Real-World Treatment Patterns, Clinical Outcomes, and Costs in Patients with Higher-Risk Myelodysplastic Syndromes Across France, Germany, and the United Kingdom.

IF 2.1 Q3 HEMATOLOGY
Journal of Blood Medicine Pub Date : 2025-06-25 eCollection Date: 2025-01-01 DOI:10.2147/JBM.S516558
Mark Drummond, Carlo Finelli, Fjoralba Kristo, Sneha S Kelkar, Shelby Corman, Rutika Raina, Ajibade Ashaye, Mehul Dalal, Detlef Haase
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引用次数: 0

Abstract

Background: Higher-risk myelodysplastic syndromes (HR-MDS) are associated with increased progression to acute myeloid leukemia (AML) and poor prognosis.

Patients and methods: This chart review characterizes real-world treatment patterns, outcomes, and costs of HR-MDS in France, Germany, and the United Kingdom (UK). Treating oncologists collected data (01 January 2014-31 December 2016) for adult patients with HR-MDS (revised International Prognostic Scoring System [IPSS-R] score >3), who received first-line treatment (1LOT) and had ≥1 year follow-up post diagnosis or until death. Demographics, clinical characteristics, treatment patterns, outcomes, and healthcare resource use were collected during 1LOT. Kaplan-Meier methods were used for time-to-event outcomes. Costs, applied to resource use, were calculated through 1LOT.

Results: Forty-one physicians provided data for 95 patients (France, n=31; Germany, n=29; UK, n=35). At HR-MDS diagnosis, median patient age was 75 years, 62.1% were men, and 60.0% had very high-risk disease per the IPSS-R. Median follow-up was 34.5 months. In 1LOT, 89.5% of patients received azacitidine (median, 12.0 cycles). At the end of 1LOT, 24.2% of patients had a complete and 30.5% a partial remission. From start of 1LOT, median progression-free survival was 24.3 months. Overall survival (unadjusted) was 32.9 months in all patients and shorter in the 33.7% of patients with versus without AML transformation (17.0 vs 52.9 months). Costs for 1LOT were driven by adjunctive therapy and were higher for patients who were transfusion-dependent versus -independent at the start of therapy and who did versus did not have transformation to AML.

Conclusion: These results provide real-world data from France, Germany, and the UK on HR-MDS treatment patterns, clinical outcomes, and costs.

法国、德国和英国高风险骨髓增生异常综合征患者的现实世界治疗模式、临床结果和成本
背景:高风险骨髓增生异常综合征(HR-MDS)与急性髓系白血病(AML)进展加快和预后不良相关。患者和方法:这张图表回顾了现实世界中法国、德国和英国HR-MDS的治疗模式、结果和成本。治疗肿瘤学家收集的数据(2014年1月1日至2016年12月31日)为接受一线治疗(1LOT)的HR-MDS(修订的国际预后评分系统[IPSS-R]评分bbbb3)的成年患者,这些患者在诊断后或死亡后随访≥1年。 在1LOT期间收集了人口统计学、临床特征、治疗模式、结果和医疗资源使用情况。Kaplan-Meier方法用于时间-事件结果。应用于资源使用的成本是通过1LOT来计算的。结果:41位医生为95例患者提供了资料(法国,n=31;德国,n = 29;英国,n = 35)。在HR-MDS诊断时,患者中位年龄为75岁,62.1%为男性,根据IPSS-R, 60.0%为高危疾病。中位随访时间为34.5个月。在1LOT中,89.5%的患者接受阿扎胞苷治疗(中位数,12.0周期)。在1LOT结束时,24.2%的患者完全缓解,30.5%的患者部分缓解。从1LOT开始,中位无进展生存期为24.3个月。所有患者的总生存期(未经调整)为32.9个月,33.7%的AML转化患者比未转化患者的总生存期更短(17.0个月对52.9个月)。1LOT的成本是由辅助治疗驱动的,在治疗开始时依赖输血的患者与非依赖输血的患者以及转化为AML的患者与未转化为AML的患者的成本更高。结论:这些结果提供了来自法国、德国和英国关于HR-MDS治疗模式、临床结果和成本的真实数据。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
3.50
自引率
0.00%
发文量
94
审稿时长
16 weeks
期刊介绍: The Journal of Blood Medicine is an international, peer-reviewed, open access, online journal publishing laboratory, experimental and clinical aspects of all topics pertaining to blood based medicine including but not limited to: Transfusion Medicine (blood components, stem cell transplantation, apheresis, gene based therapeutics), Blood collection, Donor issues, Transmittable diseases, and Blood banking logistics, Immunohematology, Artificial and alternative blood based therapeutics, Hematology including disorders/pathology related to leukocytes/immunology, red cells, platelets and hemostasis, Biotechnology/nanotechnology of blood related medicine, Legal aspects of blood medicine, Historical perspectives. Original research, short reports, reviews, case reports and commentaries are invited.
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