Azacitidine for Myelodysplasia

Myelodysplasia is a chronic disorder of marrow stem cells that is associated with ineffective hematopoiesis, peripheral blood cytopenias, and a variable risk for transformation into acute leukemia. In addition to supportive therapy with transfusions and stimulating factors, drugs that affect stem-cell proliferation are often given to restore marrow function. Azacitidine, a hypomethylating agent, is one of the oldest drugs used for this purpose.

Now, investigators in France report on the disease outcomes of 282 patients with myelodysplastic syndrome (median age, 71; 61% men) who received azacitidine (75 mg/m²/day for 7 days every 28 days). Patients received a median 6 cycles of therapy; 28% received a reduced dose, mainly to avoid weekend inj…