Real-world impact of luspatercept on red blood cell transfusions among patients with myelodysplastic syndromes: A United States healthcare claims database study

Abstract

Myelodysplastic syndromes (MDS) are associated with anemia and the need for blood transfusions. In clinical trials, luspatercept reduced transfusion dependency among patients with lower-risk MDS. This United States (US) study describes real-world clinical outcomes pre- and post-luspatercept initiation among patients with MDS. Symphony Health Integrated Dataverse claims data (August 1, 2010−December 29, 2022) were used to identify patients with MDS treated with luspatercept (first luspatercept claim = index date). Transfusion-dependent (TD) or non-TD (NTD) patients at baseline were described as 8-week transfusion-independent (TI) or as maintaining NTD, respectively, if they had no transfusion for 8 weeks in the 6 months post-index (similarly for 12, 16, and 24 weeks). Transfusion status was measured overall and among patients who were baseline NTD, TD, TD and exposed to erythropoiesis-stimulating agents (ESA) (TD+ESA-exposed), MDS with ring sideroblasts (RS) (MDS-RS), and MDS-non-RS. MDS-related treatments were measured pre- and post-index. Among the 871 patients who received luspatercept (mean age: 74.7 years), 87.4 % achieved 8-week TI/maintained NTD within 6 months post-index, 64.9 % of patients did not receive additional MDS-related treatments post-luspatercept initiation (median follow-up: 14.8 months), 98.5 % of baseline NTD patients maintained 8-week NTD 6 months post-luspatercept initiation, and 88.6 % did not receive a transfusion for 24 weeks. Baseline TD (64.2 %) and TD+ESA-exposed (64.2 %) patients achieved 8-week TI 6 months post-luspatercept initiation. Eight-week TI proportions were similar between MDS-RS (89.8 %) and MDS-non-RS (84.8 %) subgroups. These findings corroborate clinical trial data by showing the high effectiveness of luspatercept among real-world patients with MDS in the US. • Anemia-related burden is high for patients with myelodysplastic syndromes. • Luspatercept reduced transfusion dependency and limited use of other medications. • Most transfusion-dependent patients achieved 8-week transfusion-independence. • Luspatercept had high effectiveness regardless of patient study baseline status. • These real-world findings corroborate pivotal clinical trial results.