Luspatercept 对骨髓增生异常综合征患者输注红细胞的实际影响:美国医疗索赔数据库研究

IF 2.1 4区 医学 Q3 HEMATOLOGY Leukemia research Pub Date : 2024-11-17 DOI:10.1016/j.leukres.2024.107624
Leslie A. Andritsos , Ali McBride , Derek Tang , Victoria Barghout , Enrico Zanardo , Rui Song , Lynn Huynh , Mihran Yenikomshian , Adeola Y. Makinde , Christina Hughes , Kirollos S. Hanna , Kashyap Patel
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引用次数: 0

摘要

骨髓增生异常综合征(MDS)与贫血和输血需求有关。在临床试验中,Luspatercept降低了低风险MDS患者的输血依赖性。这项美国研究描述了 MDS 患者在开始使用 luspatercept 前后的实际临床结果。Symphony Health Integrated Dataverse理赔数据(2010年8月1日-2022年12月29日)被用于识别接受鲁帕特罗治疗的MDS患者(首次鲁帕特罗理赔=索引日期)。如果基线时的输血依赖(TD)或非输血依赖(NTD)患者在索引后的 6 个月内 8 周(12、16 和 24 周类似)没有输血,则将其分别描述为 8 周输血无关(TI)或维持 NTD。输血状态的测量对象包括整体患者以及基线 NTD、TD、TD 并暴露于红细胞生成刺激剂(ESA)(TD+ESA-exposed)、MDS 伴有环形红细胞(RS)(MDS-RS)和 MDS 非 RS 患者。对指数前后的 MDS 相关治疗进行了测量。在接受鲁帕特罗治疗的871名患者中(平均年龄:74.7岁),87.4%的患者在指标发布后6个月内达到8周TI/维持NTD,64.9%的患者在鲁帕特罗治疗后未接受额外的MDS相关治疗(中位随访时间:14.8个月),98.5%的基线NTD患者在鲁帕特罗治疗后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TD(64.2%)和TD+ESA暴露(64.2%)患者在开始接受Luspatercept治疗6个月后实现了8周TI。MDS-RS亚组(89.8%)和MDS-非RS亚组(84.8%)的8周TI比例相似。这些研究结果证实了临床试验数据,显示了luspatercept在美国MDS实际患者中的高度有效性。
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Real-world impact of luspatercept on red blood cell transfusions among patients with myelodysplastic syndromes: A United States healthcare claims database study
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.
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来源期刊
Leukemia research
Leukemia research 医学-血液学
CiteScore
4.00
自引率
3.70%
发文量
259
审稿时长
1 months
期刊介绍: Leukemia Research an international journal which brings comprehensive and current information to all health care professionals involved in basic and applied clinical research in hematological malignancies. The editors encourage the submission of articles relevant to hematological malignancies. The Journal scope includes reporting studies of cellular and molecular biology, genetics, immunology, epidemiology, clinical evaluation, and therapy of these diseases.
期刊最新文献
Recent progress in pathological understanding of adult T-cell leukemia/lymphoma in the new classification era. Immunological aspects of HTLV-1 persistence; for the prevention and treatment of Adult T-cell leukaemia-lymphoma (ATL). Editorial Board Predictive modeling of outcomes in acute leukemia patients undergoing allogeneic hematopoietic stem cell transplantation using machine learning techniques Real-world impact of luspatercept on red blood cell transfusions among patients with myelodysplastic syndromes: A United States healthcare claims database study
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