Real-World Evidence on Treatment Utilization in Lower-Risk Myelodysplastic Syndromes: Findings from a Medical Record Review in the United States
Authors: Germing Ulrich , Goyal Ravi K. , Yucel Aylin , Parikh Rohan C. , Jimenez Maria , Sluga-O'Callaghan Martina , Tang Derek , Hughes Christina , Diez-Campelo Maria 
Published: November 2021

Abstract
Introduction: Patients diagnosed with myelodysplastic syndromes (MDS) are at an increased risk for developing infections, bleeding events, cardiopulmonary complications, and progressing to acute myeloid leukemia (AML). Patients with lower-risk MDS experience a mean life-year loss of about 6 years, and the main focus of treatment is on management of cytopenias, in particular anemia. Currently, limited real-world evidence exists on prevailing treatment patterns and outcomes in lower-risk MDS in the United States (US).
Methods: In this retrospective, non-interventional review of medical records, eligible patients (≥ 18 years of age) with diagnosis of lower-risk MDS (with or without ring sideroblasts and with single-lineage or multilineage dysplasia) between July 1, 2013 and September 30, 2018, were identified by participating clinicians. Patients with prior history of AML or other malignant neoplasms were excluded. This is an ongoing study in the US and interim data are described. Study measures descriptively summarized patient demographics, clinical characteristics at MDS diagnosis, and utilization of medication treatments for the management of anemia.
Results: Data from medical records of 50 patients with lower-risk MDS were abstracted by 26 hematologist-oncologists in the US. Participating clinicians had been managing treatment for hematology/oncology patients for an average (standard deviation [SD]) of 16.3 (6.7) years, 53.9% practiced in an academic hospital or cancer center, and 46.2% practiced in a community setting. The mean (SD) age for patients with lower-risk MDS at diagnosis was 64.2 (9.9) years, 64.0% were male, 70.0% were White, and 60% were insured primarily through Medicare. Most patients (54.0%) had Low-risk MDS (as categorized by Revised International Prognostic Scoring System) followed by Very low-risk (28.0%), and Intermediate-risk (18.0%) at initial diagnosis. The most common gene mutations observed were JAK2 (22.0%) and ASXL1 (8.0%). The most common karyotype abnormalities observed were del(5q) (28.0%), −Y (14.0%), and del(7q) (12.0%). During the 12 months before diagnosis of lower-risk MDS, cardiac complications were observed among 18.0% of patients, and 58.0% of patients were either current or former smokers at the time of diagnosis of lower-risk MDS. At the time of data abstraction, mean (SD) follow-up time was 39.6 (21.6) months, and 46 (92.0%) patients had received ≥ 1 line of treatment for MDS-associated anemia. Of these 46 patients, 93.5% received first-line treatment with an erythropoietin-stimulating agent (ESA). In the first-line, most patients received ESA monotherapy (n = 37; 80.4%) followed by ESA-based combination therapy (n = 6; 13.0%), lenalidomide only (n = 2; 4.4%), and azacitidine (n = 1; 2.2%). All 18 patients who received a second-line treatment had received an ESA-based first-line treatment. Second-line treatments received were ESA-based treatment (n = 5; 27.8%), lenalidomide only (n = 5; 27.8%), luspatercept only (n = 4; 22.2%), azacitidine only (n = 3; 16.7%), and filgrastim only (n = 1; 5.6%). Third-line treatment was only observed in 3 patients.
Conclusions: In this ongoing study, current analysis for 50 patients with lower-risk MDS in the US showed ESA-based regimens were the most common first-line therapy. ESA-based treatment was again utilized as second-line therapy among some patients who were previously treated with ESA.

Disclosures
Germing: Janssen: Honoraria; Celgene: Honoraria; Novartis: Honoraria, Research Funding; Jazz Pharmaceuticals: Honoraria; Bristol-Myers Squibb: Honoraria, Other: advisory activity, Research Funding. Goyal: RTI Health Solutions: Other: Full-time employee of RTI Health Solutions, which is business unit of RTI International, a non-profit research organization, which received funding from BMS to conduct this study.. Yucel: BMS: Current Employment, Current holder of individual stocks in a privately-held company. Parikh: RTI-Health Solutions: Other: Full-time employee of RTI-HS, which received funding to conduct this study from BMS.. Jimenez: RTI Health Solutions: Current Employment. Sluga-O'Callaghan: RTI Health Solutions: Current Employment. Tang: Bristol-Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Hughes: BMS: Current Employment. Diez-Campelo: Takeda Oncology: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; BMS: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau.