Diez-Campelo M, Yucel A, Goyal R, Parikh R, Dhuliawala S, Jimenez M, Sluga-O'Callaghan M, Hughes C, Tang D, Germing U. Systemic therapy utilization and hematologic outcomes in lower-risk myelodysplastic syndromes (LR-MDS): findings from a real-world medical record review study in the US, UK, and Europe (EU). Poster presented at the 2022 European Hematology Association Hybrid Congress; June 10, 2022. Vienna, Austria.

BACKGROUND: Patients (pts) with LR-MDS experience a mean life-year loss of 6 years, and the treatment focus is improvement of quality of life and prolongation of life expectancy.

AIMS: This retrospective medical record review assessed real-world data to determine prevailing LR-MDS treatment patterns and hematologic outcomes.

METHODS: Eligible pts (≥18 years of age) with LR-MDS (Jul 1, 2013–Sep 30, 2018) were identified by participating hematologist-oncologists in the US, Canada (CAN), UK, and EU (France, Germany, Spain). Study measures and hematologic outcomes were descriptively assessed (data abstraction Jun–Nov 2021). Aggregated analyses used pooled US/CAN and UK/EU data sets for the overall population and pt subgroups treated with clinician-defined first-line (1L) and second-line (2L) erythropoiesis-stimulating agents (ESAs)

RESULTS: Medical record data were abstracted by 114 US/CAN (n=174 pts; median age 60.6 years; 68.4% male) and 166 UK/EU clinicians (n=319 pts; median age 67.1 years; 72.4% male). Median follow-up duration from MDS diagnosis was 60.6 months (US/CAN) and 67.6 months (UK/EU). Approximately half of the pts had “low” risk status (49.4% US/CAN; 53.9% UK/EU) categorized by the Revised International Prognostic Scoring System at initial diagnosis. The most common karyotype abnormality was del(5q) (27.6% US/CAN; 20.4% UK/EU). SF3B1 mutation was recorded for 7.5% US/CAN and 8.2% UK/EU pts. Almost all pts had received ≥1 line of systemic treatment for managing MDS-associated anemia (US/CAN: 165 [94.8%] 1L, 77 [44.3%] 2L; UK/EU: 294 [92.2%] 1L, 133 [41.7%] 2L). In 1L, ESA-based regimens were the most common treatment with median duration of therapy of ≥2 years (US/CAN: 142 [86.1%], of which the majority (80.3%) was ESA monotherapy; UK/EU: 264 [89.8%], comprising 82.2% ESA monotherapy). Treatment characteristics and outcomes are shown in the Table. Among pts who were transfusion dependent (TD) before 1L ESA, a very small proportion achieved transfusion independence (TI) or red blood cell transfusion reduction ≥50%, which was maintained for a median of <1.5 years. Among the 1L ESA-treated pts who were TD, TI was achieved by 5.7% (US/CAN) and 12.9% (UK/EU). In 2L ESA-treated pts (n=23 US/CAN; n=46 UK/EU), almost all were previously treated with a 1L ESA-containing regimen (95.7% US/CAN; 84.8% UK/EU). The most common reasons for reinitiating ESA in 2L (n=22, 15.5% US/CAN; n=39, 14.8% UK/EU) were overall health status (40.9% US/CAN; 33.3% UK/EU), treatment efficacy (40.9% US/CAN; 48.7% UK/EU), and compliance with national guidelines (13.6% US/CAN; 46.2% UK/EU). Among the pts who reinitiated ESA in 2L, the most common reasons for discontinuation of 1L ESA were completion of planned course of therapy (45.5% US/CAN; 28.2% UK/EU), pt decision (40.9% US/CAN; 20.5% UK/EU), progressive disease (36.4% US/CAN; 51.3% UK/EU), and adverse events (13.4% US/CAN; 15.4% UK/EU). Lenalidomide was used in 1L therapy among 8.5% pts in US/CAN and 5.8% in UK/EU. Among those with del(5q) mutation, ESA was used in 1L therapy among 83.3% pts in US/CAN and 87.7% in UK/EU.

CONCLUSIONS: ESA-based regimens were the most common treatment for anemia management in pts with LR-MDS; >1/3 of pts were TD before initiating 1L ESA therapy. A considerable proportion of 1L ESA-treated pts reinitiated ESA in 2L despite the majority having discontinued 1L treatment due to planned therapy completion, pt decision, or progressive disease. This real-world study suggests that there is an unmet need in addressing anemia burden with ESAs, and novel and effective treatments are needed.

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