Introduction: Real-world studies of lower-risk myelodysplastic syndromes myelodysplastic syndromes: (my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or development. People with MDS have low blood cell count for at… (LR-MDS) are limited. We evaluated treatment patterns, clinical outcomes, and healthcare resource utilization (HCRU) among patients with LR-MDS treated with erythropoiesis-stimulating agents (ESAs) in the United States.
Patients and methods: This retrospective study included patients with LR-MDS who initiated treatment with ESAs between January 1, 2016 and June 30, 2019. The primary analysis assessed patient demographic and clinical characteristics, treatment patterns, clinical outcomes (hematologic response, transfusion requirements, disease progression), and HCRU (medical encounters, laboratory tests, and medication use). Subgroup analyses of patients repeatedly treated with ESA therapy evaluated selected clinical outcomes and primary ESA failure by SF3B1 mutational status, per recently updated NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines©).
Results: A total of 142 patients were included with a median follow-up time of 17 months (interquartile range [IQR], 7-33). Median age at ESA initiation was 79 years (IQR, 73-85). Patients were predominantly male (54%), overweight or obese (32% and 23%, respectively), of White race (96%) and non-Hispanic ethnicity (89%). Overall, 57% patients were initially treated with darbepoetin alfa darbepoetin alfa: It is in a class of medicines called erythropoiesis-stimulating agents (ESAs). Darbepoetin alfa can help improve red blood cell counts in bone marrow failure patients whose natural erythropoietin levels are low. It is given by injection under the skin (subcutaneous) or in the vein (intravenous)… and 43% with epoetin alfa epoetin alfa: Epoetin alfa can help improve red blood cell counts in bone marrow failure disease patients whose natural erythropoietin levels are low. It is given by injection under the skin (subcutaneous) or in the vein (intravenous). Epoetin alfa is approved by the U.S. Food and Drug Administration (FDA) for… . Clinical outcomes were poor, and there was a significant burden on both the health system and individual patients treated with ESA therapies. Hematologic improvement- erythroid was only seen in 26% of 142 patients treated with ESAs, and 65% of 82 retreated patients experienced primary ESA failure.
Conclusion: Our results indicate that primary ESA failure is largely unrecognized and that many patients should be considered for alternative treatments.
Keywords: Clinical Outcomes; Erythropoiesis-Stimulating Agents; Healthcare Resource Utilization Outcomes; Lower-Risk Myelodysplastic Syndromes.