplastic syndromes (MDS): practical recommendations of the German MDS Study Group. Annals of Hematology, Springer Verlag, 2010, 89 (9) Abstract Myelodysplastic syndromes (MDS) are a group of common bone marrow disorders characterized by ineffective hematopoiesis, peripheral cytopenias, and a substantial risk of progression to acute myeloid leukemia (AML). For many years, the main treatment option for MDS was best supportive care which alleviates symptoms, but has no effect on the natural course of the disease. Recently, demethylating agents have become available as a promising new treatment for patients with MDS. In two randomized clinical trials, the demethylating agent azacitidine has demonstrated a reduced risk of transformation to AML, improvement of peripheral blood values, an improved quality of life, and a definite survival advantage compared to conventional care regimens for patients with International Prognostic Scoring System score of intermediate-2 or high-risk MDS. This review aims to provide practical recommendations for the use of azacitidine and the management of its side effects in patients with MDS, assuring safe administration and best efficacy of treatment.

C/EBPγ deregulation results in differentiation arrest in acute myeloid leukemia

“…However, our data support an important role of this balance in myeloid differentiation. Currently, demethylating agents are the standard of care for patients with high-risk myelodysplastic syndromes (MDS) (47,48), reducing the risk of transformation to AML and improving overall survival. But although these agents are being used as therapeutic agents, very few specific gene targets have been identified, and certainly it is difficult to explain the effect on AML cells based on previously identified methylation targets (49,50).…”

EBV-positive Diffuse Large B-cell Lymphoma as a Secondary Malignancy Arising in a Myelodysplastic Syndrome Patient who Was Treated with Azacitidine

“…The hypomethylating agent azacitidine (AZA) has improved the cytopenia of high-risk MDS patients and prolonged their overall survival (1,2). Although AZA has been widely used in the treatment of MDS, there are no reports of the occurrence of secondary malignancies other than AML following its use.…”

Response to treatment with azacitidine in children with advanced myelodysplastic syndrome prior to hematopoietic stem cell transplantation

“…The short-and long-term side effect profile of AZA is better than that of AML-type chemotherapy. 25,27 AZA prior to HSCT was shown to improve outcome in a smaller MDS cohort, including pediatric patients without separate evaluation of this subgroup. 25 In a single case report, AZA was shown to induce complete remission in a child with treatment-related MDS and signs of early relapse after HSCT, however, long-term follow-up was not reported.…”