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RBC transfusion independence among lower risk MDS patients receiving hypomethylating agents: a population-level

Amer M Zeidan1,2, Weiwei Zhu2,3, Maximilian Stahl1,2,4

  • 1Department of Internal Medicine, Section of Hematology, Yale School of Medicine, New Haven, CT, USA.

Leukemia & Lymphoma
|June 8, 2019
PubMed

Insights

Hypomethylating agents (HMAs) help some lower-risk myelodysplastic syndromes (LR-MDS) patients achieve red blood cell (RBC) transfusion independence (TI). However, achieving TI with HMAs varies, and frequent transfusions before treatment reduce success rates.

Area of Science:

  • Hematology
  • Oncology
  • Clinical Effectiveness Research

Background:

  • Lower-risk myelodysplastic syndromes (LR-MDS) frequently lead to red blood cell (RBC) transfusion dependence.
  • Hypomethylating agents (HMAs) are a common treatment, but their effectiveness in achieving transfusion independence (TI) is inconsistently reported.

Purpose of the Study:

  • To evaluate the real-world clinical effectiveness of HMAs in inducing RBC TI in anemic LR-MDS patients.
  • To provide population-level estimates of HMA effectiveness for RBC TI in LR-MDS.

Main Methods:

  • Utilized the Surveillance, Epidemiology, and End Results (SEER)-Medicare database for real-world data analysis.
  • Analyzed outcomes for LR-MDS patients receiving RBC transfusions or dependent on them at HMA initiation.

Main Results:

  • Approximately 40% of LR-MDS patients receiving RBC transfusions and 33% dependent on transfusions at HMA initiation achieved TI.
  • Receiving three or more RBC transfusions in the eight weeks prior to HMA initiation was linked to lower odds of achieving TI.

Conclusions:

  • HMAs demonstrate variable effectiveness in achieving RBC TI in LR-MDS patients in a real-world setting.
  • Pre-treatment transfusion burden is a significant factor associated with reduced likelihood of achieving HMA-induced TI.
Keywords:
HMAMDSazacitdinedecitabinetransfusion

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