EMMY 纵向队列研究:随着更多治疗方案的出现,用真实世界的数据描述多发性骨髓瘤的管理和疗效。

Q4 Health Professions
Clinical hematology international Pub Date : 2024-07-23 eCollection Date: 2024-01-01 DOI:10.46989/001c.121371
Olivier Decaux, Ronan Garlantézec, Karim Belhadj-Merzoug, Margaret Macro, Laurent Frenzel, Aurore Perrot, Philippe Moreau, Bruno Royer, Denis Caillot, Xavier Leleu, Mohamad Mohty, Lionel Karlin, Pierre Feugier, Sophie Rigaudeau, Jean Fontan, Cécile Sonntag, Laure Vincent, Thomas Chalopin, Herve Avet Loiseau, Zakaria Maarouf, Louni Chanaz, Nathalie Texier, Cyrille Hulin
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引用次数: 0

摘要

多发性骨髓瘤(MM)患者的治疗管理非常复杂。尽管取得了重大进展,但多发性骨髓瘤仍无法治愈,治疗涉及治疗反应、疾病复发和进一步治疗的循环。目前,支持治疗决策的证据非常有限。因此,EMMY 纵向真实世界研究旨在每年评估法国 MM 的治疗管理,为医生提供证据支持。在每年预设的 3 个月招募期间,将从符合条件的患者病历中确定其身份。年龄≥18 岁、确诊为无症状 MM 且需要系统治疗的成人患者将符合条件。研究将描述主要目标,即 MM 治疗管理的演变,以及对以下结果的影响:下次治疗时间(TTNT)、无进展生存期(PFS)和总生存期(OS)。该研究计划在 6 年内招募 5000 名患者:每年招募 700 到 900 名患者。EMMY 是一个收集真实世界数据的独特机会,可用于描述不断发展的 MM 治疗情况并记录法国的治疗结果。这些数据将提供 MM 管理各个方面的年度快照。这些知识将为医生的治疗决策提供真实的循证数据,并最终改善 MM 患者的治疗。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The EMMY longitudinal, cohort study: real-world data to describe multiple myeloma management and outcomes as more therapeutic options emerge.

The therapeutic management of patients with multiple myeloma (MM) is complex. Despite substantial advances, MM remains incurable, and management involves cycles of treatment response, disease relapse, and further therapy. Currently, evidence to support the therapeutic decision is limited. Thus, the EMMY longitudinal, real-world study was designed to annually assess therapeutic management of MM in France to provide evidence to support physicians. During an annual prespecified 3-month recruitment period, eligible patients will be identified from their medical records. Adults aged ≥18 years diagnosed with symptomatic MM and requiring systemic treatment will be eligible. The primary objective, the evolution of MM therapeutic management, will be described, as well as the impact on the following outcomes: time-to-next treatment (TTNT), progression-free survival (PFS), and overall survival (OS). The study plans to recruit 5000 patients over 6 years: 700 to 900 patients annually. EMMY is a unique opportunity to collect real-world data to describe the evolving MM therapeutic landscape and record outcomes in France. These data will provide annual snapshots of various aspects of MM management. This knowledge will provide physicians with real-life, evidence-based data for therapeutic decision-making and ultimately improve treatment for MM patients.

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CiteScore
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