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Leuk Lymphoma. 2019 Jun 7:1-7. doi: 10.1080/10428194.2019.1622700. [Epub ahead of print]

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

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a Department of Internal Medicine, Section of Hematology , Yale School of Medicine , New Haven , CT , USA.
b Cancer Outcomes, Public Policy, and Effectiveness Research (COPPER) Center , Yale University , New Haven , CT , USA.
c Department of Chronic Disease Epidemiology, School of Public Health , Yale University , New Haven , CT , USA.
d Department of Medicine, Section of Hematologic Malignancies , Memorial Sloan Kettering Cancer Center , New York , NY , USA.
e Department of Health Policy and Management , School of Public Health, Yale University , New Haven , CT , USA.


Most patients with lower risk myelodysplastic syndromes (LR-MDS) become red blood cell (RBC) transfusion dependent at some time during their disease course. Hypomethylating agents (HMAs) are frequently used in this setting; however, reported rates of in RBC transfusion independence (TI) achieved with HMA therapy vary significantly between studies. Here we study the real-life clinical effectiveness of HMA in inducing RBC TI in anemic LR-MDS patients using the Surveillance, Epidemiology and End Results (SEER)-Medicare database. We find that approximately 40% of LR-MDS patients who were receiving RBC transfusions and 33% who were dependent on RBC transfusions at HMA initiation ultimately achieved TI. The receipt of ≥3 transfusions in the 8-week period before HMA initiation was significantly associated with lower odds of achieving TI. Our study provides important population level estimates of clinical effectiveness of HMAs in LR-MDS.


HMA; MDS; azacitdine; decitabine; transfusion

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