Treatment patterns and outcomes in patients with lower-risk myelodysplastic syndromes treated with erythropoiesis-stimulating agents in a large US community oncology practice: a retrospective chart review.

Background: Erythropoiesis-stimulating agents (ESAs) are an established treatment for lower-risk myelodysplastic syndromes (LR-MDS). However, real-world data on the outcomes of patients with LR-MDS treated with ESAs are limited. This study describes treatment patterns and clinical outcomes in this population.

Methods: This is a retrospective review of electronic medical records compiled from Florida Cancer Specialists & Research Institute databases. Inclusion criteria were: LR-MDS diagnosis (between January 2018 through December 2022), initiation of ESA treatment, ≥18 years at diagnosis, availability of ≥6 months of data prior to diagnosis, and ≥1 year of follow-up data. Patients treated with luspatercept prior to ESAs or enrolled in clinical trials during the study period were excluded. Patients were followed for 1 year from the initial diagnosis until loss to follow-up/death. Outcomes included treatment patterns (occurrence of and time to ESA failure, and time on ESA treatment after failure), and clinical outcomes related to ESA failure (hematologic improvement and transfusion independence), with ESA failure defined as  < 1.5 g/dL rise in hemoglobin or no decrease in red blood cell transfusions by 6-8 weeks of treatment.

Results: At baseline, of 359 eligible patients with LR-MDS most were male (65.2%), White (88.0%), non-transfusion dependent (74.9%), and had hemoglobin levels of 8-10 g/dL (56.3%) and ring sideroblasts <5% (59.3%). Most patients (68.0%) experienced ESA failure, with a median of 56.0 days from ESA initiation to failure, and a median of 466.5 days receiving ESA treatment after failure. Hematologic improvement was observed in 33.7% of patients.

Conclusions: In this real-world evaluation of ESA treatment in a population with LR-MDS, only one-third of patients experienced hematologic improvement and two-thirds of patients remained on ESA treatment for ≥1 year after failure. These results demonstrate high rates of ESA failure and indicate patients in community oncology settings in the US may be continuing with ESA treatment despite failure.