文章：

临床试验环境之外的多发性骨髓瘤患者管理：理解疗效、安全性和耐受性与生活质量之间的平衡

Management of patients with multiple myeloma beyond the clinical-trial setting: understanding the balance between efficacy, safety and tolerability, and quality of life

原文发布日期：2021-02-18

DOI: 10.1038/s41408-021-00432-4

类型: Review Article

开放获取: 是

英文摘要：

Treatment options in multiple myeloma (MM) are increasing with the introduction of complex multi-novel-agent-based regimens investigated in randomized clinical trials. However, application in the real-world setting, including feasibility of and adherence to these regimens, may be limited due to varying patient-, treatment-, and disease-related factors. Furthermore, approximately 40% of real-world MM patients do not meet the criteria for phase 3 studies on which approvals are based, resulting in a lack of representative phase 3 data for these patients. Therefore, treatment decisions must be tailored based on additional considerations beyond clinical trial efficacy and safety, such as treatment feasibility (including frequency of clinic/hospital attendance), tolerability, effects on quality of life (QoL), and impact of comorbidities. There are multiple factors of importance to real-world MM patients, including disease symptoms, treatment burden and toxicities, ability to participate in daily activities, financial burden, access to treatment and treatment centers, and convenience of treatment. All of these factors are drivers of QoL and treatment satisfaction/compliance. Importantly, given the heterogeneity of MM, individual patients may have different perspectives regarding the most relevant considerations and goals of their treatment. Patient perspectives/goals may also change as they move through their treatment course. Thus, the ‘efficacy’ of treatment means different things to different patients, and treatment decision-making in the context of personalized medicine must be guided by an individual’s composite definition of what constitutes the best treatment choice. This review summarizes the various factors of importance and practical issues that must be considered when determining real-world treatment choices. It assesses the current instruments, methodologies, and recent initiatives for analyzing the MM patient experience. Finally, it suggests options for enhancing data collection on patients and treatments to provide a more holistic definition of the effectiveness of a regimen in the real-world setting.
 

摘要翻译： 

随着随机临床试验中引入基于多种新药的复杂联合方案，多发性骨髓瘤（MM）的治疗选择日益增加。然而，在真实世界环境中应用这些方案（包括其实施可行性和患者的依从性）可能因患者个体差异、治疗相关因素及疾病特征而受到限制。此外，约40%的真实世界MM患者不符合作为药物批准依据的III期研究的入组标准，导致这类患者缺乏具有代表性的III期数据。因此，治疗决策必须基于临床试验疗效和安全性之外的额外考量进行个体化制定，例如治疗可行性（包括就诊频率）、耐受性、对生活质量（QoL）的影响以及合并症的影响。对真实世界MM患者而言，疾病症状、治疗负担与毒性、维持日常活动的能力、经济负担、治疗及治疗中心的可及性、治疗便利性等多种因素均至关重要。所有这些因素都是生活质量和治疗满意度/依从性的驱动因素。值得注意的是，鉴于MM的异质性，不同患者对其治疗的核心考量因素和治疗目标可能存在不同认知。随着治疗进程推进，患者视角/目标亦可能随之变化。因此，治疗"疗效"对不同患者具有不同含义，在个体化医疗背景下的治疗决策必须以患者个人对"最佳治疗方案"的综合定义为指导。本综述总结了决定真实世界治疗方案时必须考虑的各种重要因素和实践问题，评估了当前用于分析MM患者体验的工具、方法及最新研究计划，并提出了加强患者和治疗数据收集的方案建议，以在真实世界环境中更全面地定义治疗方案的"有效性"。

原文链接：

Management of patients with multiple myeloma beyond the clinical-trial setting: understanding the balance between efficacy, safety and tolerability, and quality of life