Clinical Outcomes and Healthcare Resource Utilization for Patients with Lower-Risk Myelodysplastic Syndromes Treated with Erythropoiesis-Stimulating Agents

Introduction Real-world studies of lower-risk myelodysplastic syndromes (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 1 January 2016 and 30 June 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, 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 and 43% with epoetin alfa. 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. Micro Abstract There are limited studies describing clinical outcomes and healthcare resource utilization in patients with lower-risk myelodysplastic syndromes (LR-MDS) treated with erythropoiesis-stimulating agents (ESAs). This analysis of 142 patients with LR-MDS treated with ESAs showed poor clinical outcomes and a substantial burden on patients and the health system. Most ESA-treated patients with LR-MDS did not achieve hematologic improvement and alternative treatments should be considered.