Azacitidine Reduces Transfusions and Costs for Patients with High-Risk MDS

Caroline Helwick

January 2013, Vol 4, No 1 - Health Economics


Atlanta, GA—The use of azacitidine (Vidaza) in patients with high-risk myelodysplastic syndrome (MDS) is associated with the reduced need for red blood cell (RBC) transfusion and transfusion dependence, a report from the 2012 American Society of Hematology meeting showed.

“At 12 and 18 months after azacitidine treatment, there were 26% and 38% reductions in RBC transfusion costs, respectively, per patient compared with the 6 months before therapy,” said Eric Tseng, MD, Department of Hematology, University of Toronto, Ontario, Canada.

In 2009, azacitidine was shown in the AZA001 study to improve overall survival, leukemia-free survival, and hematologic response, while reducing transfusion dependence in intermediate- and high-risk MDS, compared with conventional care. Transfusion dependence in patients with MDS is associated with greater health resource utilization and high transfusion-related costs in the inpatient and the outpatient settings. It is also associated with increased mortality, Dr Tseng said.

“Hypomethylating agents, including azacitidine, have been shown to be cost-effective in treating high-risk MDS,” he said, “but there is a lack of studies assessing the effect of azacitidine on transfusion requirements and transfusion-related costs in the real-world clinical setting.”
Using real-world data, Dr Tseng and colleagues retrospectively reviewed data of 51 patients with MDS who were treated with the approved dose and schedule of azacitidine at their tertiary care center between 2008 and 2012.

The cost per unit of RBCs was determined to be $1183 (2008 US dollars) and $1273 (2012 Canadian dollars). Transfusion costs per patient were determined for 6-month intervals before and after the initiation of azacitidine.
Substantial Decline in Transfusions and Costs

“The number of units transfused decreased in both the inpatient and outpatient settings after treatment with azacitidine was initiated,” Dr Tseng added. The number of RBC transfusions and costs declined sub­stantially between baseline and 12-
month to 18-month follow-up (shown in Canadian dollars):

  • 6 months before azacitidine:
    11 RBC units transfused; cost, $14,048
  • 0 to 6 months after azacitidine:
    10.9 RBC units; cost, $13,898
  • 6 to 12 months after azacitidine:
    8.2 RBC units; cost, $10,407
  • 12 to 18 months after azacitidine:
    6.9 RBC units; cost, $8744.

The patients in this study had similar response rates with azacitidine as those in the AZA001 trial, which were robust in approximately 66% of patients. The median number of cycles was 11, and the median time to best response was 6 months.

Overall, 63% became transfusion independent within 18 months of the initiation of azacitidine. Median time to transfusion independence was 3 months, Dr Tseng reported.