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罗特西普对骨髓增生异常综合征患者红细胞输注的真实世界影响:一项美国医疗保健索赔数据库研究。

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

作者信息

Andritsos Leslie A, McBride Ali, Tang Derek, Barghout Victoria, Zanardo Enrico, Song Rui, Huynh Lynn, Yenikomshian Mihran, Makinde Adeola Y, Hughes Christina, Hanna Kirollos S, Patel Kashyap

机构信息

The University of New Mexico, Albuquerque, NM, USA.

Bristol Myers Squibb, Princeton, NJ, USA.

出版信息

Leuk Res. 2025 Jan;148:107624. doi: 10.1016/j.leukres.2024.107624. Epub 2024 Nov 17.

DOI:10.1016/j.leukres.2024.107624
PMID:39602855
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.

摘要

骨髓增生异常综合征(MDS)与贫血及输血需求相关。在临床试验中,罗特西普降低了低危MDS患者的输血依赖。这项美国研究描述了MDS患者在开始使用罗特西普之前和之后的真实世界临床结局。利用Symphony Health综合数据集的理赔数据(2010年8月1日至2022年12月29日)来识别接受罗特西普治疗的MDS患者(首次罗特西普理赔日期 = 索引日期)。如果基线时的输血依赖(TD)或非输血依赖(NTD)患者在索引日期后的6个月内有8周未输血(12周、16周和24周同理),则分别描述为8周无输血依赖(TI)或维持NTD。总体上以及在基线时为NTD、TD、TD且暴露于促红细胞生成素(ESA)(TD + ESA暴露)、伴有环形铁粒幼细胞(RS)的MDS(MDS - RS)和非RS的MDS患者中测量输血状态。在索引日期之前和之后测量MDS相关治疗。在871例接受罗特西普治疗的患者中(平均年龄:74.7岁),87.4%在索引日期后的6个月内实现了8周TI/维持NTD,64.9%的患者在开始使用罗特西普后未接受额外的MDS相关治疗(中位随访时间:14.8个月),98.5%的基线NTD患者在开始使用罗特西普后的6个月内维持8周NTD,88.6%的患者在24周内未接受输血。基线时的TD(64.2%)和TD + ESA暴露(64.2%)患者在开始使用罗特西普后的6个月内实现了8周TI。MDS - RS(89.8%)和非RS的MDS(84.8%)亚组之间的8周TI比例相似。这些发现通过显示罗特西普在美国真实世界的MDS患者中具有高效性,证实了临床试验数据。

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