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在新型药物引入台湾新诊断多发性骨髓瘤之前和之后,治疗途径和疾病进程有所不同。

Treatment pathways and disease journeys differ before and after introduction of novel agents in newly diagnosed multiple myeloma in Taiwan.

机构信息

Global Epidemiology, Janssen Research and Development, Singapore, Singapore.

School of Health Care Administration, College of Management, Taipei Medical University, Taipei, Taiwan.

出版信息

Sci Rep. 2021 Jan 13;11(1):1112. doi: 10.1038/s41598-020-80607-4.

Abstract

Limited real-world data are available regarding treatment practices and outcomes of multiple myeloma (MM) in Asia. We conducted a retrospective cohort study using the Taiwan National Healthcare Insurance Research database and Taiwan Death Registry and used a Markov model to describe disease progression and outcomes in 4092 patients newly diagnosed with MM from 01-Jan-2007 to 31-Dec-2015. We observed marked differences in the characteristics, length and outcome of the clinical journey between patients who did/did not receive autologous stem cell transplant, and between patients initiated on novel agents versus other treatment regimens. In transplant recipients, initiation with combined thalidomide + bortezomib increased over time (12.2-77.5%). Progression-free survival after first-line treatment improved and a lower percentage of patients died. Lenalidomide in second and third-line regimens increased (15.5-31.5%). In non-transplanted patients, initiation with novel agents increased (17.5-54.6%), but death rates remained high (60.3%). The treatment landscape of MM in Taiwan has evolved, with increased use of combined bortezomib + thalidomide for first-line and lenalidomide for second/third-line but many patients die before receiving second-line treatment. Novel agents with different modes of action should be used as early as possible to maximize their benefits. Improved MM treatments remains a critical medical need.

摘要

关于亚洲多发性骨髓瘤(MM)的治疗实践和结局,仅有有限的真实世界数据。我们使用台湾全民健康保险研究数据库和台湾死亡登记处进行了一项回顾性队列研究,并使用马尔可夫模型描述了 2007 年 1 月 1 日至 2015 年 12 月 31 日期间 4092 例新诊断为 MM 的患者的疾病进展和结局。我们观察到,接受/未接受自体干细胞移植的患者、起始使用新型药物与其他治疗方案的患者之间,在特征、病程长短和临床结局方面存在显著差异。在接受移植的患者中,联合使用沙利度胺+硼替佐米的起始治疗比例随着时间的推移而增加(12.2-77.5%)。一线治疗后的无进展生存期得到改善,死亡患者比例降低。来那度胺在二线和三线方案中的应用增加(15.5-31.5%)。在未接受移植的患者中,起始使用新型药物的比例增加(17.5-54.6%),但死亡率仍然很高(60.3%)。台湾的 MM 治疗格局发生了变化,一线治疗中联合使用硼替佐米+沙利度胺的比例增加,二线/三线治疗中使用来那度胺的比例增加,但许多患者在接受二线治疗前死亡。具有不同作用机制的新型药物应尽早使用,以最大限度地发挥其效益。改善 MM 治疗仍然是一个关键的医疗需求。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/bac2/7806818/cf71ee6dbbd0/41598_2020_80607_Fig1_HTML.jpg

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