Interpreting clinical trial data in multiple myeloma: translating findings to the real-world setting.
Interpreting clinical trial data in multiple myeloma: translating findings to the real-world setting.
复制标题
DOI：
10.1038/s41408-018-0141-0
复制
发表时间：
2018-11-09
影响因子：
12.8
通讯作者：
Anderson KC
中科院分区：
文献类型：
--
作者：
Richardson PG;San Miguel JF;Moreau P;Hajek R;Dimopoulos MA;Laubach JP;Palumbo A;Luptakova K;Romanus D;Skacel T;Kumar SK;Anderson KC
近年来，随着多种新型药物和方案的引入和广泛使用，以及持续或长期治疗的新治疗模式的出现，多发性骨髓瘤（MM）的生存率有了实质性的改善。然而，这些疗法和方法在社区环境中可能存在局限性，与毒性负担、患者负担和其他因素（包括费用）有关。因此，尽管在严格控制的临床试验环境中疗效有所改善，但在现实世界的实践中并不总是取得相同的结果。此外，在各种MM环境中，大量不同的治疗方案和方案正在研究中，这排除了获得正面临床试验数据的可行性，并且存在使用交叉试验比较来评估不同方案数据的诱惑。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都可能影响临床试验结果，并导致研究之间的差异，从而混淆数据之间的直接比较。在这篇综述中，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，以及可能影响这些发现转化为日常MM管理的其他考虑因素。我们还通过对基于新型药物的治疗方案的复发/难治性MM的非临床试验数据的文献回顾，研究了临床试验疗效与实际疗效之间的差异，并在最近批准和常用方案的3期试验结果的背景下对这些数据进行了评估。因此，我们展示了在MM临床研究中解释数据的复杂性，以及临床研究和常规护理分析之间的复杂性，目的是帮助临床医生在定制个体患者的治疗方法时考虑所有必要的问题。
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.
登录
查看更多内容
影响因子：
10.1
作者：
Engelhardt, Monika;Domm, Anne-Saskia;Waesch, Ralph
通讯作者：
Waesch, Ralph
复发/难治性多发性骨髓瘤的反应动力学和结果之间的关联：来自Tourmaline-MM1的分析。
影响因子：
11.4
作者：
Garderet L;Laubach JP;Stoppa AM;Hari P;Cavo M;Ludwig H;Mateos MV;Luptakova K;Lin J;Yung G;van de Velde H;Berg D;Moreau P;Richardson PG
通讯作者：
Richardson PG
影响因子：
8.8
作者：
Booth, C. M.;Tannock, I. F.
通讯作者：
Tannock, I. F.
影响因子：
11.4
作者：
Durie, B. G. M.;Harousseau, J-L;Rajkumar, S. V.
通讯作者：
Rajkumar, S. V.
影响因子：
51.1
作者：
Dimopoulos, Meletios A.;Moreau, Philippe;Chng, Wee-Joo
通讯作者：
Chng, Wee-Joo