Abstract

Background: The median survival for patients with lower-risk myelodysplastic syndromes (LR-MDS) is estimated to be 5–10 years. Erythropoiesis-stimulating agents (ESAs) are utilized as first-line treatment for anemia in LR-MDS; however, real-world treatment patterns, clinical outcomes, and healthcare resource utilization (HCRU) for patients with LR-MDS who reinitiated ESAs following previous discontinuation are not well-established. Aims: To examine treatment patterns, clinical outcomes, and HCRU in patients with LR-MDS who reinitiated treatment with ESAs. Methods: This was a retrospective study of patients with LR-MDS in a network of US community health systems who initiated ESAs as first-line therapy between January 1, 2016 and June 30, 2019 and were followed through June 30, 2021. Patients were required to have discontinued (had a ≥ 3-week gap between treatment with epoetin alfa, or a ≥ 6-week gap between treatment with darbepoetin alfa) and subsequently reinitiated ESA therapy at least once during follow-up. Outcomes included failure to achieve transfusion independence (TI), progression to acute myeloid leukemia (AML), overall survival (OS), number and type of health system visits, and medication use. TI was defined per IWG 2006 criteria in the subset of patients who had received ≥ 1 transfusion in the 8 weeks prior to ESA initiation. Results: There were 108 patients with confirmed LR-MDS who initiated ESA-based therapy. Patients had a median age of 79 years at diagnosis (interquartile range [IQR] 73–85), were predominantly male (58%), White (97%), and overweight or obese (52%). The median follow-up period was 17.0 months (IQR 7.2–34.7). The most used ESA was darbepoetin alfa (60%), followed by epoetin alfa (38%) and epoetin alfa-epbx (2%). Of the 33 patients who received a baseline transfusion, 52% did not achieve TI; 53% and 43% failed to achieve TI at 6 and 12 months, respectively. The majority of patients were repeatedly treated with ESAs (n = 61, 56%). Among these patients, 2% progressed to AML and 48% died during follow-up; median OS was 45 months. The median number of health system visits per patient-month was 3.2 (IQR 0.6–4.9); 89%, 62%, and 72% of pts had ≥ 1 outpatient, emergency department, or inpatient visit respectively, with median length of hospitalization of 12 days (IQR 4.8–27.8). Most patients (54%) received an antibiotic at least once during follow-up; overall, use of immunosuppressive therapy (2%) or iron chelation (3%) was rare. Summary/Conclusion: In this real-world study of community practice in the USA, reinitiation of ESA treatment after prior discontinuation in LR-MDS was prevalent. In these patients, poor clinical outcomes and frequent health system visits were observed. This study highlights the need to consider alternative treatment options for patients with LR-MDS.

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