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Azacitidine May Be Effective in Myelodysplastic Syndrome

It improves survival in patients with higher-risk myelodysplastic syndrome

MONDAY, Feb. 23 (HealthDay News) -- Azacitidine, as opposed to conventional care, improves overall survival in patients with higher-risk myelodysplastic syndrome, according to research published online Feb. 19 in The Lancet Oncology.

Pierre Fenaux, M.D., of the Universite Paris XIII in France, and colleagues conducted a phase III international open-label trial of 358 patients with higher-risk myelodysplastic syndrome. Patients were randomized to receive either azacitidine or conventional care consisting of best supportive care, low-dose cytarabine or intensive chemotherapy. A median follow-up of 21.1 months was reported.

Median overall survival was significantly lengthened in the azacitidine group compared with conventional care (24.5 months versus 15 months), the investigators found. After two years, 50.8 percent of patients in the azacitidine group were alive compared with 26.2 percent in the conventional care group. Patients in both groups reported grade 3 or 4 peripheral cytopenias as the most common adverse event, the researchers report.

"The results of this study indicate that azacitidine significantly lengthens overall survival and changes the natural history of myelodysplastic syndrome in patients with higher-risk disease," the authors state, but add "intensive chemotherapy may remain the appropriate treatment in some situations in higher-risk myelodysplastic syndrome, especially before allogeneic stem-cell transplantation in candidates for this procedure who have an excess of marrow blasts."

Several of the study authors report financial relationships with pharmaceutical companies, including Celgene, which sponsored the research.

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