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多发性骨髓瘤患者的长期预后:基于荷兰人群的血液学观察性研究登记系统(PHAROS)的回顾性分析

Long-term Outcomes in Patients With Multiple Myeloma: A Retrospective Analysis of the Dutch Population-based HAematological Registry for Observational Studies (PHAROS).

作者信息

Verelst Silvia G R, Blommestein Hedwig M, De Groot Saskia, Gonzalez-McQuire Sebastian, DeCosta Lucy, de Raad Johan B, Uyl-de Groot Carin A, Sonneveld Pieter

机构信息

Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands.

Erasmus School of Health Policy and Management, Erasmus University Rotterdam, Rotterdam, The Netherlands.

出版信息

Hemasphere. 2018 May 4;2(4):e45. doi: 10.1097/HS9.0000000000000045. eCollection 2018 Aug.

Abstract

Registry data are important for monitoring the impact of new therapies on treatment algorithms and outcomes, and for guiding clinical decision making in multiple myeloma (MM). This observational study analyzed real-world data from patients in the Population-based HAematological Registry for Observational Studies who were treated for symptomatic MM from 2008 to 2013 in the Netherlands. The primary endpoint was overall survival (OS) from initiation of first-line treatment. Secondary endpoints included OS and progression-free survival per treatment line, treatment patterns, and treatment response. Between 2008 and 2013, 917, 583, 283, and 139 patients had initiated first, second, third, and fourth treatment lines, respectively. Thalidomide-based regimens were the most frequently used first-line treatment (66%); bortezomib- and lenalidomide-based regimens were most often used in the second line (41% and 27%, respectively). The median OS (95% confidence interval) ranged from 37.5 months (34.8-41.8 months) in the first line to 9.2 months (6.2-12.3 months) in the fourth line. Univariate analyses showed that survival benefits were most apparent in younger patients (≤65 vs >65 years). These analyses provide important real-world information on treatment patterns and outcomes in patients with MM.

摘要

登记数据对于监测新疗法对治疗方案和治疗结果的影响,以及指导多发性骨髓瘤(MM)的临床决策非常重要。这项观察性研究分析了来自荷兰基于人群的血液学观察性登记处中2008年至2013年接受症状性MM治疗的患者的真实世界数据。主要终点是从一线治疗开始的总生存期(OS)。次要终点包括每条治疗线的OS和无进展生存期、治疗模式以及治疗反应。在2008年至2013年期间,分别有917、583、283和139例患者开始了第一、第二、第三和第四线治疗。基于沙利度胺的方案是最常用的一线治疗方案(66%);基于硼替佐米和来那度胺的方案最常用于二线治疗(分别为41%和27%)。OS的中位数(95%置信区间)从一线治疗的37.5个月(34.8 - 41.8个月)到四线治疗的9.2个月(6.2 - 12.3个月)不等。单因素分析表明,生存获益在年轻患者(≤65岁与>65岁)中最为明显。这些分析提供了关于MM患者治疗模式和治疗结果的重要真实世界信息。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/673e/6746001/03df1f39d82c/hs9-2-e045-g003.jpg

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