文章：

解读多发性骨髓瘤的临床试验数据：将研究结果转化为真实世界情境

Interpreting clinical trial data in multiple myeloma: translating findings to the real-world setting

原文发布日期：2018-11-09

DOI: 10.1038/s41408-018-0141-0

类型: Review Article

开放获取: 是

英文摘要：

Substantial improvements in survival have been seen in multiple myeloma (MM) over recent years, associated with the introduction and widespread use of multiple novel agents and regimens, as well as the emerging treatment paradigm of continuous or long-term therapy. However, these therapies and approaches may have limitations in the community setting, associated with toxicity burden, patient burden, and other factors including cost. Consequently, despite improvements in efficacy in the rigorously controlled clinical trials setting, the same results are not always achieved in real-world practice. Furthermore, the large number of different treatment options and regimens under investigation in various MM settings precludes the feasibility of obtaining head-to-head clinical trial data, and there is a temptation to use cross-trial comparisons to evaluate data across regimens. However, multiple aspects, including patient-related, disease-related, and treatment-related factors, can influence clinical trial outcomes and lead to differences between studies that may confound direct comparisons between data. In this review, we explore the various factors requiring attention when evaluating clinical trial data across available agents/regimens, as well as other considerations that may impact the translation of these findings into everyday MM management. We also investigate discrepancies between clinical trial efficacy and real-world effectiveness through a literature review of non-clinical trial data in relapsed/refractory MM on novel agent−based regimens and evaluate these data in the context of phase 3 trial results for recently approved and commonly used regimens. We thereby demonstrate the complexity of interpreting data across clinical studies in MM, as well as between clinical studies and routine-care analyses, with the aim to help clinicians consider all the necessary issues when tailoring individual patients’ treatment approaches.

摘要翻译： 

近年来，多发性骨髓瘤（MM）患者的生存期获得显著改善，这与多种新药和疗法的推出及广泛使用，以及持续或长期治疗这一新兴治疗模式密切相关。然而，在社区医疗环境中，这些疗法和方法可能存在局限性，涉及毒性负担、患者负担及成本等因素。因此，尽管在严格控制的临床试验环境中疗效有所改善，但在实际临床实践中并非总能达到相同结果。此外，在多发性骨髓瘤的各种治疗场景中，大量不同的治疗选择和方案正在研究中，这使得获得头对头临床试验数据的可行性受限，且存在使用跨试验比较来评估不同方案数据的倾向。然而，包括患者相关、疾病相关和治疗相关因素在内的多个方面可能影响临床试验结果，并导致研究间差异，从而混淆数据间的直接比较。在本篇综述中，我们探讨了在评估现有药物/方案的临床试验数据时需要注意的各种因素，以及可能影响将这些研究结果转化为日常多发性骨髓瘤治疗的其他考量。我们还通过文献回顾，基于新药方案在复发/难治性多发性骨髓瘤中的非临床试验数据，研究了临床试验疗效与实际临床效果之间的差异，并在近期获批和常用方案的3期试验结果背景下评估这些数据。由此，我们阐述了在多发性骨髓瘤的临床研究之间，以及临床研究与常规治疗分析之间解读数据的复杂性，旨在帮助临床医生在为患者量身定制治疗方案时综合考虑所有必要问题。

原文链接：

Interpreting clinical trial data in multiple myeloma: translating findings to the real-world setting