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多发性骨髓瘤的真实世界结局:捷克单克隆丙种球蛋白病登记处的回顾性分析

Real-world Outcomes of Multiple Myeloma: Retrospective Analysis of the Czech Registry of Monoclonal Gammopathies.

作者信息

Hájek Roman, Jarkovsky Jiri, Maisnar Vladimír, Pour Ludek, Špička Ivan, Minařík Jiri, Gregora Evžen, Kessler Petr, Sýkora Michal, Fraňková Hana, Campioni Marco, DeCosta Lucy, Treur Maarten, Gonzalez-McQuire Sebastian, Bouwmeester Walter

机构信息

Department of Hemato-oncology, University Hospital Ostrava, Ostrava, Czech Republic; Faculty of Medicine, University of Ostrava, Ostrava, Czech Republic.

Institute of Biostatistics and Analyses, Faculty of Medicine and Faculty of Science, Masaryk University, Brno, Czech Republic.

出版信息

Clin Lymphoma Myeloma Leuk. 2018 Jun;18(6):e219-e240. doi: 10.1016/j.clml.2018.04.003. Epub 2018 Apr 17.

Abstract

INTRODUCTION

Real-world data on patient outcomes and treatment patterns in multiple myeloma (MM) are limited.

MATERIALS AND METHODS

The present noninterventional, observational, retrospective analysis of prospectively collected Czech patient medical record data from the Registry of Monoclonal Gammopathies estimated real-world outcomes in adults with a diagnosis of symptomatic MM made between May 2007 and June 2014.

RESULTS

In total, 2446 patients had initiated first-line treatment. The median overall survival since the diagnosis (primary endpoint) was 50.3 months (95% confidence interval, 46.1-54.5 months) and decreased with each successive treatment line. A similar trend was observed for progression-free survival and the depth of response. In line with European guidelines and clinical practice, bortezomib-, thalidomide-, and lenalidomide-based regimens were most commonly used across all treatment lines (42.3%, 28.9%, and 18.4%, respectively). In the first line, bortezomib and thalidomide were used most often, with lenalidomide the most commonly used agent in the relapse setting (second to fourth lines). Exploratory analyses revealed that younger age (≤ 65 years), lower international staging system stage, and previous stem cell transplantation were associated with significant improvements in overall and progression-free survival, especially in the early treatment lines.

CONCLUSION

The present study is the first analysis of Czech data from the Registry of Monoclonal Gammopathies, and it provides important insights into the real-world management of MM for physicians and healthcare providers.

摘要

引言

关于多发性骨髓瘤(MM)患者预后和治疗模式的真实世界数据有限。

材料与方法

本研究对前瞻性收集的来自单克隆丙种球蛋白病登记处的捷克患者病历数据进行了非干预性、观察性、回顾性分析,以评估2007年5月至2014年6月期间确诊为有症状MM的成年患者的真实世界预后。

结果

共有2446例患者开始一线治疗。自诊断以来的中位总生存期(主要终点)为50.3个月(95%置信区间,46.1 - 54.5个月),且随着后续治疗线数的增加而降低。无进展生存期和缓解深度也观察到类似趋势。与欧洲指南和临床实践一致,基于硼替佐米、沙利度胺和来那度胺的方案在所有治疗线中最常使用(分别为42.3%、28.9%和18.4%)。在一线治疗中,硼替佐米和沙利度胺使用最为频繁,而来那度胺是复发情况下(二线至四线)最常用的药物。探索性分析显示,年龄较轻(≤65岁)、国际分期系统分期较低以及既往进行过干细胞移植与总生存期和无进展生存期的显著改善相关,尤其是在早期治疗线中。

结论

本研究是对单克隆丙种球蛋白病登记处捷克数据的首次分析,为医生和医疗服务提供者对MM的真实世界管理提供了重要见解。

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