TRANSFUSION BURDEN IN PATIENTS WITH LOWER-RISK MYELODYSPLASTIC SYNDROMES IN FRANCE SINCE 202_THE MYOSOTYS STUDY
Author(s)
Thibaut Comont, MCU_PH1, Jerome Fernandes, DR2, Nicolas Molinari, MCU-PH3, FRANCOIS-EMERY COTTE, MA, MPH, PharmD, PhD4, Caroline Guilmet, PharmaD5, Mallory Cals-Maurette, MSc6, Aurélie Schmidt, MSc6, Victor Morilhas, PharmaD7, Melanie Chartier, MSc8, Maud D' Aveni?piney, MCU-PH9.
1Université de toulouse, CHU de Toulouse, Toulouse, France, 2centre hospitalier de la côte basque, BAYONNE CEDEX, France, 3CHU Montpellier, Montpellier, France, 4Bristol Myers Squibb, RUEIL MALMAISON, France, 5BMS, Reuil-Malmaison, France, 6HEVA, Lyon, France, 7Medical, Bristol Myers Squibb, Rueil Malmaison, France, 8HEOR, Bristol Myers Squibb, Rueil Malmaison, France, 9hematology, CHRU Nancy, Vandoeuvre-Les-Nancy, France.
1Université de toulouse, CHU de Toulouse, Toulouse, France, 2centre hospitalier de la côte basque, BAYONNE CEDEX, France, 3CHU Montpellier, Montpellier, France, 4Bristol Myers Squibb, RUEIL MALMAISON, France, 5BMS, Reuil-Malmaison, France, 6HEVA, Lyon, France, 7Medical, Bristol Myers Squibb, Rueil Malmaison, France, 8HEOR, Bristol Myers Squibb, Rueil Malmaison, France, 9hematology, CHRU Nancy, Vandoeuvre-Les-Nancy, France.
OBJECTIVES: In patients with lower-risk myelodysplastic syndromes (LR-MDS), treatment mainly aims to correct cytopenias, particularly chronic anemia. First-line treatment relies on erythropoiesis-stimulating agents (ESAs) to reduce transfusion needs, but their efficacy remains limited and declines over time. Luspatercept was approved in the EU in 2020 as a second-line option for a subset of patients with ring sideroblasts. This study aimed to describe the management of adults with LR-MDS in France since 2020, with a focus on luspatercept use under current French availability conditions.
METHODS: This retrospective study used claims data from the French National Health Data System (SNDS), covering outpatient and hospital care. Adults with LR-MDS initiating first-line treatment for anemia between 2020 and 2024 were included and followed until death, end of study, or loss to follow-up. Transfusion response to luspatercept at 24 weeks was assessed using adapted 2018 IWG transfusion criteria, according to transfusion dependence at initiation. Transfusion numbers and costs (from French National Health Insurance perspective, in €2024) were compared 48 weeks before and after luspatercept initiation.
RESULTS: Between 2020 and 2024, 27,003 incident patients initiated treatment for LR-MDS. Among them, 1,675 received luspatercept, 94.3% after prior ESA; 59% were male and median age was 78 years. After luspatercept initiation, 799 patients achieved a transfusion response. In this population, the mean number of transfusions decreased from 9.3 (±12.0) before initiation to 5.3 (±9.8) after initiation, corresponding to savings of €4,176 per patient over 48 weeks (cost per transfusion : €1,044). In addition, the proportion of patients with high-intensity transfusion need (≥6 transfusions over 48 weeks) decreased from 47.2% to 26.0%.
CONCLUSIONS: This study provides a real-world overview of LR-MDS management in France and the first nationwide evidence on luspatercept use in routine practice before its availability in first-line. Among responders, luspatercept was associated with reduced transfusion burden and lower transfusion intensity.
METHODS: This retrospective study used claims data from the French National Health Data System (SNDS), covering outpatient and hospital care. Adults with LR-MDS initiating first-line treatment for anemia between 2020 and 2024 were included and followed until death, end of study, or loss to follow-up. Transfusion response to luspatercept at 24 weeks was assessed using adapted 2018 IWG transfusion criteria, according to transfusion dependence at initiation. Transfusion numbers and costs (from French National Health Insurance perspective, in €2024) were compared 48 weeks before and after luspatercept initiation.
RESULTS: Between 2020 and 2024, 27,003 incident patients initiated treatment for LR-MDS. Among them, 1,675 received luspatercept, 94.3% after prior ESA; 59% were male and median age was 78 years. After luspatercept initiation, 799 patients achieved a transfusion response. In this population, the mean number of transfusions decreased from 9.3 (±12.0) before initiation to 5.3 (±9.8) after initiation, corresponding to savings of €4,176 per patient over 48 weeks (cost per transfusion : €1,044). In addition, the proportion of patients with high-intensity transfusion need (≥6 transfusions over 48 weeks) decreased from 47.2% to 26.0%.
CONCLUSIONS: This study provides a real-world overview of LR-MDS management in France and the first nationwide evidence on luspatercept use in routine practice before its availability in first-line. Among responders, luspatercept was associated with reduced transfusion burden and lower transfusion intensity.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
SA74
Topic
Clinical Outcomes, Epidemiology & Public Health, Study Approaches
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology