The MDS Beacon™

At the European Hematology Association 14^th Congress in Berlin, researchers from Australia showed high-risk myelodysplastic syndromes (MDS) patients receiving more treatment cycles of Vidaza (azacitidine) were more likely to become transfusion-independent and have longer overall survival.

Red blood cell (RBC) or platelet transfusions are types of conventional supportive care that are necessary for patients whose bone marrow is unable to make sufficient amounts of healthy cells. These courses of therapy, however, only treat symptoms caused by low blood cell counts, and do not cure MDS or prevent acute myeloid leukemia (AML) transformation.

The study monitored 179 high-risk MDS patients with refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-t), chronic myelomonocytic leukemia (CMML), and Intermediate-2 or High-risk MDS. Each patient received Vidaza (75 mg/m²) subcutaneously for daily for seven days without additional red blood cell production stimulants. The treatment cycle was repeated after 28 days.

Transfusion dependence at the beginning of the treatment and transfusion independence measured patients’ responses to Vidaza. These were defined as requiring more than one transfusion during the 28 days prior to receiving Vidaza, and maintaining a transfusion-free status for 56 days or longer, respectively.

Patients initially RBC transfusion-dependent who achieved transfusion independence during treatment received a median of 14 cycles, while those who continued to need RBC transfusions throughout the study received four cycles. Likewise, platelet transfusion-dependent patients who became transfusion independent received 11 cycles, compared to a median of three cycles for continually platelet-dependent patients.

Results showed all patients who achieved transfusion independence at some point during treatment received Vidaza for a longer period of time and had longer overall survival than patients who did not. Forty-five percent (50 out of 111) of patients who were RBC transfusion-dependent prior to treatment achieved transfusion independence, while 42 percent (16 out of 38) of patients needing platelet transfusions prior to treatment became transfusion independent. Transfusion-dependence reflects overall survival; thus, the median length of overall survival was extended for patients who received more Vidaza treatment cycles.

The study concluded patients who received Vidaza for a longer period of time had longer median overall survival and were more likely to become or remain independent of RBC or platelet transfusions.

For more information, see abstract 0813 from the “Myelodysplastic syndromes” session of the 14^th Congress of the EHA.